Skip to Main Content

Sarepta Therapeutics always wins. That was a given, even before the Food and Drug Administration expanded approval of the company’s Duchenne muscular dystrophy drug on Thursday. Will boys with Duchenne also benefit? I hope they do, but it’s uncertain. And is this a good look for the FDA? No, it is not.

There is much to unpack from the unilateral decision by Peter Marks, the FDA’s top regulator of gene therapies, to expand the use of Sarepta’s Elevidys to nearly all Duchenne patients. Some thoughts:


Sarepta is unshackled. The broad approval — its best-case scenario — gives the company freedom to sell its gene therapy almost without restrictions. Manufacturing capacity — and how fast it can be upgraded — might be one of the only brakes right now on Sarepta’s effort to hit peak U.S. sales of $2-3 billion.

STAT+ Exclusive Story


This article is exclusive to STAT+ subscribers

Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.

Already have an account? Log in

Already have an account? Log in



Totals $468 per year

$39/month Get Started

Totals $468 per year



for 3 months, then $39/month

$30 for 3 months Get Started

Then $39/month



Save 15%

$399/year Get Started

Save 15%

11+ Users


Savings start at 25%!

Request A Quote Request A Quote

Savings start at 25%!

2-10 Users


Annually per user

$300/year Get Started

$300 Annually per user

View All Plans

Get unlimited access to award-winning journalism and exclusive events.


STAT encourages you to share your voice. We welcome your commentary, criticism, and expertise on our subscriber-only platform, STAT+ Connect

To submit a correction request, please visit our Contact Us page.