Category: FDA
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Inside an FDA drug rejection, and layoffs at Sarepta
Inside an FDA drug rejection, and layoffs at Sarepta Just how many employees is Sarepta Therapeutics laying off? And why did the Food and Drug Administration reject Ultragenyx’s rare disease drug over manufacturing qualms? We discuss all that and more on this week’s episode of “The Readout LOUD”. Read the rest… Allison DeAngelis, Adam Feuerstein,…
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STAT+: A troubled Sun Pharma plant fails yet another FDA inspection
STAT+: A troubled Sun Pharma plant fails yet another FDA inspection A Sun Pharmaceutical plant in India that has been repeatedly cited by the U.S. Food and Drug Administration for serious quality control issues failed another inspection last month over concerns the company was not doing enough to prevent medicines from becoming contaminated. The problems…
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STAT+: FDA won’t pay 2024 bonuses to workers who are getting laid off
STAT+: FDA won’t pay 2024 bonuses to workers who are getting laid off You’re reading the web edition of D.C. Diagnosis, STAT’s twice-weekly newsletter about the politics and policy of health and medicine. Sign up here to receive it in your inbox on Tuesdays and Thursdays. I thought the news would slow down after Republicans passed their…
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FDA grants full approval to Moderna’s Covid vaccine for children but limits eligibility
FDA grants full approval to Moderna’s Covid vaccine for children but limits eligibility Moderna’s Covid-19 vaccine for children has been given full Food and Drug Administration approval, making it the first Covid vaccine for kids in the United States that will no longer be administered under an emergency use authorization. But the approval comes with…
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NIH grant cuts, FDA transparency questions, and biotech M&A
NIH grant cuts, FDA transparency questions, and biotech M&A On this week’s episode of “The Readout LOUD”: a closer look at the NIH’s grant-cutting legal playbook, a not-so-transparent transparency push by the FDA commissioner, and another big biotech acquisition. Our colleague Anil Oza joins us to unravel a previously unseen memo he obtained from HHS…
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STAT+: FDA publishes rejection letters sent to drugmakers, with a big caveat
STAT+: FDA publishes rejection letters sent to drugmakers, with a big caveat The Food and Drug Administration on Thursday published more than 200 letters that it sent to companies when it rejected their medicines, focusing attention on what’s often an opaque part of the drug review process. The agency only highlighted letters that went to…
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STAT+: Fed-up and demoralized, FDA staff are leaving.
STAT+: Fed-up and demoralized, FDA staff are leaving. You’re reading the web edition of D.C. Diagnosis, STAT’s twice-weekly newsletter about the politics and policy of health and medicine. Sign up here to receive it in your inbox on Tuesdays and Thursdays. Between innings of the July 4 Chesapeake Baysox game against the Richmond Flying Squirrels there was…
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STAT+: RFK Jr.’s vaccine advisers meet, Monarez gets grilled
STAT+: RFK Jr.’s vaccine advisers meet, Monarez gets grilled You’re reading the web edition of D.C. Diagnosis, STAT’s twice-weekly newsletter about the politics and policy of health and medicine. Sign up here to receive it in your inbox on Tuesdays and Thursdays. After making a brief visit to the hospital due to feeling lightheaded, Senate Minority Leader…
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STAT+: Top FDA drug regulator tells staff much is still in flux, as she prepares to retire
STAT+: Top FDA drug regulator tells staff much is still in flux, as she prepares to retire WASHINGTON — Outgoing Food and Drug Administration regulator Jacqueline Corrigan-Curay acknowledged to staff that much is still in flux at the agency, weeks before she retires. “We are leaner and therefore we have to find ways to be…
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STAT+: Disagreements over Duchenne therapy, management style may have led to ouster of key FDA official
STAT+: Disagreements over Duchenne therapy, management style may have led to ouster of key FDA official The ouster of the Food and Drug Administration’s chief regulator of cell and gene therapies earlier this week came immediately after a disagreement with her boss over the review of a cell therapy for Duchenne muscular dystrophy, STAT has…
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FDA’s gene therapy turmoil, and an alternative model for funding research
