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.

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.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER