The treatment from Sarepta Therapeutics was approved Thursday for children ages 4 and 5 with Duchenne muscular dystrophy, a rare muscle-wasting disease that causes early death.
So, Sarepta has multiple approved therapies that haven’t reached positive clinical endpoints? How many times can the agency/drugmaker justify accelerated approval based on an unmet clinical need?
Imo, this just adds to the growing criticism of the agency’s growing use of accelerated approvals without holding pharma companies to their post-approval obligations.
So, Sarepta has multiple approved therapies that haven’t reached positive clinical endpoints? How many times can the agency/drugmaker justify accelerated approval based on an unmet clinical need?
Imo, this just adds to the growing criticism of the agency’s growing use of accelerated approvals without holding pharma companies to their post-approval obligations.