Sarepta receives new FDA guidance on data needed for eteplirsen's NDA in Duchenne Muscular Dystrophy

28 October 2014

Rare and infectious disease specialist Sarepta (Nasdaq: SRPT) provided an update on its discussions with the US Food and Drug Administration regarding its planned New Drug Application submission for the approval of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD).

The FDA provided updated guidance regarding the specific data to be included as part of Sarepta’s NDA submission in meeting minutes the company received last week from a Type B Pre-NDA meeting in September 2014.

This guidance states that additional information is now required as part of the submission for an NDA, including results from an independent assessment of dystrophin images and the 168-week clinical data from study 202. More specific data including minimum duration of safety in new patients exposed to eteplirsen, patient-level natural history data and MRI data from a recent independent academic study are also required.

This article is accessible to registered users, to continue reading please register for free. A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.

Try before you buy

Free

7 day trial access

Take a Free Trial

All the news that moves the needle in pharma and biotech
Exclusive features, podcasts, interviews, data analyses and commentary from our global network of life sciences reporters.
Receive The Pharma Letter daily news bulletin, free forever.

Become a subscriber

£820

Or £77 per month

Subscribe Now

Unfettered access to industry-leading news, commentary and analysis in pharma and biotech.
Updates from clinical trials, conferences, M&A, licensing, financing, regulation, patents & legal, executive appointments, commercial strategy and financial results.
Daily roundup of key events in pharma and biotech.
Monthly in-depth briefings on Boardroom appointments and M&A news.
Choose from a cost-effective annual package or a flexible monthly subscription

The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed

Chairman, Sanofi Aventis UK