The first gene therapy for muscular dystrophy nears approval, but will it work?
and others found that FDA staff planned to reject Sarepta’s application. But Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research intervened — scheduling a public meeting with an independent scientific advisory committee on 12 May, to weigh in on whether the drug should receive an accelerated approval. This committee narrowly recommended the approval by eight votes to six. Although the FDA doesn't have to follow the advice of the committee, it typically does.
Sarepta’s chief scientific officer, Louise Rodino-Klapac, doesn’t think that the drug actually functions better in these younger boys. Rather, she says, the older boys in the placebo control group were healthier at the beginning of the study than their younger counterparts, making the drug’s effects look weaker in this group by comparison.
Others are more concerned by what looks like an overall lack of efficacy. “I understand the appeal of ‘Something is better than nothing’, but the question is at what cost,” says Caleb Alexander, an epidemiologist at Johns Hopkins University in Baltimore who was also a committee member and voted against the drug.
To investigate the problem, Pfizer, Sarepta and Solid Biosciences, based in Charlestown, Massachusetts, decided to pool their microdystrophin data. Their study, which will soon be published in the, found that certain mutations in the dystrophin gene cause the immune system to recognize microdystrophin as a foreign invader and attack it, causing dangerous inflammation in the muscle and heart.
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