A panel of independent advisors to the Food and Drug Administration unanimously recommended Thursday that the antibody nirsevimab be approved for use to protect infants from respiratory syncytial virus, the leading cause of hospitalization among newborns.
If the FDA approves nirsevimab, the antibody would become the first medical intervention available in the U.S. that can protect all infants from RSV. The FDA, which is not obligated to follow the recommendation of its advisory panel, is expected to make a final decision on nirsevimab in the third quarter of this year.
Nirsevimab is a monoclonal antibody made by AstraZeneca. The medication would be marketed by Sanofi.
The advisory panel voted 21-0 to recommend its approval.
RSV kills nearly 100 babies in the United States every year.
Infants hospitalized with RSV often require oxygen support, intravenous fluids and are sometimes placed on a ventilator to support their breathing.
The virus is a major public health threat. A surge in RSV infections last year overwhelmed children’s hospitals leading to calls for the Biden administration to declare a public health emergency in response.
RSV circulates at the same time as the flu and Covid-19, which puts added pressure on hospitals.
There is another monoclonal antibody used against RSV called palivizumab. But this antibody is only for preterm infants and those with lung and congenital heart conditions that are high risk of severe disease. Palivizumab also has to be administered monthly.
Nirsevimab, by contrast, would also be administered to healthy infants, who make up a majority of the hospitalizations. It is also given as a single dose, which would make administration easier.
Nirsevimab is not considered a vaccine because it is a monoclonal antibody.
It is unclear whether the federal Vaccines for Children program will provide nirsevimab for uninsured and underinsured children for free because the antibody is regulated as a drug.
Nirsevimab is already approved in Canada, Europe and the United Kingdom.
Nirsevimab was up to 75% effective at preventing lower respiratory tract infections that required medical attention and 78% effective at preventing hospitalizations, according a review by the FDA.
A more conservative estimate by FDA put the antibody’s effectiveness at about 48% against lower respiratory tract infections that required medical attention. This estimate assumed patients with missing data on their health outcomes had lower respiratory tract infections that required medical attention.
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Nirsevimab is administered as a single injection with the dose depending on the infant’s weight. Infants that weigh less than 5 kilograms would receive a 50 mg injection for their first RSV season, and those weighing 5 kilograms or greater would receive a 100 mg injection.
Children less than two years old who remain at risk for severe RSV in their second season would receive a single 200 mg injection of nirsevimab.
The FDA did not identify any safety concerns in its review of nirsevimab.
Other monoclonal antibodies have been associated with serious allergic reactions, skin rashes and other hypersensitivity reactions.
The FDA did not find any cases of serious allergic reactions in the nirsevimab trials and cases of skin rash and hypersensitivity reactions were low in infants who received the antibody. But Dr. Melissa Baylor, an FDA official, said cases of these side effects will likely occur if nirsevimab is approved.
Twelve infants who received nirsevimab in the trials died. None of these deaths were related to the antibody, according to the FDA’s review.
Four died from cardiac disease, two died from gastroenteritis, two died from unknown causes but were likely cases sudden infant death syndrome, one died from a tumor, one died from Covid, one died from a skull fracture, and one died of pneumonia.
“Most deaths were due to an underlying disease,” Baylor said. “none of the deaths appeared to be related to nirsevimab.”
There is very close attention to safety due to historical failures in the development of RSV vaccines. Scientists first tried to develop a vaccine in the 1960s with an inactivated virus, but that shot actually made disease from RSV worse in some children when they received their first natural infection, resulting in the death of two infants.
Manish Shroff, head of patient safety at AstraZeneca, said the company will keep a close eye on the safety of nirsevimab through a large global monitoring system: “Safety is of utmost importance,” he said.
Baylor said there are also unanswered questions about how nirsevimab would interact with vaccines in development that confer protective antibodies to the fetus by administering the shot to the mother.
It’s unclear if giving nirsevimab to infants whose mothers received such RSV vaccines would provide additional protection or create potential safety issues,” Baylor said.
Qilu Pharmaceutical, which makes and markets cisplatin injections in China, received FDA permission to export the drug to the U.S. market weeks ago, a document shows.
A letter dated May 24 from Qilu’s deputy general manager notified health care professionals of the approval.
Qilu is coordinating with a Toronto-based company, Apotex, to distribute 50-milligram cisplatin vials in the U.S.
Health care providers can begin ordering the drug Tuesday through their wholesalers.
Cisplatin is a generic drug that has been available for decades in the U.S. and is distributed by several approved manufacturers. Those manufacturers have been unable to keep up with demand. Qilu’s version of cisplatin is not approved in the U.S.
Qilu, which is headquartered in the city of Jinan in Shandong province, says it is one of the 10 largest drug manufacturers in China.
The FDA told CNBC this week the agency was considering imports of unapproved chemotherapy drugs, but it did not at that time disclose the names of any manufacturers who might provide that medication.
An FDA spokesperson said the agency assesses the quality of unapproved drug imports to make sure they are safe for U.S. patients.
Doctors say some cancer patients could die if the national shortage of drugs such as cisplatin is not resolved soon. At least 13 other cancer drugs are in short supply across the U.S.
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The cancer drug shortages have forced some hospitals to ration medications by reducing the dosage to extend the supply and prioritizing patients who have a better chance of being cured.
Cisplatin is widely used to treat testicular, lung, bladder, cervical and ovarian cancers among other disease states. Up to 20% of cancer patients are treated with cisplatin and other platinum-based chemotherapy drugs, according to the National Cancer Institute.
The World Health Organization says the drug is an essential part of basic health care.
The national shortage of cisplatin began in February after a pharmaceutical company based in India temporarily halted production for the U.S. market.
Intas Pharmaceuticals decided to temporarily stop production after an FDA inspection last year found a “cascade of failure” in its quality control unit.
A spokesperson for Intas told CNBC this week the company is working with the FDA to restart production for the U.S., but no date has been set yet.
Illumina Inc. ILMN, -9.08%
shareholders voted Thursday to elect one of activist investor Carl Icahn’s nominees to its board, and voted against the re-election of company Chairman John Thompson, according to media reports on Thursday. The genomics company has been engaged in a proxy battle with Icahn since March, who was seeking to unseat its chairman and chief executive and add three of his own candidates to the board. Illumina had blasted back that Icahn’s board nominees were “unqualified,” urging shareholders to reject all three and instead support its “highly qualified” nominees. Icahn had said that Chief Executive Francis de Souza and other “protectors” seem “dead set on destroying the company.” Illumina had defended its own track record. “Illumina is the only pure-play genomics company with profitable revenue growth,” it said in an early May statement. Icahn started the proxy fight with Illumina in March, criticizing the company’s plan to buy cancer-screening company Grail Inc. But Icahn himself is in a weakened position, under fire from a recent report from short seller Hindenburg Research accusing the firm of inflating asset values and causing his company to trade at a large premium. The report has cost IEP $10.9 billion in lost market cap since it was published on May 2. Last week, the storied investor admitted he was wrong to take a huge short position that led to losses of $9 billion. And earlier this month, IEP disclosed a federal probe into its corporate governance and other issues. It’s not clear if that was related to the Hindenburg report.
The committee unanimously said the vaccine efficacy data was sufficient. Ten of the advisors said the safety data on Pfizer’s shot was adequate, while four said it was not.
