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Nov 17, 2023 · With Megan R. Wilson. WOODCOCK SET TO RETIRE — Longtime FDA regulator Janet Woodcock, who has worked at the agency since 1986, plans to retire in early 2024, FDA Commissioner Robert Califf told ...
- Buying Time
- ‘A Significant Adversary’
- Uncertain Benefit
- Echoes of Eteplirsen
- A New Controversy?
- Weighing Sarepta’s Case
- ‘Not The End Game’
Seventeen years ago, Tim Revell’s son Timothy was having trouble walking. A whirlwind of brain and blood tests followed, confirming that Duchenne was why Timothy, then 2, had fallen behind his peers. The Revells’ doctor told Tim that all he could do was go home and love his son. Timothy is one of an estimated 300,000 people worldwide, almost exclus...
Jerry Mendell saw his first Duchenne patient in 1969, during a postdoctoral fellowship at the National Institutes of Health. “We didn’t know anything about the disease” then, he said. “Just what it looked like under a microscope.” What started as a fellowship became a lifelong mission for Mendell, who in the following decades emerged as a top resea...
Jennifer Handt wrestled with whether to enroll her son Charlie in one of Sarepta’s trials. She met with other parents, visited different trial sites and “did her homework,” including vetting the doctor who would treat Charlie. Still, it wasn’t a decision she made lightly. “I had that moment of, ‘Is this the right thing to do?’” Handt said. Early st...
Seven years ago, hundreds of patients and family memberstraveled to a Washington, D.C., suburb to testify in support of an emerging treatment for Duchenne. The drug, known then as eteplirsen and also developed by Sarepta, was up for an accelerated approval. The FDA had gathered a panel of experts to review the evidence. The data primarily came from...
In March, Peter Marks, head of an FDA office that reviews new drugs, stood before more than 1,000 researchers, patient advocates and doctors to give a speechon gene therapy. Marks spoke of the agency’s urgency to speed development of gene therapies for rare and life-threatening diseases, and the tools the FDA had to help. Among those was the accele...
The decision to file early, rather than wait for the results of the company’s ongoing Phase 3 trial, was made “after a lot of contemplation” within the company, according to Ingram. One factor was time. If Sarepta waited, at least one more year would pass before the gene therapy could possibly become available. “In the life of a Duchenne kid, that’...
Christine Miller was cautiously optimistic when she enrolled her 7-year-old son George, who has Duchenne, in one of Sarepta’s trials. (At her request, BioPharma Dive changed her and her son’s name to protect their privacy.) School had become difficult for George. He couldn’t keep up with other kids during soccer, and lagged behind on the playground...
Sep 20, 2016 · Dr. Janet Woodcock won a huge power struggle within the FDA this week when the agency approved a controversial new drug from Sarepta Therapeutics.
Jun 23, 2023 · Sarepta has been in this position before. Seven years ago, Janet Woodcock, then the FDA’s top drug evaluator, overruled her own review team in recommending an accelerated approval for a Duchenne medicine now known as Exondys 51. Woodcock concluded the drug’s effect on dystrophin levels were “reasonably likely” to result in a benefit.
- Ben Fidler
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Sep 19, 2016 · In a 13-page memo last Friday, he deferred to Dr. Janet Woodcock, the controversial head of the drug review division, who pushed hard to approve the Sarepta medication but clashed with other FDA ...
Oct 25, 2016 · He noted the delays that came after this meeting, and the further controversy when CDER head Janet Woodcock “overruled” concerns about the drug’s approval. “The internal FDA review staff ...
Sep 21, 2016 · Woodcock, for her part, has thirty years at the agency, and is noted for pushing the FDA to partner with the drug industry rather than act as an adversary. She received her medical degree from Northwestern University and held teaching appointments at Pennsylvania State University and the University of California at San Francisco .
