[ad_1]
Two weeks ago, bluebird bio secured Food and Drug Administration approval for its gene therapy for sickle cell disease, a significant milestone for the roughly 100,000 people in the U.S. who suffer from the condition.
Source link
Tag: Product/Service Approvals
-
Pfizer gets FDA green light for new shot that can streamline teenagers’ vaccinations
[ad_1]
Pfizer Inc.
PFE,
-1.73%
said Friday that the U.S. Food and Drug Administration has approved the first five-in-one vaccine designed to protect teenagers and young adults against meningococcal disease.The new Pfizer shot, Penbraya, protects against the five most common subgroups of meningococcal disease, a rare but serious and potentially fatal illness that most often affects babies and teenagers.
Penbraya “has the potential to protect more adolescents and young adults from this severe and unpredictable disease by providing the broadest meningococcal coverage in the fewest shots,” Annaliesa Anderson, Pfizer senior vice president and head of vaccine research and development, said in a statement.
The U.S. Centers for Disease Control and Prevention currently recommends that all 11- to 12-year-olds get a meningococcal vaccine protecting against four of the subgroups — A, C, W and Y — and get a booster dose of the same vaccine type at age 16. Teenagers and young adults age 16 to 23 may also get a meningococcal B vaccine, the CDC says, particularly if they’re at increased risk due to other health conditions.
The complex vaccination schedule has weighed on uptake of the meningococcal shots, and the COVID-19 pandemic may have compounded the problem, as many families missed routine appointments when vaccinations were due, researchers say. Among teenagers who were born in 2008 — who were due for their routine adolescent vaccinations as the pandemic was raging in 2020 — uptake of meningococcal and other recommended vaccines declined, according to CDC research. Only about 60% of the 17-year-olds surveyed by the CDC last year had received both recommended doses of the ACWY vaccine, and fewer than 30% had received at least one dose of the meningococcal B vaccine.
The new Pfizer shot combines components of a meningococcal group B vaccine and an ACWY vaccine.
A CDC immunization advisory committee is set to meet Oct. 25 to discuss recommendations for the use of Penbraya in teenagers and young adults, Pfizer said.
The green light for Penbraya gives Pfizer the edge in its race with GSK
GSK,
+0.54% ,
which is also working on a five-in-one meningococcal shot. GSK earlier this year released positive late-stage clinical-trial results for that vaccine.The FDA approval of Pfizer’s shot caps a rocky week for the pharmaceutical giant, which late last Friday cut $9 billion from its full-year revenue guidance due to reduced COVID sales expectations and announced a cost-cutting program designed to deliver savings of at least $3.5 billion. Pfizer executives said on a call with analysts Monday that development of combination respiratory vaccines, such as those that provide COVID and flu protection in one shot, remains a focus for the company, in part because they can help boost vaccine uptake.
Pfizer shares were down 1.7% Friday and have dropped 40% in the year to date, while the S&P 500
SPX
has gained 10%.[ad_2]
-
Ardelyx wins long battle for FDA approval of kidney-disease treatment
[ad_1]
Ardelyx Inc.
ARDX,
-0.58%
on Tuesday won its long fight for U.S. regulatory approval of a treatment designed to help patients with chronic kidney disease.The biopharma company said late Tuesday that the U.S. Food and Drug Administration approved tenapanor, marketed under the brand name Xphozah, for control of serum phosphorus in patients with chronic kidney disease on dialysis. A high level of phosphorus in the blood is often a sign of kidney damage and can lead to weak bones, joint pain, cardiovascular problems and other issues.
The approval concludes an extraordinary comeback for Xphozah. Ardelyx’s application for approval of the treatment was previously rejected by the FDA in July 2021, when the agency said the drug’s effect was “small and of unclear clinical significance.” To preserve cash, Ardelyx a few months later said it would cut its workforce by 65%, but it also pursued multiple appeals of the agency’s decision. An FDA advisory committee voted last November that the benefits of Xphozah outweigh its risks. The agency late last year ultimately granted Ardelyx’s appeal, and the company resubmitted its Xphozah application to the FDA in April.
More than 550,000 people in the U.S. have chronic kidney disease and are on maintenance dialysis. The vast majority of them have high phosphorus levels, also called hyperphosphatemia, according to Ardelyx.
High phosphorus levels “must be taken care of in these patients, and the current therapies are mostly insufficient in doing so,” Ardelyx CEO Mike Raab told MarketWatch.
