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


US FDA Versus Its Own Precedents

Executive Summary

The US FDA has shown astonishing flexibility in approving new drugs for neurodegenerative diseases based on efficacy evidence that falls well short of historical standards. Now the agency faces the delicate challenge of demonstrating that there are still some lines it will not cross.

You may also be interested in...

BrainStorm Eyes New Phase III Trial For NurOwn In Mild ALS Following BLA Withdrawal

Highly negative FDA advisory committee review and subsequent discussions with the agency made clear the only path forward was a new study, company executives said. Troubled application shows the limits of FDA’s willingness to exercise regulatory flexibility.

Regulatory Flexibility: BrainStorm’s Problematic NurOwn BLA May Be A Bridge Too Far For US FDA

Litany of deficiencies, including product quality shortcomings and a failed Phase III clinical efficacy study, may make it impossible for the agency to exercise the type of flexibility it has with the approval of other treatments for ALS and neurodegenerative diseases.

Sarepta’s DMD Gene Therapy Helped Across Accelerated Approval Finish Line By CBER’s Peter Marks

FDA clinical, clinical pharmacology and statistical review teams did not recommend approval of Elevidys, but the CBER director concluded that randomized data in a subgroup of patients ages four and five years old were ‘compelling,’ justifying accelerated approval; expansion beyond this age-restricted subgroup will hinge on data from the EMBARK confirmatory trial.

Related Content


Related Companies

Latest Headlines
See All



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