“If the vaccine actually lives up to the data we’ve seen today, I can guarantee many infants and their parents will breathe easier in the coming years,” said Dr. Jay Portnoy, medical director at the Children’s Mercy Hospital in Kansas City, after voting in favor of the safety and efficacy of the shot.
Dr. Paul Offit, a vaccine expert at the Children’s Hospital of Philadelphia, said he doesn’t believe there is enough data that indicates the safety of the vaccine is “reassuring.”
“If you’re in any sense risking premature births with this vaccine, I think there’ll be a big price to pay,” said Offit, who voted against the shot’s safety data.
Adam Berger, the director of clinical and healthcare research policy at the National Institutes of Health, voted in favor of the shot’s safety and efficacy, but said Pfizer’s post-marketing studies need to examine the risk of premature births.
Post-marketing refers to studies conducted on a product after it receives FDA approval.
The FDA typically follows the advice of its advisory committees but is not required to do so. The agency is slated to make a final decision on the shot in August, right before RSV season in the fall.
If approved, Pfizer’s jab would become the world’s first vaccine that protects infants against RSV, a goal scientists have been working toward for decades.
The FDA earlier this month approved the first RSV shot for adults ages 60 and older from GlaxoSmithKline. The agency is expected to make a decision within weeks on Pfizer’s other RSV shot for that same age group.
RSV is a common respiratory infection that causes cold-like symptoms. Older adults and younger children are particularly vulnerable to more severe RSV infections.
Each year, the virus kills 6,000 to 10,000 seniors and a few hundred children younger than 5, according to the Centers for Disease Control and Prevention. About one out of every 100 children younger than 6 months of age with an RSV infection may need to be hospitalized, the CDC said.
Pfizer’s shot for infants is administered to expectant mothers in the late second or third trimester of their pregnancy. The single-dose vaccine triggers antibodies that are passed to the fetus, which provides it with protection against RSV from birth through the first six months of life.
A phase three trial found Pfizer’s shot was nearly 82% effective at preventing severe disease from RSV in newborns during the first 90 days of life. The shot was also about 70% effective during the first six months of the baby’s life.
But the advisory panel’s concerns stemmed from safety data in that trial.
A slightly higher number of premature births occurred among mothers who took the shot compared to those who received a placebo: 5.7% versus 4.7%, respectively.
Both Pfizer and the FDA said the difference does not appear to be statistically significant.
Most infants, even when born prematurely, were also delivered after 34 weeks of pregnancy, just a few weeks shy of their due dates.
But Dr. Hana El Sahly, who chairs the FDA advisory committee, said “even if it is late preterm delivery, the fact that we’re putting them into preterm delivery while we’re sitting here debating the matter intellectually is not trivial.”
Premature deliveries aren’t a new issue when it comes to RSV vaccines for infants.
Pfizer’s rival GSK halted its own trial on its RSV shot for newborns after noticing concerning data on preterm births and neonatal deaths, or when a baby dies during the first 28 days of life.
Offit also said GSK’s trial is “hanging over” Pfizer’s own RSV shot for infants.
“If GSK truly abandons a program on a similar, almost identical vaccine, that is going to hang over [Pfizer’s] program,” he said during the meeting. “I think it does need to be addressed.”
Portnoy added preterm deliveries could potentially dampen the benefits of the shot.
“The problem is if the child is born earlier, that also reduces the efficacy of the treatment because earlier birth means less antibodies are transferred,” he said. “So, this is a very complex thing because now the harm actually makes the benefit less so. There’s an interaction between the two.”
But some panel members cast doubt on whether there’s a clear causal relationship between the vaccine and preterm births.
“Am I concerned about the preterm birth imbalance? Yes. Am I convinced that it’s real? No,” said Dr. Daniel Feikin, a scientific advisor and respiratory diseases consultant, who voted in favor of the shot’s safety.
Representatives from Pfizer also pushed back on the safety concerns, emphasizing the benefits of the shot outweigh the risks.
“Certainly, in our eyes, there is no definitive evidence to suggest there is a risk of prematurity,” said Dr. William Gruber, Pfizer’s senior vice president of vaccine clinical research and development. “So the question is, do you hold hostage the potential benefits of the vaccine for something which you have no statistical significance at this point?”
Some of the participants’ children also had low birth weights and experienced developmental delays, the FDA staff review said.
Most of the more than 3,000 mothers who received the shot in the trial experienced mild to moderate adverse reactions, according to the FDA staff’s review of data.
The most common reactions were fatigue, muscle pain, headache and pain at the injection site. Most reactions resolved within three to four days after vaccination, the staff review noted.
Since 2020, nearly 30 states have used a Bluetooth system developed by Apple and Google to track the spread of Covid infections and send push alerts to any smartphone user who came in close contact with a person who tested positive for the virus.
On Thursday, the organization said “the majority of states” stopped using the exposure notification system after the Biden administration ended the public health emergency on May 11.
APHL added it will no longer support the system, which aimed to helpmillions of Americans trace their exposures and make decisions about isolating and testing for the virus.
In a joint statement, Apple and Google did not address states’ decisions to stop using the system.
The tech giants told CNBC the system helped public health departments fight Covid in a way that preserves privacy, referring to how it tracks infections without collecting the location or identity of users.
“These systems were timed to shut down on the same date that the nation’s COVID-19 State of Emergency ends,” the California Department of Public Health said in a statement late Thursday.
Several states used the system to create apps that smartphone users could download, such as CA Notify and WA Notify.
States also provided exposure notifications through a built-in feature on Apple and Google’s operating systems.
For that method, state health departments had to submit a configuration file with their contact information and Covid guidance to Apple and Google. The two tech companies would use the file to set up a feature on phones that users could activate to receive notifications.
On Friday, some Apple users who opted in for that feature received push alerts informing them that their iPhones “will no longer log nearby devices and you won’t be notified of possible exposures.”
One Apple user shared in a Twitter post that their alert said, “Your Health Authority Turned Off Exposure Notifications.”
But not all Apple and Google users in states that stopped using the exposure notification system have received similar alerts, as of Friday afternoon.
Neither Apple or Google addressed why some users received alerts while others did not.
There is no clear tally of how many Americans activated the exposure notification feature on their phones or downloaded apps over the past three years.
Virginia estimates that more than 3 million users have downloaded the state’s app or used the notification feature since those tools launched in 2020.
New Mexico said the “majority” of residents activated the notification feature on their phones. More than 1.5 million alerts were sent to users who may have been exposed to Covid, according to the state.
Washington said the state generated more than 2.5 million exposure alerts through its app or the notification feature.
Researchers in Washington found that the state’s notification tools saved an estimated 30 to 120 lives and likely prevented about 6,000 Covid cases during the first four months after they launched in November 2020.
Despite these benefits, some Americans have been skeptical of the Covid exposure notification tools.
A 2021 report by the U.S. Government Accountability Office said that the public expressed concerns about privacy. The report said the public may not trust both local governments and technology companies to handle sensitive health information.
State decisions to end Covid exposure notifications are part of a broader shift in how the country responds to the pandemic.
Health departments last year loosened Covid restrictions like masking and social distancing as more Americans got vaccinated and boosted against the virus.
That culminated in the end of the public health emergency, which phased out much of the funding and flexibility that helped expand Covid testing, insurance coverage and access to care during the pandemic.
Still, more than 1,000 Americans are still dying each week from Covid, according to the Centers for Disease Control and Prevention.
