Eteplirsen Review Offers Lessons For FDA, Advocacy Groups, Industry
Agency reviewers believed the line between patient input and external intimidation had been crossed; early data from the Sarepta muscular dystrophy drug's flawed development program stoked patient community expectations and made regulatory review difficult.
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US FDA’s external advisors will meet May 18 to consider allowing use of central venous access lines in a study of two Duchenne muscular dystrophy drugs. Agency briefing documents show decision-making on placebo-controlled trial design was informed by problems in the clinical development of Exondys 51 (eteplirsen).
US agency has started transitioning to a new model for Patient-Focused Drug Development meetings, with agency officials attending four externally hosted meetings so far, with at least three more planned for the next few months. So far, the Neurology Products Division has been particularly active with the external meetings.
Two former Genzyme execs, one of whom now works at CDER, warn small companies to avoid over-emphasizing results from early, uncontrolled trials in their planning and business decision-making.