Eteplirsen Review Offers Lessons For FDA, Advocacy Groups, Industry
Executive Summary
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.
You may also be interested in...
Sarepta Trial Protocol Changes May Ignite Debate On Placebo Design
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).
FDA Patient Feedback Meetings: External Model Ramping Up In 2017
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.
Small Biopharmas Should Be Wary Of Early Results – US FDA Official
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.
Need a specific report? 1000+ reports available
Buy Reports
Register for our free email digests: