Internal documents shed new light on Sarepta FDA decision, which top agency official says was ‘exceptional’

While he has re­peat­ed­ly ad­vo­cat­ed for ac­cel­er­at­ed gene ther­a­py ap­provals, the FDA’s top cell and gene ther­a­py of­fi­cial Pe­ter Marks said that the agency’s land­mark de­ci­sion to ap­prove Sarep­ta’s Duchenne gene ther­a­py shouldn’t be viewed as an ex­am­ple of a usu­al ac­cel­er­at­ed ap­proval.

“I think that was ex­cep­tion­al. As you may be aware of from all the mem­os that are now pub­lic, there was a lot that went around that ap­proval. I think we’ll use age re­stric­tions on­ly when it ab­solute­ly makes sense from a safe­ty per­spec­tive and an ef­fi­ca­cy per­spec­tive,” Marks, the di­rec­tor of the FDA’s Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search (CBER), said at Bio­phar­ma Con­gress on Mon­day.

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.