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Sarepta Complained of “Dire Financial” Ramifications, FDA Caved In on Approval of Duchenne Drug, According to Emails

Sarepta Complained of “Dire Financial” Ramifications, FDA Caved In on Approval of Duchenne Drug, According to Emails | Pharma Industry Regulation |

Two senior FDA officials mounted a vehement assault on Janet Woodcock’s decision to push through an approval of Sarepta’s Duchenne muscular dystrophy drug Exondys 51. New documents posted by the FDA (here;, including a round of memos on the issue in September, warned FDA Commissioner Robert Califf that he was allowing an approval even though Woodcock had not considered all the analysis they had done to underscore the company’s weak case, adding that there was no scientific basis to conclude that the drug was reasonably likely to benefit patients.


The agency has already posted some of the memos offered up in the lead-up to the approval decision, along with Califf’s decision that he would defer to Woodcock, who overruled the agency’s reviewers as well as Luciano Borio and Ellis Unger, the acting chief scientist and director of the Office of Drug Evaluation at the FDA. But these memos from September hit hard on their conclusion that Woodcock was deviating widely from the agency’s standards for an approval in allowing Exondys 51 on the market after the biotech had provided only a “mere scintilla” of evidence of efficacy (read “Janet Woodcock Won a Huge Power Struggle Within #FDA Over eteplirsen Approval”;


In a memo to Califf on September 14, Unger wrote that he had conducted an analysis of the evidence on dystrophin production, which Woodcock did not take into consideration before reaching her decision.


“I think it will be important for the regulatory record to reflect that there was no scientific basis underlying the conclusion of “reasonably likely” in this case. This was simply a judgment call by Dr. Woodcock. (Dr. Woodcock might have also taken the position that, in this desperate patient population, any dystrophin production would suffice as a basis for accelerated approval, but she didn’t state this.)”


In Unger’s opinion, the FDA also needed to more carefully consider the impact the decision on eteplirsen would have on other rare disease drug applications.


“We all agree that each situation must be evaluated on its own merits; however, I fail to see how DMD differs intrinsically from other rare neurological diseases, e.g., Alexander disease, Canavan disease, Early infantile GM1 gangliosidosis, Krabbe disease, Metachromatic leukodystrophy, Niemann–Pick disease, Pelizaeus–Merzbacher disease, Pompe disease, Sandhoff disease, and X- linked adrenoleukodystrophy. Based on what you have written in your draft memo, it is not clear to me why a standard of any increase in the surrogate endpoint wouldn’t apply for these diseases.


“Perhaps granting accelerated approval to drugs that show a mere scintilla of an effect on a surrogate endpoint represents a stroke of brilliance – one that will stimulate investment in the development of drugs for these disorders. But in my opinion, this approach should receive broader public (and FDA) input before being implemented.”

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