Pink Sheet is part of Pharma Intelligence UK Limited

This site is operated by Pharma Intelligence UK Limited, a company registered in England and Wales with company number 13787459 whose registered office is 5 Howick Place, London SW1P 1WG. The Pharma Intelligence group is owned by Caerus Topco S.à r.l. and all copyright resides with the group.

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

The Eteplirsen Precedent: No Denying It – So Let’s Define It

Executive Summary

US FDA allowing Sarepta to seek accelerated approval for follow-on DMD agent – following 'precedent' set by eteplirsen. Now the new challenge is to define exactly what that precedent is.

You may also be interested in...



US FDA Advisory Committee Reboot (Part I): Open Public Meeting A Place To Start?

CDER Director Patrizia Cavazzoni raised eyebrows when she stated an intent to remove the ‘emotional overtone’ of committee meetings. The open public hearing may be a place to start.

Aduhelm’s ‘Complex’ Circumstances Drove Extensive In-House Advice Process Before Accelerated Approval, Cavazzoni Says

Accelerated approval was considered as option in response to the negative advisory committee vote, but it was not a ‘default,’ CDER officials say. ‘It fit like a glove.’

PTC’s Ataluren and Accelerated Approval: Is Eteplirsen A Precedent After All?

US FDA appears willing to consider accelerated approval for PTC's Duchenne Muscular Dystrophy therapy ataluren if company is able to provide compelling evidence of increased levels of dystrophin in boys with nonsense-mutation DMD. That is quite a reversal from the agency’s view heading into PTC’s appeal. 

Related Content

Topics

Related Companies

Latest News
UsernamePublicRestriction

Register

PS122711

Ask The Analyst

Ask the Analyst is free for subscribers.  Submit your question and one of our analysts will be in touch.

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