Regulatory Update: Amylyx’s ALS drug faces rejection by the European Medicines Agency (EMA)

Regulatory Update: Amylyx's ALS drug faces rejection by the European Medicines Agency (EMA)

Source – Amylyx Pharmaceuticals

On June 23, 2023, the European Medicines Agency (EMA) has formally issued a negative opinion regarding the approval of Amylyx Pharmaceuticals’ amyotrophic lateral sclerosis (ALS) therapy, known as Relyvrio in the US and Albrioza outside the US In May, Amylyx had already indicated that the EMA was leaning towards a negative opinion on the drug’s application.

The EMA expressed concerns that the primary CENTAUR study did not convincingly demonstrate the effectiveness of Albrioza in slowing down the progression of the disease. The agency questioned the reliability of the collected and analyzed survival data.

In response, Amylyx has announced its intention to request a formal re-examination of the application, a process that is expected to take approximately four months.

“We are confident in the strength of our CENTAUR trial data, which we believe meets the criteria for conditional approval. These data were also the basis of the full approval received from the U.S. Food and Drug Administration and the approval with conditions from Health Canada. We disagree with the CHMP’s opinion and will request a formal re-examination procedure of the current Marketing Authorisation Application (MAA).”

Tammy Sarnelli, Global Head, Regulatory Affairs and Clinical Compliance at Amylyx

The CENTAUR trial successfully achieved its predetermined primary outcome, establishing AMX0035 as the first therapy for ALS to demonstrate both a statistically significant improvement in function and a observed benefit in overall survival during a longer-term post hoc analysis. Throughout the CENTAUR trial, AMX0035 exhibited a generally well-tolerated safety profile, with comparable rates of adverse events and discontinuations reported between the AMX0035 and placebo groups over the 24-week randomized phase. However, gastrointestinal events were more frequent (≥2%) in the AMX0035 group.

“While our MAA continues to be under review, we will also work towards completing our global Phase III PHOENIX study, with topline results anticipated in mid-2024, which will provide important additional data on the efficacy and safety profile of ALBRIOZA. We remain committed to exploring all potential paths forward. There have been no new innovations approved in Europe for this devastating disease in over 25 years, and we recognize the urgent need of the ALS community in Europe to access new treatment options.”

Stephanie Hoffmann-Gendebien, General Manager and Head of EMEA at Amylyx

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