While Amylyx’s ALS drug, known as Albrioza in Europe and Relyvrio in the US, has made significant strides in the United States following its long-awaited FDA approval, it is encountering regulatory obstacles overseas.
This week, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) dealt a blow to the drug, recommending against approval for the second time this year. This repeated rejection by CHMP came after a formal re-examination of the committee’s initial negative opinion in June.
Despite the positive Phase 2 data from the Centaur trial, which formed the basis for the FDA’s approval in 2022, European regulators remained unconvinced.
“We share the frustration felt by the European ALS community, who has no time to wait for new, safe, and effective treatment options. We remain committed to exploring all potential paths forward in support of the Company’s goal of getting AMX0035 to people living with ALS in the EU as quickly as possible.”
– Stéphanie Hoffmann-Gendebien, General Manager and Head of EMEA at Amylyx
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Amylyx is now directing its efforts toward the global Phase 3 Pheonix study, with an expected topline readout in mid-2024. This study will provide additional data on the drug’s efficacy and safety profile. The 48-week trial has enrolled 664 participants at 69 sites across Europe and the US. If the data from this study support an application, Amylyx plans to reapply for EU approval as expeditiously as possible.
Following CHMP’s negative recommendation, the final decision on the Albrioza application rests with the European Commission, expected by the end of 2023.
In the United States, Relyvrio has performed well commercially, amassing $400 million in sales since its launch. Priced at approximately $158,000 per year, the drug has secured broad insurance coverage, according to Amylyx executives.
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“AMX0035 is the first and only drug to show an effect on both function and survival in the same trial. Since the medication’s approval with conditions in Canada and full approval in the US, thousands of people have been prescribed AMX0035 in North America. ALS has no geographical boundaries, and we are working with urgency toward providing timely, broad, and sustainable access to AMX0035 for eligible people living with ALS who may benefit.”
– Joshua Cohen and Justin Klee, Co-CEOs of Amylyx