FDA’s gene therapy turmoil, and an alternative model for funding research Why did the Food and Drug Administration suddenly oust one of its top gene therapy regulators? Is the agency growing too political? And should private firms fund academic research? We chat about all that and more on this on this week’s episode of “The…
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Opinion: Following second patient death, Duchenne muscular dystrophy families deserve answers about Elevidys
Opinion: Following second patient death, Duchenne muscular dystrophy families deserve answers about Elevidys On Sunday at 1 a.m. Eastern time, an announcement went out from the first-generation gene therapy company for Duchenne muscular dystrophy: A second patient who had been treated with Elevidys has died. This second family said yes to hope, yes to science,…
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STAT+: Top gene therapy regulator forced out at FDA
STAT+: Top gene therapy regulator forced out at FDA Nicole Verdun, director of the office that reviews cell and gene therapies at the Food and Drug Administration, and her deputy Rachael Anatol have been placed on administrative leave and escorted out of the agency, according to a recording of a meeting obtained by STAT. Verdun…
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STAT+: FDA announces a new priority review program in bid to further administration goals
STAT+: FDA announces a new priority review program in bid to further administration goals WASHINGTON — The Food and Drug Administration is launching a program to reward companies with accelerated drug review and speedy agency feedback, if their actions align with the agency’s national health priorities. Those priorities include addressing unmet public health needs or health…
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STAT+: George W. Bush drops by BIO, and so does the FDA commissioner
STAT+: George W. Bush drops by BIO, and so does the FDA commissioner This is the online version of our BIO 2025 newsletter. Get more BIO updates directly to your inbox by signing up here, and sign up for our morning biotech news roundup newsletter here. Happy Tuesday, folks! I have an exciting update for you: Friend…
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Opinion: I study conflicts of interest. Here’s why RFK Jr.’s approach to the vaccine advisory committee could backfire
Opinion: I study conflicts of interest. Here’s why RFK Jr.’s approach to the vaccine advisory committee could backfire If Health and Human Services Secretary Robert F. Kennedy Jr. wants to “clean up the corruption and conflicts” at HHS, he is going about it the wrong way. I study conflicts of interest at federal agencies. While…
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Merck’s RSV antibody treatment for infants is approved by FDA
Merck’s RSV antibody treatment for infants is approved by FDA A third monoclonal antibody product to protect babies against respiratory syncytial virus was approved by the Food and Drug Administration on Monday, a development public health experts hope may eventually help to bring down the price of this effective but costly way of reducing the…
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STAT+: FDA reassures rare disease advocates that ‘being flexible’ is plan for gene therapy
STAT+: FDA reassures rare disease advocates that ‘being flexible’ is plan for gene therapy What will the Trump administration mean for gene therapy? Researchers, advocates, and executives got a glimpse of the answer Thursday, as the Food and Drug Administration convened a “Cell and Gene Therapy Roundtable.” It was an unusual event. Multiple panelists told…
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An AI experiment at the FDA & Novo trailing in the obesity drug race
An AI experiment at the FDA & Novo trailing in the obesity drug race On this week’s episode of “The Readout LOUD”: The Food and Drug Administration’s AI adventure, a deep look inside Novo Nordisk’s obesity stumble, and finally, another big M&A deal in biotech. The FDA has rolled out an internal AI tool called…
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FDA commissioner evades questions on Covid shot, calls CDC panel a ‘kangaroo court’
FDA commissioner evades questions on Covid shot, calls CDC panel a ‘kangaroo court’ In an interview meant to clarify the federal government’s position on Covid-19 vaccines, Food and Drug Administration Commissioner Marty Makary had few answers. Instead, he urged Americans to consult with their doctors. In a Sunday appearance on CBS News’ “Face the Nation,”…
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Opinion: Former FDA commissioner: ‘Cost-cutting’ may undo one of Trump’s best drug pricing achievements
Opinion: Former FDA commissioner: ‘Cost-cutting’ may undo one of Trump’s best drug pricing achievements President Trump often touted during his first term that his administration had “approved more affordable generic drugs than any administration in history.” He had good reason to highlight these accomplishments. Over the first two years of his presidency, the Food and…
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STAT+: What’s already been lost from CDC layoffs
STAT+: What’s already been lost from CDC layoffs You’re reading the web edition of D.C. Diagnosis, STAT’s twice-weekly newsletter about the politics and policy of health and medicine. Sign up here to receive it in your inbox on Tuesdays and Thursdays. Summer holiday weekends are a challenge. Do I seek a “relaxing” long weekend sitting in traffic…
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STAT+: What Baby KJ means for the CRISPR gene editing industry
STAT+: What Baby KJ means for the CRISPR gene editing industry For the ailing gene editing industry, hope came earlier this month in the tiny, smiling, fuzzy-headed form of KJ Muldoon. At just 6 months old, KJ received a gene editing treatment custom-built to correct his unique mutation. He’s not cured, researchers explained at the…
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FDA advisory panel split on whether agency should require updates of Covid-19 vaccine
FDA advisory panel split on whether agency should require updates of Covid-19 vaccine The Food and Drug Administration’s expert vaccine panel appeared split Thursday on whether Covid-19 vaccine manufacturers should be advised to update the strain of the virus that their products target, with some favoring recommending a newer version of the virus and others…
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Opinion: The FDA is circumventing key roles and procedures for licensure and use of Covid vaccines
Opinion: The FDA is circumventing key roles and procedures for licensure and use of Covid vaccines The Food and Drug Administration’s recent activities regarding Covid-19 vaccines stray far from the well-established standards of vaccine regulatory processes. The first sign of trouble was the failure to rule on the licensure of the Novavax protein-based Covid-19 vaccine…
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Uncertainty in CRISPR world and the start of more M&A
Uncertainty in CRISPR world and the start of more M&A What was the mood like at a big gene therapy conference last week? Why is Novo Nordisk’s CEO stepping down? And why has Adam been in such a good mood? We discuss all that and more on this week’s episode of “The Readout LOUD,” STAT’s…
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STAT+: Five burning questions on the FDA’s new Covid-19 vaccine framework
STAT+: Five burning questions on the FDA’s new Covid-19 vaccine framework WASHINGTON — Food and Drug Administration Commissioner Marty Makary promised stakeholders last week that the agency’s new Covid-19 vaccine framework would answer all of their questions. But the framework has perhaps created as much confusion as clarity. Broadly, the FDA said it will limit approval…
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New FDA framework on Covid vaccines leaves pediatricians confused and concerned
New FDA framework on Covid vaccines leaves pediatricians confused and concerned A new framework for Covid-19 vaccines announced by Food and Drug Administration leaders Tuesday suggests the agency will no longer approve new Covid vaccines for healthy individuals under 65, including babies, without data from new randomized clinical trials showing their benefit. The plan —…
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STAT+: Novavax wins narrowed approval for its Covid-19 shot after delay
STAT+: Novavax wins narrowed approval for its Covid-19 shot after delay The Food and Drug Administration has finally approved Novavax’s Covid-19 vaccine, but in doing so has placed restrictions on it that its two competitors in the U.S. market do not face. The long-awaited license limits use of the vaccine to people 65 and older…
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STAT+: At gene therapy meeting, no one wants to talk about the Vinay Prasad (not) in the room
STAT+: At gene therapy meeting, no one wants to talk about the Vinay Prasad (not) in the room NEW ORLEANS — Last year, hundreds of people poured in to see Peter Marks, the Food and Drug Administration’s head of biologics, speak at the American Society of Gene and Cell Therapy conference. The regulator was seen by…
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STAT+: Day 3 at ASGCT: Vinay Prasad speculation, screening newborns, promising data
STAT+: Day 3 at ASGCT: Vinay Prasad speculation, screening newborns, promising data NEW ORLEANS — Greetings from the home stretch of American Society of Gene and Cell Therapy conference, where everyone is still talking about Baby KJ and no one wants to talk about Vinay Prasad. The Vinay Prasad in the room Last year, hundreds…
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STAT+: MAHA has a new think tank. The Trump administration is listening
STAT+: MAHA has a new think tank. The Trump administration is listening The Make America Healthy Again movement is coalescing around a new effort to turn its goals into federal policy. The Trump administration is listening. The MAHA Institute, a policy center launched Thursday, is pushing to change the American health and food systems: from…
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Former FDA head Robert Califf says RFK Jr.’s vaccine rhetoric is ‘doing harm’ to Americans
Former FDA head Robert Califf says RFK Jr.’s vaccine rhetoric is ‘doing harm’ to Americans SAN FRANCISCO — Former Food and Drug Administration Commissioner Robert Califf said health secretary Robert F. Kennedy Jr.’s hesitance to endorse vaccines is harming the American public. “Fundamental vaccination saves millions of lives every year already, and he’s busy eroding public…
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STAT+: Trump order on drug pricing appears to issue subtle threat to pharma
STAT+: Trump order on drug pricing appears to issue subtle threat to pharma File this under “Lower your price — or else!” Tucked inside the executive order on drug pricing from President Trump is a sentence that appears to threaten pharmaceutical companies that their medicines may be withdrawn if they do not lower prices. Specifically,…
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First at-home test kit for cervical cancer approved by the FDA, company says
First at-home test kit for cervical cancer approved by the FDA, company says WASHINGTON — U.S. regulators have approved the first cervical cancer testing kit that allows women to collect their own sample at home before shipping it to a laboratory, according to a medical device company. Teal Health said Friday the Food and Drug Administration approved…
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STAT+: 5 burning questions about FDA’s ‘aggressive’ deployment of AI for scientific review
STAT+: 5 burning questions about FDA’s ‘aggressive’ deployment of AI for scientific review The Food and Drug Administration said it will rapidly roll out a generative artificial intelligence model to assist scientific reviews across the agency, setting up a high-stakes test of the technology’s use in vetting products used in the care of millions of…
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A firebrand takes oversight of vaccines, gene therapies
A firebrand takes oversight of vaccines, gene therapies What does the appointment of Vinay Prasad as the head of biologic drug evaluation mean for biotechs? Is his fiery public persona suited for government service? We discuss these and other burning questions on a new episode of “The Readout LOUD,” STAT’s biotech podcast. Read the rest……
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STAT+: Vinay Prasad tapped to run FDA center that regulates vaccines, gene therapies
STAT+: Vinay Prasad tapped to run FDA center that regulates vaccines, gene therapies WASHINGTON — Vinay Prasad, an academic and fierce critic of the medical mainstream, will be the next director of the Food and Drug Administration center that oversees the regulation of vaccines, gene therapies, and the blood supply. Prasad, previously an epidemiology professor at…
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STAT+: Trump signs order in bid to boost pharma manufacturing in the U.S.
STAT+: Trump signs order in bid to boost pharma manufacturing in the U.S. Amid ongoing anticipation over tariffs on pharmaceuticals, President Trump on Monday signed an executive order designed to lower regulatory hurdles and make it faster for drug companies to manufacture their products in the U.S. The move also includes plans to place more…
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STAT+: Capricor Therapeutics forges ahead with FDA on Duchenne drug, despite political tumult
STAT+: Capricor Therapeutics forges ahead with FDA on Duchenne drug, despite political tumult The Food and Drug Administration is nearly halfway through a review of Capricor Therapeutics’ cell therapy for Duchenne muscular dystrophy. Even with the tumult inside the agency, interactions between the company and the agency staff have been unaffected, according to the company’s…
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Day by day, how Trump is roiling science and health
Day by day, how Trump is roiling science and health It’s hard to remember all the earthshaking decisions and events that have transpired with unprecedented speed in the first few months of the Trump administration. We’re tracking, day by day, what’s happened in the worlds of science and health. Key Legal action Covid DEI and…
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Food companies agree to phase out synthetic dyes, handing MAHA a victory
Food companies agree to phase out synthetic dyes, handing MAHA a victory Food manufacturers will phase out eight synthetic dyes from all U.S. products by the end of 2026, the federal government announced today in a move that reflects the growing reach of the Make America Healthy Again movement. Decrying the “toxic soup of synthetic…
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STAT+: Research grants as political ammo; Death to dyes?
STAT+: Research grants as political ammo; Death to dyes? You’re reading the web edition of D.C. Diagnosis, STAT’s twice-weekly newsletter about the politics and policy of health and medicine. Sign up here to receive it in your inbox on Tuesdays and Thursdays. STAT won its first National Magazine Award for General Excellence. Not bad, huh? Help us…
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STAT+: Trump’s plans to lower drug prices have echoes of past efforts
STAT+: Trump’s plans to lower drug prices have echoes of past efforts WASHINGTON — The Trump administration has an expansive view for how it can lower drug prices for Americans. It has been here before. The question is whether this time will be different. During his first term, President Trump tried to tie U.S. drug…
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FDA OKs trial of pig livers as dialysis-like treatment for liver failure
FDA OKs trial of pig livers as dialysis-like treatment for liver failure WASHINGTON — U.S. researchers will soon test whether livers from a gene-edited pig could treat people with sudden liver failure — by temporarily filtering their blood so their own organ can rest and maybe heal. The first-of-its-kind clinical trial has been cleared by the Food…
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BIO’s John Crowley on RFK Jr. and tariffs, plus calls for more federal biotech funding
BIO’s John Crowley on RFK Jr. and tariffs, plus calls for more federal biotech funding Is there more funding coming for the biotech industry? Will the pharmaceutical industry be hit with tariffs? And is it possible to reason with RFK Jr.? We talk about all that and more on this week’s episode of “The Readout…
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STAT+: Why the closure of an FDA office may impact generics manufacturers — and everyday Americans
STAT+: Why the closure of an FDA office may impact generics manufacturers — and everyday Americans Amid the hard-to-follow cutbacks at the U.S. Food and Drug Administration, a little-known but important office was eliminated — and the implications will be felt not only by drugmakers, but consumers. The Division of Policy Development in the Office…
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STAT+: FDA tells drugmakers to redo studies run by a contract research firm due to data integrity issues
STAT+: FDA tells drugmakers to redo studies run by a contract research firm due to data integrity issues In a rare move, the Food and Drug Administration told an unspecified number of drug companies that studies used to support therapeutic equivalence of some of their medicines have been rejected due to false data generated by…
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Cuts, ousters and drama at the FDA
Cuts, ousters and drama at the FDA Turmoil, job cuts and forced ousters of high-ranking officials at federal health agencies. Marty Makary’s rough first week as FDA Commissioner. Biotech stocks tumble even further. President Trump’s “Liberation Day” tariffs roil global markets. We talk about biotech’s volatile week, plus interview AstraZeneca R&D executive Sharon Barr, on…
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STAT+: A running list of senior FDA officials who have left the agency
STAT+: A running list of senior FDA officials who have left the agency WASHINGTON — The Food and Drug Administration always looks a little different after an administration change. Political appointees are swapped out, maybe some offices are restructured. But the FDA under the new Trump administration is already starting to look dramatically different. It’s hard…
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STAT+: Makary’s first speech to FDA staff focuses on challenging norms, fighting chronic disease
STAT+: Makary’s first speech to FDA staff focuses on challenging norms, fighting chronic disease WASHINGTON — Marty Makary’s first address to staff as FDA commissioner on Wednesday afternoon sounded a bit like an introduction to one of his books. It included sweeping declarations about the need to challenge scientific norms, and to identify the root…
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STAT+: FDA Commissioner Marty Makary gets off to a bruising start as agency is wracked by layoffs
STAT+: FDA Commissioner Marty Makary gets off to a bruising start as agency is wracked by layoffs WASHINGTON — Marty Makary’s first official day as commissioner of the Food and Drug Administration began with employees in tears, learning from security guards that they were losing their jobs. The news release announcing the start of his…
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STAT+: Cassidy gave himself a difficult task. RFK Jr. isn’t making it any easier on him
