CBER Director Peter Marks (Greg Nash/Pool via AP Images)

FDA's Pe­ter Marks over­rode re­view­ers’ call to re­ject Sarep­ta’s new Duchenne gene ther­a­py

Pe­ter Marks, the se­nior FDA of­fi­cial who over­sees the reg­u­la­tion of gene ther­a­pies, over­rode agency re­view­ers’ de­ci­sion to re­ject Sarep­ta Ther­a­peu­tics’ Duchenne mus­cu­lar dy­s­tro­phy gene ther­a­py af­ter he dis­agreed with parts of their in­ter­pre­ta­tion of the lim­it­ed ef­fi­ca­cy da­ta.

The FDA on Thurs­day grant­ed ac­cel­er­at­ed ap­proval to Sarep­ta’s gene ther­a­py, to be mar­ket­ed as Ele­v­idys, in boys aged 4 and 5 years old. The de­ci­sion fol­lowed months of reg­u­la­to­ry twists and a close vote by an ad­vi­so­ry com­mit­tee.

Endpoints News

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.