FDA sets June decision deadline for potential full approval, label expansion of Sarepta’s Duchenne gene therapy

Sarep­ta Ther­a­peu­tics could soon notch an­oth­er reg­u­la­to­ry win.

De­spite the com­pa­ny and its Duchenne gene ther­a­py get­ting dogged by de­bate, and then a failed con­fir­ma­to­ry study, the treat­ment ap­pears head­ed for a la­bel ex­pan­sion that would broad­en its use be­yond boys ages 4 and 5.

On Fri­day, the com­pa­ny an­nounced that the FDA set a June 21 dead­line to make de­ci­sions on ex­pand­ing Ele­v­idys’ la­bel and con­vert­ing its ac­cel­er­at­ed ap­proval in­to a full one. And Sarep­ta won’t have to face an FDA ad­vi­so­ry com­mit­tee for the treat­ment, a pos­i­tive sig­nal from the agency that it won’t ask a pub­lic pan­el of out­side ex­perts for in­put.

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.