Swedish Orphan Biovitrum AB SE:SOBI on Wednesday announced that it has entered into an agreement to acquire U.S.-based CTI BioPharma Corp. CTIC in a deal valued at $1.7 billion. The Stockholm-based drugmaker said it will offer $9.10 for each share of CTI, which focuses on blood-related cancers and rare diseases. The offer represents a premium of 88.8% over CTI’s the Tuesday closing price of $4.82 per share. “CTI represents a perfect fit for Sobi’s haematology franchise today, adding a powerful and highly differentiated new product that will make a significant difference for patients”, said Guido Oelkers, president and…
Novavax Inc. NVAX reported positive results from three vaccine trials on Tuesday, one targeting both COVID and flu, one for stand-alone flu and one a high-dose COVID vaccine. All three candidates produced robust immune response in Phase 2 trials and showed a reassuring preliminary safety profile, said Novavax. “The stand-alone influenza vaccine candidate achieved statistically significant hemagglutination inhibition (HAI) antibody responses 31 to 56% higher for all four strains compared to Fluad,” said the company. The highest-dose stand-alone COVID vaccine achieved statistically significant neutralization responses that…
Tedros Adhanom Ghebreyesus, Director-General of the World Health Organization (WHO), speaks during a news conference in Geneva, Switzerland, December 20, 2021.
Denis Balibouse | Reuters
The spread of Covid-19 is no longer a global public health emergency, the World Health Organization declared Friday.
“For more than a year, the pandemic has been on a downward trend with population immunity increasing from vaccination and infection, mortality decreasing, and the pressure on health systems easing,” WHO Director-General Tedros Adhanom Ghebreyesus said at a news conference in Geneva.
“This trend has allowed most countries to return to life as we knew it before Covid-19,” Tedros said. “It is therefore with great hope that I declare Covid-19 over as a global health emergency.”
Nearly 7 million people have died from the virus worldwide since the WHO first declared the emergency on Jan. 30, 2020, according to the U.N. organization’s official data. Tedros said the true death toll is at least 20 million.
The WHO’s decision comes as the U.S. is set to end its national public health emergency on Thursday.
Tedros said there is still a risk that a new variant could emerge and cause another surge in cases. He warned national governments against dismantling the systems they have built to fight the virus.
“This virus is here to stay. It’s still killing and it’s still changing,” he said.
But the WHO chief said the time has come for countries to transition from an emergency response to managing Covid like other infectious diseases.
Covid was first observed in Wuhan, China, in December 2019, when several patients began to experience pneumonia symptoms with unknown cause.
Covid moved rapidly around the globe in early 2020 leading to an unprecedented shutdown of international travel and border closures as countries unsuccessfully tried to prevent the spread of the virus.
Covid devastated the elderly and other vulnerable populations and ravaged hospitals that didn’t have the bed capacity or supplies to manage the sudden surge of suffering and death.
Many national governments shut down public life in a desperate effort to stop the death, leading to a severe economic downturn and social disruption, the long-term consequences of which likely won’t be fully understood for years to come.
“Covid-19 has been so much more than health crisis,” Tedros said. “It has caused severe economic upheaval, erasing trillions from GDP, disrupting travel and trade, shattering businesses and plunging millions into poverty,” he said.
“It has caused severe social upheaval with borders closed, movement restricted, schools shut and millions of people experiencing loneliness, isolation, anxiety and depression,” Tedros said.
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China has faced fierce criticism for not alerting the world earlier, an allegation Beijing denies. Critics have also accused the WHO of relying too much on information from Beijing at the outset of the pandemic.
More than three years later, the origins of the virus are still a hotly contested mystery. Scientists, government officials and the general public continue to debate whether Covid spilled over to humans from an infected animal, or leaked from a lab in China.
The U.S. intelligence community is divided in its assessment of Covid’s origins.
The U.S. government, allied nations and the WHO have criticized the Chinese government for not providing transparent access to data that could help determine how the pandemic started.
Small Business Innovative Research Program Provides $175,000 in Funding for Chi Botanic’s Sustainable Vanilla Technology
ALAMEDA, Calif., May 4, 2023 (Newswire.com)
– ChiBotanic Inc. is pleased to announce that its research and commercialization project for a new cell-cultured Vanilla 2.0 technology for a plant-based full-spectrum natural vanilla flavor has been awarded a highly competitive Small Business Innovative Research (SBIR) Phase I Award of $175,000 from the United States Department of Agriculture (USDA) National Institute of Food and Agriculture (NIFA).
The proposal, “Vanilla 2.0: A reliable, sustainable, domestic supply of authentic Vanilla planifolia flavor and fragrance,” will help support Chi Botanic’s efforts to solve the many problems facing the natural vanilla supply chain, including a very difficult, laborious, slow-growing agricultural crop limited to tropical climates representing biodiversity hotspots. Political instability and extreme weather in regions where vanilla is grown further contribute to severe price fluctuations.
The technology Chi Botanic is creating will achieve a Vanilla planifolia plant cell culture that can be grown inexpensively anywhere and produce high concentrations of vanillin plus the many other flavor and fragrance metabolites of real vanilla. Major advantages to creation of a plant cell suspension culture specialized for production of the distinctive blend of vanilla phenylpropanoids are as follows:
Increased value of a complex, plant-based vanilla product over the inferior synthetic vanillin that makes up 99% of the vanilla flavor on the market today.
Unlike other synthetic biology-based methods, the system will provide the sustainability of fermentation-type methods, yet the end-product will be as complex as pure vanilla extract, which the FDA strictly defines as from vanilla seed pods. Thus, the Chi Botanic product will initially be labeled as “natural vanilla flavor” due to the source plant (Vanilla planifolia) from which the culture is derived, though not strictly from seed pods.
Economically viable, domestic production with tight control of quality and composition. Co-location with product manufacturing possible.
Faster growing, processing, and production times compared to farm-grown vanilla.
Over 18,000 products contain vanillin, yet less than 1% is sourced from the plant (Sethi, 2017). Chi Botanic’s own market research with potential enterprise customers has proven an eagerness to assess and willingness to pay a premium for our proposed product, with one of the world’s largest suppliers of vanilla quoted as “dreaming of the day” when this could be possible.
For more information, visit the corporate website at www.chibotanic.com or email info@chibotanic.com. Digital photos are available.
An Eli Lilly and Company pharmaceutical manufacturing plant is pictured at 50 ImClone Drive in Branchburg, New Jersey, March 5, 2021.
Mike Segar | Reuters
The Alzheimer’s treatment donanemab, which is made by Eli Lilly, significantly slowed progression of the mind-robbing disease, according to clinical trial data released Wednesday by the company.
Patients who received the monthly antibody infusion during an 18-month study demonstrated a 35% slower decline in memory, thinking and their ability to perform daily activities compared with those who did not receive the treatment, Eli Lilly’s data showed.
Patients who took donanemab were 39% less likely to progress to the next stage of the disease during the study, according to the trial results.
But the treatment’s benefits will have to be weighed against the risk of brain swelling and bleeding that can be serious and even fatal in rare cases. Three participants in the trial died from these side effects.
Eli Lilly’s stock was up more than 6% in premarket trading Wednesday.
Lilly plans to apply for Food and Drug Administration approval of donanemab as soon as this quarter, according to the company. The trial studied individuals in the early stages of Alzheimer’s who had a confirmed presence of brain plaque associated with the disease.
