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

Amylyx’s ALS Drug Brings Questions On Efficacy Data Robustness To US FDA Panel

Executive Summary

Citing numerous concerns about design of the Phase II CENTAUR trial and statistical robustness of the results, agency reviewers question whether a single study provides substantial evidence of efficacy for approval of AMX0035.

You may also be interested in...



Regulators Still To Get To Grips With Neurodegenerative Outcome Measures

Outcomes measures in the neurodegenerative disease space are not yet well understood by regulators, according to Amylyx, which recently received a negative opinion from the EMA for its amyotrophic lateral sclerosis drug, Albrioza.

At Tofersen Panel Review, US FDA Shined A Light On Internal Differences Of Opinion

Agency’s presentations on Biogen/Ionis’ ALS drug included differing viewpoints from the statistical and clinical pharmacology teams; the transparency and emphasis on a multidisciplinary review approach may reflect lessons learned from missteps at the Aduhelm panel review in 2020.

Regulatory Flexibility: US FDA Approves Amylyx’s Relyvrio For ALS Despite ‘Degree Of Residual Uncertainty’

The single positive Phase II CENTAUR study, along with confirmatory evidence of benefit on long-term survival in the open-label extension, demonstrate substantial evidence of effectiveness, the agency says, acknowledging a level of uncertainty that is acceptable in the disease context.

Related Content

Topics

Related Companies

Latest Headlines
See All
UsernamePublicRestriction

Register

PS145928

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