Chronic kidney disease patients with high phosphorus levels have traditionally been treated with phosphate binders that can soak up phosphorus from food during digestion, but that approach can require patients to swallow a large number of pills. The Xphozah treatment requires two pills a day, “each the size of a Tic Tac,” Raab said.
The FDA approved Xphozah as add-on therapy in patients who can’t tolerate or have an inadequate response to phosphate binders, Ardelyx said in a release.
Xphozah, which will be Ardelyx’s second U.S. product launch, should be available sometime in November, Raab said.
Ardelyx will present updated data on Xphozah for hyperphosphatemia at an American Society of Nephrology meeting in early November, the company said in a release Monday.
Ardelyx shares jumped in late September after Japanese regulators approved tenapanor for hyperphosphatemia in adults with chronic kidney disease on dialysis.
Ardelyx shares fell 0.6% on Tuesday and have gained 21% in the year to date, while the S&P 500
SPX
has gained 13.9%.[ad_2]
-
FDA approves first-ever pill for postpartum depression in new mothers
[ad_1]
The Food and Drug Administration late Friday approved the first-ever pill that can be taken at home for postpartum depression.
The medication, called zuranolone, and jointly developed by pharmaceutical companies Biogen Inc.
BIIB,
+0.44%
and Sage Therapeutics
SAGE,
+0.25% ,
is taken daily for two weeks, the FDA said in its release.In a pair of clinical trials involving women who experienced severe depression after having a baby, the drug improved symptoms including anxiety, trouble sleeping, loss of pleasure, low energy, guilt or social withdrawal as soon as three days after the first pill.
“Postpartum depression is a serious and potentially life-threatening condition in which women experience sadness, guilt, worthlessness — even, in severe cases, thoughts of harming themselves or their child,” said Tiffany Farchione, M.D., director of the Division of Psychiatry in the FDA’s Center for Drug Evaluation and Research.
”And, because postpartum depression can disrupt the maternal-infant bond, it can also have consequences for the child’s physical and emotional development,” she said.
Women who are breastfeeding or had mild or moderate depression weren’t included in the trials.
Until now, the only available option for this condition has been an intravenous injection that the FDA approved in 2019. It requires patients to stay in a hospital for two-and-a-half days.
Postpartum depression affects one in eight new mothers in the U.S., according to the Centers for Disease Control and Prevention. Researchers suggest the actual rate may be higher and that half of such cases go undiagnosed.
Research finds that postpartum depression is more intense and lasts longer than the typical worries, sadness or tiredness that many women experience after giving birth. The condition can make it harder for mothers to bond with their babies and may increase the likelihood of developmental delays in infants.
Drug overdoses and suicides are leading causes of maternal death in the U.S., contributing to nearly one in four pregnancy-related deaths, according to the CDC.
Zuranolone stimulates a brain receptor called GABA that slows down the brain and helps control anxiety and stress. The drug, through trials, is thought to calm women suffering from postpartum depression enough to allow them to rest, which also improves symptoms.
Shares of Biogen are up 23% over the past year, and Sage has lost 14%, while the S&P 500
SPX
is up 8% over the same time.[ad_2]
-
FDA approves Alzheimer’s treatment Leqembi, clearing the way for Medicare coverage
[ad_1]
The U.S. Food and Drug Administration on Thursday granted full approval to the Biogen BIIB and Eisai Co. Ltd. ESALF Alzheimer’s treatment Leqembi, a step that secures Medicare reimbursement for the first drug shown to slow the progress of the disease, rather than just treating its symptoms.
Leqembi, also known as lecanemab, is a monoclonal antibody designed to reduce the buildup of amyloid beta plaque in the brain, a marker of Alzheimer’s disease.
The…
[ad_2]
-
AstraZeneca Says US Approved Lynparza as a Prostate Cancer Treatment
[ad_1]
By Anthony O. Goriainoff
AstraZeneca said Thursday that its and MSD’s Lynparza cancer treatment had been approved in the U.S. for the treatment of metastatic castration-resistant prostate cancer, or mCRPC.
The Anglo-Swedish pharma giant said Lynparza, in combination with abiraterone and prednisone, reduced the risk of disease progression or…
[ad_2]
-
Biogen wins accelerated FDA approval for treatment for rare form of ALS
[ad_1]
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.