Dr. Daniel Skovronsky, Lilly’s chief scientific and medical officer, said donanemab demonstrated the highest level of efficacy of any Alzheimer’s treatment in a clinical trial. The company is working to get donanemab approved and on the market as quickly as possible, he said.
And Skovronsky believes the FDA feels the same sense of urgency.
“Every day that goes by, there are some patients who pass through this early stage of Alzheimer’s disease and become more advanced and they won’t benefit from treatment,” he said in an interview with CNBC. “That’s a very pressing sense of urgency.”
Lilly previously applied for expedited approval of donanemab.
The FDA rejected that request in January and asked the company for more data on patients who received the antibody for at least 12 months. Lilly said the data wasn’t available at the time because many patients were able to stop dosing at six months because the treatment cleared plaque quickly.
Nearly half of patients — 47% — who received donanemab showed no disease progression a year after treatment began, compared with 29% who did not receive the antibody, according to the data released Wednesday.
More than half of patients completed the treatment in the first year and 72% completed it in 18 months due to clearance of brain plaque.
In a separate measure, patients who received donanemab showed 40% less decline in their ability to conduct daily activities at 18 months. This means they could better manage finances, drive, pursue hobbies and hold conversations than those who did not receive the treatment.
“These are the strongest phase 3 data for an Alzheimer’s treatment to date. This further underscores the inflection point we are at for the Alzheimer’s field,” said Maria Carrillo, the Alzheimer’s Association chief scientific officer, in a statement.
Donanemab targets brain plaque associated with Alzheimer’s disease. The treatment significantly reduced the plaque as early as six months after treatment, according to Lilly. Many patients saw such significant reductions that they tested negative for plaque presence on their PET scans, according to the company.
Donanemab cleared the plaque at six months in 34% of patients who had intermediate levels of a protein called tau that can become toxic and kill neurons. At 12 months, donanemab cleared the plaque in 71% of patients with the same tau levels.
“It should be unequivocal that drugs that remove plaque, particularly if you can remove plaque completely and do it quickly, can lead to very significant clinical benefits for patients,” Skovronsky said in an interview.
“The earlier in the disease course you do this, the more you can slow the disease,” he said.
Dr. Eric Reiman, executive director of the Banner Alzheimer’s Institute, said the results do not necessarily mean the plaque is completely gone, but donanemab cleared the plaque to such a degree that the treatment removed measurable evidence of it. The Banner Alzheimer’s Institute had two physicians who participated in the donanemab trial as principal investigators.
Donanemab can cause brain swelling and bleeding in patients that in some cases can be severe and even fatal. Three trial participants died from these side effects, according to Lilly.
These types of side effects have been observed in other Alzheimer antibody treatments such as Eisai and Biogen’s Leqembi, which received expedited FDA approval in January.
Reiman said he’s encouraged by the potential clinical benefit to patients but it’s important to be clear about the risks.
“We also need to be clear that there are side effects, including an uncommon but potentially catastrophic risk,” said Reiman. “And we need to continue to do our best to understand what that risk is for individual patients, to inform patients and family caregivers, and do everything we can to mitigate that risk,” he said.
About 24% of patients who received donanemab showed brain swelling on an MRI, but only 6% displayed actual symptoms. About 31% of patients had small brain bleeds called microhemorrhages, compared with 13.6% among patients who didn’t receive the treatment.
Lilly said the majority of the cases of brain swelling and bleeding were mild to moderate and patients stabilized with the right care, but cautioned that serious and life-threatening events can occur. About 1.6% of the swelling and bleeding cases were serious, according to Lilly.
Skovronsky said every patient would need to have a discussion with their doctor that weighs the potential benefits of donanemab with the possible risks.
“On a population basis, our view is its benefits outweigh risks,” Skovronsky said.
“FDA is the steward of that for the U.S.,” he said of the risk-benefit analysis that will determine whether donanemab wins approval.
Travelers wearing protective masks receive nasal swabs from nurses at a COVID-19 test site inside Terminal B at Los Angeles International Airport (LAX), on Sunday, Nov. 22, 2020.
Bing Guan | Bloomberg | Getty Images
The Biden administration will end its Covid-19 vaccine mandates for federal employees, contractors and international air travelers next week.
The White House said in a statement Monday that those vaccine requirements will end on May 11, the same day the Covid public health emergency expires.
“While vaccination remains one of the most important tools in advancing the health and safety of employees and promoting the efficiency of workplaces, we are now in a different phase of our response when these measures are no longer necessary,” the White House said.
Although Covid cases, hospitalizations and deaths have declined dramatically this year, the virus is still killing more than 1,000 people per week.
The Health and Human Services Department also will start phasing out its vaccine mandate for health-care facilities that participate in Medicare and Medicaid, the White House said. In addition, it will end vaccination requirements for Head Start programs.
And the Department of Homeland Security will lift vaccination requirements for people entering the U.S via its land borders with Canada and Mexico, according to the Biden administration. U.S. citizens, nationals and permanent residents were never subject to those requirements.
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HHS and DHS will provide more details on the end of these vaccine requirements in the coming days, the White House said.
The Biden administration implemented the vaccine requirements for health workers, federal employees, contractors, and international air travelers as part of its drive to boost lackluster vaccination rates and slow the spread of the virus as the delta variant surged in late 2021 followed by omicron in the winter of 2022.
The mandates faced fierce opposition and lawsuits from critics who decried the requirements as government overreach, while the White House stressed they were essential to protect public health.
Seniors with early Alzheimer’s disease will face major hurdles to get treated even if promising new drugs roll out more broadly in the coming years, putting them at risk of developing more severe disease as they wait months or perhaps years for a diagnosis.
The U.S. health-care system is not currently prepared to meet the needs of an aging population in which a growing number of people will need to undergo evaluation forAlzheimer’s, according to neurologists, health policy experts and the companies developing the drugs.
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There are not enough dementia specialists or the needed testing capacity in the U.S. to diagnose everyone who may benefit from a new treatment like Eisai and Biogen‘s Leqembi. After patients are diagnosed, the capacity may not exist — at least initially — to provide the twice monthly intravenous infusions for everyone who is eligible.
Researchers estimate that the wait time from the initial evaluation to the confirmatory diagnostic tests to the infusions could range anywhere from a year and a half to four years or longer. Those months are critical for people with Alzheimer’s.
“The whole process from that time of the family physician conversation to the point of infusion, I worry how long it will take and the complexities of the patient navigating through all of that to successfully get to the end,” Anne White, president of neuroscience at Eli Lilly, which is developing its own Alzheimer’s treatment, told CNBC.
There are promising innovations in development, such as blood tests and injections that patients would take at home, which could make it significantly easier to get diagnosed and treated in the future.
White also said Lilly is confident that more doctors will get into the field and help to alleviate capacity issues, as awareness grows that medicines are entering the market to treat Alzheimer’s.
But time spent waiting robs early patients of their memory and ability to live independently. Alzheimer’s gets worse with time, and as patients deteriorate into more advanced stages of the disease, they no longer benefit from treatments like Leqembi that are designed to slow cognitive decline early.
More than 2,000 seniors transition from mild to moderate dementia from the disease a day, according to estimates from the Alzheimer’s Association. At that stage, they become ineligible for Leqembi.
The central challenge is that a large and rapidly growing group of people have early memory loss and other thinking problems known as mild cognitive impairment. This condition is often, though not always, a sign of early Alzheimer’s disease.
An estimated 13 million people in the U.S. had mild cognitive impairment last year, according to a study published in the Alzheimer’s and Dementia Journal. As the U.S. population ages, the number of people with this condition is expected to reach 21 million by 2060, the study projected.
The U.S. health-care system will deal with major logistical challenges in diagnosing the growing population of people with early Alzheimer’s — even before patients face potential issues with accessing treatment.
“There’s a very large population of undiagnosed cognitive impairments that need to be evaluated in order to determine if people are eligible for treatment,” said Jodi Liu, an expert on health policy at the Rand Corporation.
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Access to drugs like Leqembi is severely restricted because Medicare for nowwill only cover the $26,500-per-year treatment for people participating in clinical trials. Medicare has promised to provide broader coverage if Leqembi receives full approval from the Food and Drug Administration, which Eisai expects to happen in July.
Eisai has estimated that 100,000 people in the U.S. will be diagnosed and eligible for Leqembi by the third year of the treatment’s rollout. The sum is a fraction of the total population that could benefit.
Those patients could have other options if new treatments emerge from trials with positive marks.
Eli Lilly will publish clinical trial data on its antibody infusion donanemab in the second quarter of this year. If the data is positive, the company will ask the FDA to approve the drug.
Eisai’s U.S. CEO Ivan Cheung and Lilly’s White said during the companies’ respective earnings calls in February that they are focused on working with the U.S. health system to address the challenges of rolling out of Alzheimer’s treatments.
“The primary goal right now during this launch phase […] is really get the market ready in terms of the diagnostic pathway, the infusion capacity, the education on how to monitor for this therapy, get all the hospitals and clinics ready,” Cheung said.
Long lines are expected at the offices of geriatricians, neurologists and radiologists as millions of people with mild cognitive impairment undergo evaluation to diagnose whether they have Alzheimer’s disease.
Demand for geriatricians — doctors who are experts in diseases that affect the elderly — is expected to outstrip the number of specialists available in the field through at least 2035, according to projections from the federal Health Resources and Services Administration.
The American Academy of Neurology told Medicare in a February letter that increased demand for Alzheimer’s treatments will put substantial pressure on neurologists, who will need additional resources. The federal data predicts a substantial shortage of these specialists in rural areas through at least 2035.
“You just look at the neurologists, look at geriatricians — there are fewer and fewer geriatricians per person in the U.S.,” Rand’s Liu said. “It’s just a few number of specialists to do this kind of work.”
White said Lilly has heard stories of patients waiting six to 12 months to see a neurologist or other doctors who treat dementia due to current capacity issues.
The number of radiologists — who also play a role in diagnosing the disease — is expected to decline in the U.S. through 2035 even as demand increases, the data shows.
In a study published in 2017, Liu and other Rand researchers estimated an initial wait of 18 months for patients to get evaluated by a dementia specialist, tested to confirm a diagnosis, and then treated in the first year that an Alzheimer’s antibody treatment becomes available. The wait would decrease to 1.3 months by 2030 as the patient backlog is cleared, they estimated at the time.
But more recent research found that the wait would actually increase as demand created by an aging U.S. population outstrips the supply of specialists.
Patients seeking a first specialist visit could face an initial wait of 20 months, according to a study by researchers at the University of Southern California published in the journal Alzheimer’s and Dementia in 2021. The delay could increase to about four years as early as 2028 and grow longer through 2050, the study found.
The journal is published by the Alzheimer’s Association.
Both studies are based on assumptions made before Leqembi received expedited approval from the FDA in January. Actual wait times could differ from the studies’ projections.
Two types of tests can diagnosis Alzheimer’s disease: PET scans and spinal taps. PET scans are accurate and safe diagnostic tools, but they are also cumbersome and expensive, said Dr. David Russell, a neurologist.
Patients are injected with a tracer that makes brain abnormalities visible to the machine that does the imaging. Tracers have to be made for each patient and used on the same day.
“We don’t have the infrastructure to roll out PET scanning on a major scale,” said Russell, director of clinical research at the Institute for Neurodegenerative Disorders in New Haven, Connecticut. He is the principal investigator on the clinical trials of Leqembi and donanemab at the institute.
Medicare coverage of PET scans for Alzheimer’s patients is also limited right now. The insurance program for seniors will only cover one scan per lifetime, and only when the patient is participating in a clinical trail approved by the federal Centers for for Medicare and Medicaid Services.
“That’s concerning because people may actually test negative at one point but then obviously as they age, they may need to get tested again,” White said.
Early Alzheimer’s disease can also be diagnosed with a spinal tap, in which fluid around the spinal cord is extracted with a catheter and tested. While there’s plenty of capacity to do spinal taps, this option isn’t attractive to many patients due to unfounded fears that it’s painful and dangerous, Russell said.
Though “there’s a lot of resistance” to the procedure, it is well tolerated and safe, he noted.
“There are certainly areas that don’t have a PET scanner, rural areas, so people would need to travel to a health center that has a PET scanner,” Liu said.
In a large, sparsely populated rural state like New Mexico, many patients would have to drive three to five hours to get a PET scan in a city such as Albuquerque, said Dr. Gary Rosenberg, a neurologist and director of the New Mexico Alzheimer’s Disease Research Center.
“It’s not California or the East Coast where everything’s very compressed and people can travel and get to a center pretty easily and go through these kinds of treatments,” Rosenberg said.
The state has an estimated population of 43,000 people with dementia, and there are very few neurologists outside of the Albuquerque area, Rosenberg said. The New Mexico Alzheimer’s Disease Research Center in Albuquerque is one of only three such facilities funded by the federal National Institute of Aging in a vast region stretching west from Texas to Arizona.
To do a PET scan, a tracer has to be made for each patient off-site in Phoenix, flown on a private plane to Albuquerque and used within hours because the tracers have a short shelf life, according to Rosenberg. The whole process costs more than $12,000 per patient, he added.
“It’s logistically going to be very challenging,” Rosenberg said.
After spending months or possibly years waiting to get diagnosed with early Alzheimer’s, patients would then be eligible for intravenous infusions of Leqembi. But the U.S. doesn’t currently have the capacity to give infusions twice monthly for everyone who likely has the disease, Russell said.
“Having an IV infusion every two weeks would sort of ration people to availability and that’s a problem,” Russell said.
The University of New Mexico Hospital is already maxed out with demand for infusion therapies for cancer, rheumatoid arthritis and autoimmune diseases, and could have a “problem” adding new capacity, said Rosenberg.
Intravenous infusions of monoclonal antibodies like Leqembi aren’t difficult to administer, Russell said.
The infrastructure to offer infusions should expand rapidly once industry sees there’s demand for treatments like Leqembi. But the process of building out capacity could still take a couple years, Russell said. He believes big players like CVS will provide infusions for Alzheimer’s disease on a major scale if they see there’s a large and stable market.
“In one sense, capitalism works, and if it looks like that’s going to be the future, I think infusion centers will explode onto the scene,” the neurologist said.
Eisai and Biogen hope to move early Alzheimer’s patients to a single monthly dose of Leqembi after they’ve completed their initial course of twice monthly infusions, which could help alleviate some of the capacity issues with infusions over time. They plan to ask the FDA to approve this plan in early 2024.
