Pink Sheet is part of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By


Sarepta, FDA And The Dangers Of Strong Early Results

Executive Summary

The small Duchenne patient community is already convinced eteplirsen works, but FDA doesn't seem to agree and the advisory committee didn't seem to offer a way out except through a controlled trial.

You may also be interested in...

Duchenne Development: Six-Minute Walk Losing Favor?

Sarepta relied on the endpoint to get accelerated approval for eteplirsen, but the interim CEO says trials for follow-on products could use walk-run tests or other measures instead.

Sarepta's Eteplirsen Approved After Contentious Internal FDA Debate

CDER Director Woodcock grants accelerated approval after FDA Commissioner Califf refuses to overturn her finding that the drug's effect on dystrophin is reasonably likely to predict clinical benefit in Duchenne muscular dystrophy.

FDA Neurology Clinical Team Leader Departure May Be Mountain Disguised As Molehill

Attention to the departure of neurology reviewer Ron Farkas from FDA has focused on what it means for Sarepta’s eteplirsen application, but there are larger regulatory environment issues that deserve more scrutiny.

Related Content


Related Companies




Ask The Analyst

Please Note: You can also Click below Link for Ask the Analyst
Ask The Analyst

Your question has been successfully sent to the email address below and we will get back as soon as possible. my@email.address.

All fields are required.

Please make sure all fields are completed.

Please make sure you have filled out all fields

Please make sure you have filled out all fields

Please enter a valid e-mail address

Please enter a valid Phone Number

Ask your question to our analysts