Janet Woodcock, the Food and Drug Administration’s director of drug evaluation, told panelists reviewing a potential drug for Duchenne muscular dystrophy this morning that there is a clear danger to patients in not approving a drug that works.
In comments before the advisory committee to review a drug developed by Cambridge-based Sarepta Therapeutics (Nasdaq: SRPT), Woodcock’s comments will be seen as largely supportive of accelerated approval of the drug. She seemed to outline a rationale under which the drug could be given accelerated approval, despite much speculation on Wall Street to the contrary in the past three months. She told the panel and the audience of hundreds who packed into the room that just as there is a danger in approving a drug that does not work, “Often there is never consideration of another error — that of not approving a drug that works,” she said.
Woodcock said there is “an inherent presumption” in the idea of accelerated approval of a drug that “more uncertainty is going to be tolerated, at least at the beginning.”
She said her comments were meant to be a “framework” for the panel to consider the information that is being presented today by the company, by world-renowned experts in Duchenne muscular dystrophy, by patient advocates, and by the FDA scientists. She allowed that “it’s possible to reach different conclusions about these comparisons” to patients who have been treated with the drug to what is known about patients who have not, seemingly allowing for the wide gap in interpretations of the data between Sarepta and the FDA.
She said “there is agreement that the drug does achieve primary pharmacodynamic effect,” which is increasing a protein needed for muscle production known as dystophin. The amount that it increased that protein was not as much as hoped, at least in the small study, but she acknowledged that there is evidence that even a small amount may help patients.
Woodcock’s comments came at the end of a two-hour presentation by the company and top scientists that was mostly supportive of approval of the drug, but before the FDA’s comments that are expected to be highly critical of it. While they do not ensure the panel will approve the drug — or that she will approve the drug regardless of the vote — her comments were perceived by many observers on Twitter as more positive toward the company than expected.
Earlier comments by the FDA’s director of the Division of Neurology Products, William Dunn (a subordinate of Woodcock’s) seemed to emphasize the need for “substantial” data needed for even accelerated approval. He said accelerated approval can’t make up for bad or inconclusive data (the agency’s main argument so far has been Sarepta’s data is inconclusive), and even hinted that the company may have “misled” the agency into allowing a submission for approval of the drug.
In the end, it is Woodcock who will make the final approval decision on the drug, meaning that while she was speaking to the 10-member panel that will make the recommendation as to whether eteplirsen ought to be approved, she doesn’t necessarily have to abide by that recommendation.
Patient testimony is expected to take up most of the afternoon, to be followed by the panel votes on seven key questions.
Editor's note: This story was updated at noon Monday to add in Dunn's comments.