Eli Lilly’s Alzheimer’s candidate antibody treatment donanemab is a single monthly dose, potentially making the logistics of administration easier if the drug gets approved. Dr. Dan Skovronsky, Lilly’s chief medical officer, told analysts during the company’s first-quarter earnings call that he expects many patients will be able to stop taking donanemab at 12 months.
Though the projected wait times to get diagnosed and treated are sobering, innovations on the horizon promise to significantly improve access to Alzheimer’s drugs over time.
Blood tests for Alzheimer’s are in development and some are already on the market. Primary-care doctors could administer the tests, which would ease the burden on patients, especially those in rural communities where the closest PET scan machine is hours away.
These tests detect proteins in the blood associated with Alzheimer’s. They promise to help diagnose the disease before people display cognitive symptoms, potentially giving patients the chance to get treated before they suffer irreparable brain damage, according to the National Institutes of Health.
At least three blood tests made by C2N Diagnostics, Quest Diagnostics and Qaunterix are currently on the market. But they are used to evaluate people who are already presenting symptoms and aren’t available on the mass scale needed for the expected increase in Alzheimer’s patients.
C2N’s PrecivityAD test costs $1,250 and is not covered by insurance — though the company has a financial assistance program. Quest Diagnostics’ AD-Detect test costs $650. Quest’s test is covered by some insurance plans but not Medicare at the moment. The company also has a financial assistance program. Quanterix wouldn’t disclose the price of its test, which insurance does not cover.
Right now, these are not stand-alone tests that can definitively diagnose Alzheimer’s. But the tests could help identify the patients who likely have the disease, which would narrow the population that needs further evaluation and reduce wait times for dementia specialists or confirmatory PET scans.
A study in the journal Alzheimer’s and Dementia estimated that a cognitive test combined with a blood test could slash wait times for dementia specialists from 50 months down to 12 months.
Eisai believes that inexpensive blood tests could completely replace PET scans and spinal taps by the fourth year of Leqembi’s rollout. The quicker diagnosiscould increase the number of people eligible for treatment.
Rosenberg said widespread availability of blood tests will allow mobile clinics to go into rural communities and identify who has markers associated with Alzheimer’s. This would allow patients in remote towns avoid the hours-long drive to cities with PET scan machines, Rosenberg said.
“It’s a game changer,” the neurologist said.
Lilly is developing at least two blood tests. The company is already using one test in clinical trials and hopes to commercialize it sometime this year. It is developing a second test through a collaboration with Roche. White said it is reasonable to expect that in a few years blood tests could replace more burdensome PET scans.
Biogen and Eisai are also developing an injectable form of Leqembi which patients could administer themselves with an autoinjector similar to insulin pens, saving the trip to a site that provides intravenous infusions. They plan to ask the FDA to approve these so-called subcutaneous injections in early 2024.
Eli Lilly is also conducting clinical trials on an antibody treatment called remternetug as a self-administered injection. But the promise of injections that can be administered at home could make companies reluctant to invest in building out intravenous infusion capacity, Russell said.
In the future, Alzheimer’s diagnosis and treatment could be folded into routine checkups with a family doctor, Russell said. When people turn 50 and head in to get a colonoscopy or a cholesterol check, the doctor could also run a blood test for Alzheimer’s.
If the test comes back positive, the doctor could then schedule patients for an MRI and get them started on an autoinjector treatment, Russell said.
“That’s going to be the way that we’re looking at it in the not too distant future,” he said.
Carl Icahn speaking at Delivering Alpha in New York on Sept. 13, 2016.
David A. Grogan | CNBC
Carl Icahn on Friday called Illumina’s first-quarter results “very disappointing” and slammed the DNA sequencing company’s new plans to cut costs.
The activist investor, who owns a 1.4% stake in Illumina, is in a heated proxy fight with the company over its 2021 acquisition of cancer test developer Grail.
Icahn and Illumina have been trading jabs for more than a month.
Icahn is seeking seats on Illumina’s board of directors and pushing the company to unwind the Grail acquisition. He is also calling for the San Diego-based company to oust CEO Francis deSouza “immediately.”
Illumina on Tuesday reported quarterly revenue and earnings that topped Wall Street’s expectations.
But the company also posted net income of $3 million for the quarter, which was down more than 96% from the $86 million it raked in during the same period a year ago.
In an open letter Friday to Illumina shareholders, Icahn accused deSouza of “desperately, hilariously and, most of all, unsuccessfully” trying to spin the “decidedly mediocre” quarterly results during a press tour this week.
Icahn pointed to deSouza’s interview on CNBC’s “Squawk Box” on Wednesday, when the CEO touted strong demand for Illumina’s diagnostic testing services.
“Illumina CEO Francis deSouza seems to believe that he can fool all of the people all of the time,” Icahn wrote.
“Those not skilled in deciphering doublespeak might actually get the impression that Illumina was doing well!” he added.
Icahn also said that the price of Illumina shares fell the more its CEO during this week, “clearly signaling dissatisfaction with the earnings report and dissatisfaction with Mr. deSouza’s transparent attempt to put lipstick on a pig.”
Illumina’s stock is down more than 10% since the company reported earnings. Shares closed largely flat Friday after Icahn released his letter.
In that missive, Icahn also took shots at cost-cutting plans Illumina unveiled to improve its shrinking margins. He called those measures “vague” and “extraordinarily unambitious.”
The company on Tuesday said it will enable unnamed “activities” in more cost-effective areas of the world and will use its new NovaSeq X sequencing system to accelerate genomic discoveries, among other efforts.
Those plans will help Illumina reach its adjusted operating margin goals of 24% in 2024 and 27% in 2025, the company said in its earnings release.
Icahn called those margin targets “less than modest.” And he argued that they will “take years to realize, if they are achieved at all.”
The company has projected an estimated 22% operating margin for 2023, down from the 23.8% it reported in 2022.
Illumina reported a negative operating margin of 5.7% for the quarter, down from 15% during the same period a year ago. The company’s gross margins for the period fell to 60.3%, down from 66.6% in the first quarter of 2022.
Illumina did not immediately respond to a request for comment on Icahn’s letter.
Elsewhere in his letter, Icahn slammed deSouza’s positive remarks this week about Illumina’s $7.1 billion acquisition of Grail.
DeSouza had told CNBC the deal “makes sense” because Illumina can significantly expand the market for Grail’s early screening test for different types of cancer.
The CEO also touted Grail’s 100% revenue growth during the quarter compared with the same period a year ago.
But Icahn said the deSouza failed to tell the public about an opinion issued earlier this month by the Federal Trade Commission, which said that the deal would stifle competition and innovation.
The FTC also ordered Illumina to divest itself of the acquisition over those concerns.
The European Commission, the executive body of the European Union, also blocked the deal last year over similar concerns.
Illumina is appealing both orders and expects final decisions in late 2023 or early 2024.
Last week, a U.S. federal appeals court said that it will fast-track its review of Illumina’s challenge of the FTC order.
Icahn’s resistance to the acquisition stems from Illumina’s decision to close the deal without getting approval from those antitrust regulators.
Earlier this month he strongly criticized Illumina and its management for finalizing the “reckless deal,” calling it “a new low in corporate governance.”
Illumina has urged shareholders to reject Icahn’s three board nominees during its annual shareholder meeting scheduled for May 25.
