Amylyx announced an update on the ongoing review of its Marketing Authorisation Application (MAA) for AMX0035 (sodium phenylbutyrate and ursodoxicoltaurine [also known as taurursodiol]) for the treatment of adults with amyotrophic lateral sclerosis (ALS) by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). Following an oral explanation held at the May meeting of the CHMP, Amylyx was informed that the CHMP is trending toward a negative opinion on the application for conditional marketing authorisation of AMX0035.
There has not been a new product approved in the European Union for ALS in over 25 years. While our MAA is under review, we remain committed to exploring all potential paths forward given people living with ALS have no time to wait.

“We disagree with the current view expressed by the CHMP and remain confident in the data from the CENTAUR trial. Should a negative opinion ultimately be issued, we intend to request a formal re-examination procedure,” said Tammy Sarnelli, Global Head, Regulatory Affairs and Clinical Compliance at Amylyx.

The CHMP is expected to adopt a formal opinion on the MAA at its next meeting, which will be held June 19-22, 2023. If a negative opinion is received in June, the Company intends to request a formal re-examination of the opinion, which is an approximately four-month process.

The MAA for AMX0035 is based on data from the CENTAUR clinical trial, a randomized, multicenter, placebo-controlled trial in participants with ALS (n=137), which were the basis of the full approval received from the U.S. Food and Drug Administration (FDA) and approval with conditions by Health Canada. The trial met its prespecified primary outcome and AMX0035 is the first ALS therapy to demonstrate, in the same trial, both a statistically significant benefit in function, as well as an observed benefit on survival in a longer-term post hoc analysis. The CENTAUR data were published in the peer-reviewed medical journals the New England Journal of Medicine, Muscle & Nerve, and the Journal of Neurology, Neurosurgery, and Psychiatry. AMX0035 demonstrated a generally well-tolerated safety profile in the CENTAUR trial, with similar reported rates of adverse events and discontinuations in AMX0035 and placebo groups during the 24-week randomized phase; however, gastrointestinal events occurred with greater frequency (≥2%) in the AMX0035 group.

ALS affects approximately 29,000 people in the U.S. and more than 30,000 people are estimated to

be living with ALS in Europe (European Union and United Kingdom). In the first six months following FDA approval in the U.S., as of March 31, 2023, there were roughly 3,000 people actively taking RELYVRIO® in the U.S.
“There has not been a new product approved in the European Union for ALS in over 25 years. We know from the European ALS community that there is a desperate need for new therapeutic options for this devastating disease, and timely access to safe and effective treatments is especially important. While our MAA is under review, we remain committed to exploring all potential paths forward given people living with ALS have no time to wait,” said Stéphanie Hoffmann-Gendebien, Head, General Manager – International Markets, EMEA at Amylyx.

About RELYVRIO®/ALBRIOZA™/AMX0035

RELYVRIO®, an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol (known as ursodoxicoltaurine outside of the U.S.), is approved to treat amyotrophic lateral sclerosis (ALS) in adults in the U.S. and approved with conditions as ALBRIOZA™ for the treatment of ALS in Canada. Additionally, the European Medicines Agency (EMA) is reviewing the Company’s Marketing Authorisation Application for AMX0035 for the treatment of ALS in Europe and recently informed the Company that the CHMP is trending toward a negative opinion on the AMX0035 application. AMX0035 is being explored for the potential treatment of other neurodegenerative diseases. The formulation of RELYVRIO, ALBRIOZA and AMX0035 are identical.

Source: Press release Amylyx