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

UsernamePublicRestriction
UsernamePublicRestriction

‘File Over Protest’ At US FDA: PTC To Pursue Rarely-Used Pathway For DMD Drug

Executive Summary

Translarna’s best path forward is the file over protest pathway for the application, which received a refuse to file letter last year, PTC says; former OND Director John Jenkins calls it ‘a fairly rare event.’

You may also be interested in...



Refining A Controversial Endpoint: Sarepta Banking On Dystrophin Levels For Golodirsen Approval In DMD

While glodirsen did show higher levels of dystrophin production than eteplirsen did, increase is still unlikely to satisfy US FDA reviewers who disagreed with Janet Woodcock's decision to grant accelerated approval to eteplirsen.

Keeping Track: Apadaz Approved Without Abuse-Deterrent Label, FDA Denies Translarna Appeal, And Some Priority Reviews

The latest drug development news and highlights from our US FDA Performance Tracker.

Keeping Track: A Burst of CRLs and BTDs, A Busy Week Of Submissions And Another Approval For Soliris

The latest drug development news and highlights from our US FDA Performance Tracker.

Related Content

Topics

Related Companies

UsernamePublicRestriction

Register

PS119793

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

Cancel