In…
[ad_2]
-
U.S. Supreme Court preserves near-term access to abortion pill mifepristone
[ad_1]
WASHINGTON (AP) — The Supreme Court on Friday preserved women’s access to a drug used in the most common method of abortion, rejecting lower-court restrictions while a lawsuit continues.
The justices granted emergency requests from the Biden administration and New York–based Danco Laboratories, maker of the drug, called mifepristone. They are appealing a lower-court ruling that would roll back Food and Drug Administration approval of mifepristone.
The drug has been approved for use in the U.S. since 2000 and more than 5 million people have used it. Mifepristone is used in combination with a second drug, misoprostol, in more than half of all abortions in the U.S.
The court faced a self-imposed Friday night deadline to decide whether women’s access to a widely used abortion pill would remain unchanged or be restricted while a legal challenge to its Food and Drug Administration approval goes on.
The justices have been weighing arguments that allowing restrictions contained in lower-court rulings to take effect would severely disrupt the availability of the drug, mifepristone, which is used in the most common abortion method in the United States.
It has repeatedly been found to be safe and effective, and has been used by more than 5 million women in the U.S. since the FDA approved it in 2000.
The Supreme Court had initially said it would decide by Wednesday whether the restrictions could take effect while the case continues. A one-sentence order signed by Justice Samuel Alito on Wednesday gave the justices two additional days, without explanation.
Abortion opponents filed suit in Texas in November, asserting that FDA’s original approval of mifepristone 23 years ago and subsequent changes were flawed.
Matthew Kacsmaryk, shown listening to a question during his confirmation hearing before the Senate Judiciary Committee in 2017, is the lone federal judge in his north Texas district — a fact that led to speculation among critics that the abortion-pill case had landed in his courtroom via judge shopping.
Senate Judiciary Committee/AP
Further context (March 2023): Trump appointee in single-judge federal district in Texas could bar nationwide access to the abortifacient mifepristone
Also (April 2023): Access to abortion pill in limbo after competing rulings in Texas and Washington
They won a ruling on April 7 by U.S. District Judge Matthew Kacsmaryk, an appointee of former President Donald Trump, revoking FDA approval of mifepristone. The judge, the lone judge in his Amarillo, Texas, federal district, gave the Biden administration and Danco a week to appeal and seek to keep his ruling on hold.
Responding to a quick appeal, two more Trump appointees on the 5th U.S. Circuit Court of Appeals said the FDA’s original approval would stand for now. But Judges Andrew Oldham and Kurt Englehardt said most of the rest of Kacsmaryk’s ruling could take effect while the case winds through federal courts.
MarketWatch contributed.
[ad_2]
-
Federal judge in Texas suspends FDA approval of abortion pill mifepristone
[ad_1]
AUSTIN, Texas (AP) — A federal judge in Texas on Friday ordered a hold on the U.S. approval of the abortion medication mifepristone, throwing into question access to the nation’s most common method of abortion in a ruling that waved aside decades of scientific approval.
The abortion drug has been widely used in the U.S. since 2000 and there is essentially no precedent for a lone judge overruling the medical decisions of the Food and Drug Administration. Mifepristone is one of two drugs used for medication abortion in the United…
[ad_2]
-
FDA pulls preterm-birth drug Makena and its generics from market after 12 years
[ad_1]
The U.S. Food and Drug Administration said Thursday it had reached a final decision to fully withdraw approval of preterm-birth drug Makena and its generics, a full 12 years after the treatment hit the market.
The drug was approved in 2011 using the agency’s accelerated-approval pathway as a treatment to reduce the risk of spontaneous preterm birth in pregnant women who had a history of the condition.
The…
[ad_2]
-
China Approves Merck’s Molnupiravir for Emergency Use, Regulator Says
[ad_1]
China’s top drug regulator said Friday that it approved Merck & Co.’s Molnupiravir for emergency use on Thursday, as the country grapples with waves of infections after Beijing abruptly reversed its stringent Covid-19 restrictions earlier this month.
The National Medical Products Administration said it is requiring the approval holder to continue relevant research, complete conditional requirements and submit follow-up research results in a timely manner, according to a statement posted on its website Friday.
Write to Singapore Editors at singaporeeditors@dowjones.com
Corrections & Amplifications
This item was corrected at 0856 GMT to reflect China’s top drug regulator said it approved Merck & Co.’s Molnupiravir for emergency use on Thursday. The original version incorrectly said the approval came on Wednesday in the first paragraph.
[ad_2]