The U.S. Food and Drug Administration said Tuesday it has granted accelerated approval to Biogen Inc.’s torferson, a treatment for a rare form of amyotrophic lateral sclerosis, or ALS.
The accelerated program is used to approve drugs for serious conditions that have an unmet medical need, where a drug is shown to have an effect on an endpoint that is reasonably likely to predict a clinical benefit to patients.
When is it a good time to buy stocks? Some investors would say the current negativity dominating the financial media means you are better off sitting on the sidelines. Others would say it is always a good time to buy stocks, provided you can get them for good prices.
Count John Buckingham, editor of the Prudent Speculator, in the latter camp. He is a value investor with decades of experience. During an interview, he emphasized the importance of remaining disciplined through all market conditions. While he favors the value…
Johnson & Johnson reported adjusted earnings and revenue that topped Wall Street’s expectations on Tuesday, and lifted its full-year forecast.
J&J, whose financial results are considered a bellwether for many health companies, said its first-quarter sales grew 5.6% over the same quarter last year.
The consumer staples giant reported a net loss of $68 million, or 3 cents per share, due to a special one-time charge. That’s compared to a net income of $5.2 billion, or $1.93 per share, for the same period a year ago. Excluding certain items, adjusted earnings per share were $2.68for the period.
Here’s how J&J results compared with Wall Street expectations based on a survey of analysts by Refinitiv:
Earnings per share: $2.68 adjusted, vs. $2.50 expected
Revenue: $24.75 billion, vs. $23.67 billion expected
J&J is now forecasting 2023 sales of $97.9 billion to $98.9 billion, about $1 billion higher than the guidance provided in January. The company raised its full-year adjusted earnings outlook to $10.60 to $10.70 per share, from a previous forecast of $10.45 to $10.65.
CFO Joseph Wolk told CNBC on Tuesday that J&J raised its guidance due to strong growth across all three business sectors — consumer health, pharmaceuticals and medtech.
“If you think about how we started the year and guidance in January, we were responsibly cautious,” he said on “Squawk Box.” “First-quarter growth was much stronger than even fourth-quarter growth for all three business units, and our positions kind of change to responsibly optimistic at this point. We feel very good about 2023.”
The company’s shares rose nearly 2% in premarket trading. The stock is down more than 6% for the year through Monday’s close, putting the company’s market value at roughly $430 billion.
But the New Brunswick, New Jersey-based company entered this earnings season with its shares on the rise after it offered more clarity on the long-running legal fight over its talc-based baby powder products. Earlier this month, J&J proposed to pay nearly $9 billion over the next 25 years to settle thousands of allegations that its baby powder and other talc products caused cancer.
Pills of Misoprostol, used to terminate early pregnancies, are displayed in a pharmacy in Provo, Utah, May 12, 2022.
George Frey | Reuters
New York and California are stockpiling massive quantities of an alternative abortion medication in case a federal judge’s order suspending the Food and Drug Administration’s approval of the main drug mifepristone goes into effect later this week.
New York Gov. Kathy Hochul on Tuesday ordered the state health department to immediately start purchasing 150,000 doses of misoprostol, a five-year supply of the pill. Hochul’s announcement comes a day after Gov. Gavin Newsom said California has secured 2 million pills of misoprostol.
Medication abortions are the most common way to terminate a pregnancy the U.S. Patients typically use a two drug-regimen in which mifepristone is taken first, followed by misoprostol. The second pill is used as a standalone abortion medication in parts of the world.
Misoprostol is FDA approved to treat gastric ulcers, so it will remain on the market if U.S. District Judge Matthew Kacsmaryk’s order suspending mifepristone takes effect at 12 a.m. central time on Saturday.
“One judge in Amarillo, Texas thinks he knows better than thousands of doctors and scientists and experts. And not to mention the countless women who’ve used this medication safely for decades,” Hochul said during a press conference with Planned Parenthood’s New York chapter Tuesday.
“This isn’t just an attack on abortion, it’s an attack on democracy. Courts have never before revoked a science backed decision made by the FDA,” the governor said.
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Hochul said she is working with New York’s legislature to require private insurers to cover misoprostol when it is prescribed off-label for abortions.
The World Health Organization and the American College of Obstetricians and Gynecologists recommend misoprostol as a standalone abortion medication when the two-drug regimen is not available. But misoprostol is not as effective when it is used on its own, according to obstetricians and gynecologists.
The FDA has approved the two-drug abortion regimen through the 10th week of pregnancy. Mifepristone stops the pregnancy from developing further by blocking a hormone called progesterone. Misoprostol induces contractions that empty the uterus.
Patients take 200 milligrams of mifepristone by mouth on day one, and then take 800 micrograms of misoprostol in the cheek pouch 24 to 48 hours later, according to the FDA’s label.
Moderna Inc. said Tuesday it’s working to develop its first bacterial vaccine to protect against Lyme disease, the tick-borne illness that causes a range of painful symptoms, including fever, headaches, fatigue, joint pain and rash.
The biotech MRNA, -2.75%,
whose first product to be approved by the U.S. Food and Drug Administration was its mRNA-based COVID vaccine, said it has two candidates in development to address Lyme disease, named mRNA-1982 and mRNA-1975.
It announced the news at its fourth Vaccine Day, where it offered a full update on its clinical pipeline, which includes vaccines to protect against flu and respiratory syncytial virus, or RSV, as well as HIV, Epstein-Barr virus and herpes simplex virus, among others.
There are about 120,000 cases of Lyme disease in the U.S. and Europe every year, creating a “significant quality of life burden,” the company said in a statement. Rising temperatures are helping the disease spread more easily, and it is difficult to diagnose, because the symptoms are similar to those of many other diseases. It most seriously affects children below the age of 15 and older adults.
“Older adults appear to have higher odds of unfavorable treatment response as compared with younger patients, and neurologic manifestations are more common at presentation for this older adult population,” said the statement.
Tick and Lyme disease season is here, and scientists warn this year could be worse than ever. Dr. Goudarz Molaei joins Lunch Break’s Tanya Rivero to explain what triggered the rapid spread of the disease and how people can avoid being affected. Photo: Kent Wood/Science Source
The mRNA-1982 candidate is designed to create antibodies for Borrelia burgdorferi, the pathogen that causes almost all Lyme disease in the U.S., while mRNA-1975 is designed to elicit antibodies specific to the four major Borrelia species that cause the disease in the U.S. and Europe.
Other new candidates in Moderna’s pipeline include mRNA-1405 and mRNA-1403, which aim to address the enteric virus norovirus. Norovirus is highly contagious and is the leading cause of diarrheal disease globally, Moderna said. It’s associated with about 18% of all such illnesses worldwide and causes about 200,000 deaths every year.
Overall, Moderna is expecting to launch six major vaccine products in the next few years, all of them with large addressable markets.
The company expects the annual global endemic market for COVID boosters alone to be worth about $15 billion.
It has dosed the first participant in a late-stage trial of its next-generation, refrigerator-stable COVID-19 vaccine candidate, mRNA-1283. The vaccine “has demonstrated encouraging results in multiple clinical studies,” the company said.
A separate trial of a flu vaccine called mRNA-1010 fared less well, however.
That trial “did not accrue sufficient cases at the interim efficacy analysis to declare early success in the Phase 3 Northern Hemisphere efficacy trial and the independent DSMB recommended continuation of efficacy follow-up,” the company said.