STAT+: Cassidy gave himself a difficult task. RFK Jr. isn’t making it any easier on him WASHINGTON — Sen. Bill Cassidy (R-La.) said he would be Robert F. Kennedy Jr.’s keeper, but the physician-turned-politician is struggling to stop the nation’s health secretary from undermining public trust in vaccines. Cassidy has taken the lead for his…
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STAT+: Texas judge strikes down FDA’s plan to regulate lab-developed tests
STAT+: Texas judge strikes down FDA’s plan to regulate lab-developed tests WASHINGTON — A federal judge in Texas squashed the Food and Drug Administration’s plan to regulate lab-developed tests on Monday, ruling in favor of lab trade groups that said the agency was overstepping its bounds. “The Court VACATES and SETS ASIDE, in its entirety,…
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Opinion: STAT+: Peter Marks’ ouster is an ominous sign for biotech — and for public health
Opinion: STAT+: Peter Marks’ ouster is an ominous sign for biotech — and for public health Welcome to Lab Dish, a new First Opinion column on regenerative medicine from Paul Knoepfler. There are certain leaders who quietly make complex organizations operate well including in the toughest of times — leaders like Peter Marks, director of…
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STAT+: FDA’s ‘key man’ Peter Marks is out. Biotech investors will now deal with the consequences
STAT+: FDA’s ‘key man’ Peter Marks is out. Biotech investors will now deal with the consequences Robert F. Kennedy Jr. forcing Peter Marks out of the Food and Drug Administration has blown a hole in the agency’s leadership ranks and threatens to unmoor the oversight of drugs and vaccines from bedrock scientific principles. For biotech…
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Peter Marks, FDA’s top vaccine regulator, forced out
Peter Marks, FDA’s top vaccine regulator, forced out Peter Marks, the top Food and Drug Administration official who oversaw vaccines, gene therapies, and the blood supply, resigned Friday after being told by Trump administration officials he would be fired if he did not step down, according to people familiar with the situation. In his resignation…
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STAT+: Ouster of FDA’s Peter Marks alarms a biopharma industry that saw him as an ally
STAT+: Ouster of FDA’s Peter Marks alarms a biopharma industry that saw him as an ally When Robert F. Kennedy Jr., a longtime and visceral critic of pharmaceutical and biotech companies, was nominated by President Trump to be the nation’s health secretary, the head of biotech’s largest trade group issued a statement saying that he…
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STAT+: FDA to staff: No need to work from pantries for now
STAT+: FDA to staff: No need to work from pantries for now Some Food and Drug Administration employees who were previously working in pantries and conference rooms can work from home for now, according to an email reviewed by STAT. FDA managers sent an email Friday granting some workers temporary work-from-home exceptions, either because their…
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STAT+: HHS layoffs rile Democrats, but Republicans say they trust RFK Jr.’s cuts will not hamper core operations
STAT+: HHS layoffs rile Democrats, but Republicans say they trust RFK Jr.’s cuts will not hamper core operations WASHINGTON — News of massive layoffs at the Health and Human Services Department was met with anger and predictions of lawsuits by Democratic lawmakers, and with a shrug by Republicans. HHS Secretary Robert F. Kennedy Jr. announced…
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STAT+: Two of FDA’s top cancer regulators to depart, heightening worries about drug reviews
STAT+: Two of FDA’s top cancer regulators to depart, heightening worries about drug reviews Both deputy directors at the key Food and Drug Administration center that oversees the regulation of cancer drugs plan on departing the agency, sources told STAT Thursday, highlighting the drain on talent at the FDA created by layoffs, uncertainty, and shifts…
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Opinion: Former HHS Secretary Donna Shalala on the agency’s ‘silly new bureaucracy’
Opinion: Former HHS Secretary Donna Shalala on the agency’s ‘silly new bureaucracy’ When I was secretary of the Department of Health and Human Services under President Bill Clinton, an agency head came to me with a dramatic reorganization plan. I advised him that unless it was data- and outcome-driven, he was wasting his time. Just…
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STAT+: RFK Jr. brings FDA under tighter control with HHS workforce cuts
STAT+: RFK Jr. brings FDA under tighter control with HHS workforce cuts WASHINGTON — Around 3,500 employees are on the chopping block at the Food and Drug Administration, but they don’t yet know who they are. The Health and Human Services Department on Thursday announced a sweeping plan to cut 10,000 jobs and consolidate operations…