Patient Advocacy With FDA Review Staff Will Be Tougher Post-Sarepta
Efforts by CDER management to encourage more interaction between patients and FDA reviewers may be part of the collateral damage from the difficult FDA review of Sarepta’s Exondys 51. The hopes of parents of boys with DMD pushed the regulatory flexibility by CDER management on this application – but may end up limiting the willingness of FDA reviewers to engage with patients in the future.
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Cellular, Tissue and Gene Therapies Advisory Committee will weigh whether the increase in dystrophin production seen with SRP-9001 is reasonably likely to predict clinical benefit in Duchenne muscular dystrophy, Sarepta says; agency had previously told the company a panel meeting would not be needed, but it reversed course late in the review.
Patient advocates asked what they could do after US FDA’s complete response letter for the proposed Duchenne drug, but Sarepta's CEO said the company would get the product back on track to approval itself, a marked contrast to engaged role the community played the last time the firm ran into a roadblock at the agency.
After conducting 24 disease-focused meetings in five years, FDA moving to next phase of patient interaction under new user fee cycle.