
Senior FDA officials warned that approving $300,000 Duchenne drug will lower agency standards
In the end, Sarepta did many things wrong when it came to developing a new drug for Duchenne muscular dystrophy. But it got one very important part right. The biotech, with the support of a legion of advocates in the patient community, won over Janet Woodcock to their side early on.
The powerful CDER director pushed for an approval even in the face of a heated debate inside the FDA, as senior officials weighed in in opposition to her stand. The acting chief scientist at the agency, Luciana Borio, argued that an approval would lower the agency’s standards and encourage other developers to pursue the same kind of lobbying campaigns employed at Sarepta. And she accused Sarepta of acting irresponsibly by knowingly pushing “misleading” information about the drug. Ellis Unger, director of the office of drug evaluation, scoffed at the data Sarepta offered, calling the drug a “scientifically elegant placebo.”
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