The company expects the market for respiratory-product sales to range from $8 billion to $15 billion by 2027 and for operating profit that year to range from $4 billion to $9 billion.
The stock was down 4% Tuesday and has fallen 15% in the year to date, while the S&P 500 SPX, +0.17%
has gained 7%.
Moderna Inc. MRNA, +1.19%
outlined its clinical goals for its mRNA platform on Tuesday to mark its 4th Vaccine Day, including a new development candidate against Lyme disease that would be its first bacterial vaccine. The company, whose first product to be approved by the U.S. Food and Drug Administration was its COVID vaccine, said it has dosed a first participant in a late-stage trial of its next-generation, refrigerator-stable COVID-19 vaccine candidate, mRNA-1283. The vaccine “has demonstrated encouraging results in multiple clinical studies,” the company said in a statement. Moderna is expecting the annual global endemic market for COVID boosters to be worth about $15 billion. It expects the market for respiratory product sales in 2027 to range from $8 billion to $15 billion. The company expects to launch six major vaccine products in the next few years with “significant addressable markets.” The stock was down 6% premarket.
Abortion rights advocates gather in front of the J Marvin Jones Federal Building and Courthouse in Amarillo, Texas, on March 15, 2023.
Moises Avila | AFP | Getty Images
A federal judge in Texas on Friday suspended the Food and Drug Administration’s approval of the abortion pill mifepristone nationwide, but delayed the ruling from taking effect for a week to give the Biden administration time to appeal.
But minutes after the Texas decision was announced, a federal judge in Washington state issued a preliminary injunction that said essentially the opposite.
The seemingly conflicting federal court rulings out of Texas and Washington state means the Supreme Court may ultimately weigh in on the legality of mifepristone in the U.S., which was approved by the FDA more than two decades ago in 2000.
Used in combination with another drug called misoprostol, mifepristone is the most common method to terminate a pregnancy in the U.S., accounting for about half of all abortions.
U.S. Judge Matthew Kacsmaryk of the U.S. Northern District of Texas held a key hearing in the case weeks ago in Amarillo, but the news of the decision that could upend access to the key abortion drug only came down late on a Friday when many Americans were off for religious observances.
Kacsmaryk endorsed nearly all of the plaintiffs’ arguments about their right to sue, which called for the removal of the FDA’s approval of the drug. He argued mifepristone has serious safety issues, sidelining the FDA’s long-standing determination that the drug is safe and effective.
“The Court does not second-guess FDA’s decision-making lightly,” Kacsmaryk wrote. “But here, FDA acquiesced on its legitimate safety concerns — in violation of its statutory duty — based on plainly unsound reasoning and studies that did not support its conclusions.”
But in a dramatic turn, Judge Thomas Owen Rice of the U.S. District for the Eastern District of Washington essentially countered the Texas decision, when he barred the FDA from “altering the status quo and rights as it relates to the availability of mifepristone” in the 17 states and D.C. that sued to keep pill on the market there.
Boxes of the medication Mifepristone used to induce a medical abortion are prepared for patients at Planned Parenthood health center in Birmingham, Alabama, March 14, 2022.
Evelyn Hockstein | Reuters
U.S. Attorney General Merrick Garland said Kacsmaryk’s ruling in Texas “overturns the FDA’s expert judgment, rendered over two decades ago, that mifepristone is safe and effective.” Garland said the Justice Department will appeal the Texas ruling and defend the FDA approval.
The case will go to the U.S. 5th Circuit Court of Appeals. If the Biden administration fails to convince that court to overturn Kacsmaryk’s ruling, access to mifepristone would be in jeopardy across the U.S.
But the ruling out of Washington state may protect access at least in Arizona, Colorado, Connecticut, Delaware, Illinois, Michigan, Nevada, New Mexico, Oregon, Rhode Island, Vermont, Hawaii, Maine, Maryland, Minnesota, Pennsylvania, Washington state and D.C.
Kacsmaryk’s decision will not affect access to misoprostol, which is commonly used as a standalone abortion drug in other parts of the world. Some abortion providers have said they plan to use misoprostol as an alternative to the two-drug regimen if mifepristone is pulled from the market.
A coalition of physicians opposed to abortion, called the Alliance for Hippocratic Medicine, sued the FDA in November over its approval of mifepristone. The group argued that the FDA abused its authority by approving mifepristone through an accelerated process for new drugs that help patients with serious or life-threatening illnesses more than what is otherwise available on the market.
Kacsmaryk embraced the group’s claims Friday, arguing that pregnancy is not an illness and mifepristone does not provide a meaningful therapeutic benefit over surgical abortion.
The anti-abortion physicians were represented by attorneys from the Alliance Defending Freedom, an organization that worked with Mississippi lawmakers to draft the law at the center of Dobbs v. Jackson Women’s Health Organization. That case ultimately resulted in the Supreme Court overturning Roe v. Wade.
Kacsmaryk joined the court in 2019 after his appointment by former President Donald Trump. Kacsmaryk’s nomination was unanimously opposed by Senate Democrats as well as Republican Susan Collins of Maine, who supports abortion rights.
His nomination was also opposed by abortion and LGBTQ rights organizations such as Planned Parenthood and the Human Rights Campaign.
The FDA called the case “extraordinary and unprecedented” in its January response to the lawsuit. The agency’s lawyers said they could not find any previous example of a court second-guessing an FDA decision to approve a drug.
The agency also said mifepristone was not approved under an accelerated pathway. It took more than four years from the filing of the initial application until the pill was approved.
The FDA approved mifepristone as a safe and effective method to terminate an early pregnancy based on extensive scientific evidence, the agency’s lawyers wrote. Decades of experience among thousands of women have confirmed that the drug regimen is the safest option for many patients compared with surgical abortion or childbirth, the lawyers argued.
The FDA warned that pulling mifepristone from the U.S. market would put the health of women at risk if they cannot get access to the pill to safely end pregnancies. It would also weaken the FDA’s federal drug approval powers and hinder drug development by creating regulatory uncertainty in the marketplace, the government’s lawyers have said.
“If longstanding FDA drug approvals were so easily enjoined, even decades after being issued, pharmaceutical companies would be unable to confidently rely on FDA approval decisions to develop the pharmaceutical-drug infrastructure that Americans depend on to treat a variety of health conditions,” the Biden administration lawyers wrote.
Mifepristone has been subject to FDA restrictions since its approval in 2000 to monitor the pill’s safety and efficacy. These restrictions have been subject to criticism and litigation from medical associations such as the American College of Obstetricians and Gynecologists and more recently from attorneys general in Democratic- led states.
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The FDA has gradually eased the restrictions on mifepristone over the years as more evidence has come in. The agency dropped previous rules that required in-person visits with medical professionals, allowing the pill’s delivery by mail. The FDA recently allowed certified retail pharmacies to dispense mifepristone when the patient has a prescription from a health-care provider that’s approved under the agency’s monitoring program.
Misoprostol, the drug that’s used with mifepristone, is recommended as a stand-alone method to terminate a pregnancy by the World Health Organization. But the FDA has not approved misoprostol as an abortion medication on its own.
The American College of Obstetricians and Gynecologists recommends misoprostol as an alternative for early abortions if mifepristone is not available, though it’s not as effective as the two-drug regimen, according to the organization.
