ARTICLE | Regulation
Approval of Sarepta DMD gene therapy revives questions on FDA interactions with companies, patient advocates
CBER Director Marks met with biopharma companies, industry-funded advocates while considering gene therapy application
June 28, 2024 12:06 AM UTC
The approval of Sarepta’s gene therapy for Duchenne muscular dystrophy has revived longstanding questions about non-public communications between FDA officials and companies that have pending approval applications, and about interactions between agency officials and patient advocacy organizations that are funded by companies with products under review.
The issues are complex. ...