Even as Amylyx’s ALS drug gains steam in the U.S. after a long-awaited FDA approval, the company is struggling with regulatory hurdles overseas.
This week, the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended against an approval for the second time this year.
The med, called Albrioza in Europe and Relyvrio in the U.S., was shot down at CHMP—again—after a formal re-examination of the committee's initial negative opinion in June.
Phase 2 data from its Centaur trial, which formed the basis of the FDA’s 2022 approval decision, didn’t sway the European regulators.
Now, the company is focused on its global phase 3 Pheonix study, which has an anticipated topline readout in mid-2024 and will provide further data on the drug’s efficacy and safety profile, Amylyx said in a statement.
The 48-week study has enrolled 664 participants across 69 sites in Europe and the U.S. If the data support an application, the company would try again for EU approval “as quickly as possible," Amylyx said.
Meanwhile, after CHMP's negative recommendation, it’s up to the European Commission to make the final call on the Albrioza application. The EC's decision is expected by the end of 2023.
“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,” Amylyx’s co-CEOs Joshua Cohen and Justin Klee said in a statement.
In the U.S., the company won approval last September after overcoming several prior regulatory setbacks.
So far, Relyvrio is putting on a strong commercial show in the U.S., generating sales of $400 million since its launch. The med is priced at about $158,000 per year and has gained widespread insurance coverage, Amylyx execs have said.