The long-awaited decision by the European committee CHMP was announced recently by Amylyx Pharmaceutical (the Company) based in Cambridge, Massachusetts just as anticipated. The EMA issued its negative opinion of the conditional marketing application for authorization of AMX003 to treat adults with ALS.
Amylyx disagrees with the opinion of the Committee for Medicinal Products for Human Use (CHMP) and will request a re-examination of the recently submitted application which may take at least four months. Subsequently, after a thorough review, the CHMP will issue its final opinion.
The Company issued a news release of its confidence in the results of its CENTAUR trial which it believes brought about the FDA and Canadian approvals. Canada Health added several conditions. The FDA approved AMX003, now known by its trade name of ALBRIOZA® in September of 2022.
About the CENTAUR Clinical Trial
The CENTAUR trial Phase 2 (NCT05021536) enrolled 137 participants over a six-month period. The trial reached its desired outcome. In a later analysis, AMX0035 demonstrated benefits for patient survival. AMX0035 now known by its brand name, Relyvrio, demonstrated a safety profile that was well tolerated. Gastrointestinal events were more prevalent, however.
The CENTAUR data appeared in the NEJM and the Journal of Neurology among other publications. Amylyx Co-CEOs expressed their determination to continue to engage the EMA and the CHMP in the examination process in order to have ALBRIOZA available to ALS patients in Europe.
Amylyx is committed to ensuring acceptance of its application as they are aware that no therapies have been approved in the EU to treat ALS in the past 25 years. Life expectancy is two to five years.
An estimated 29,000 people are affected in the United States while over 30,000 are said to be living with ALS in the EU and UK.
To show that the Company recognizes the urgency of creating additional data on the profile of ALBRIOZA, Amylyx is also speeding up the development of its PHOENIX study anticipated completion is 2024.
ALBRIOZA is a fixed-dose combination of sodium phenylbutyrate and taurursodiol for the treatment of adults with ALS. It is known as ursodoxicoltaurine in other countries. RELYVRIO, ALBRIOZA, and AMX0035 use an identical formulation.
The CENTAUR Trial (NCT03127514)
Over 90% of ALS patients have sporadic disease which is defined as having no clear evidence of a family history of ALS.
The Relentless Progression of ALS
ALS is a fatal neurodegenerative disease caused by the death of neurons in the spinal cord and brain. The loss of motor neurons causes muscle function to decline and also interferes with the ability to speak, move, and eventually causes death.
Amylyx was clear that it disagrees with CHMP’s opinion, and it will request a re-examination of the recently submitted application.