- A committee advising the European Medicines Agency is signaling it may recommend against approval of Amylyx Pharmaceuticals’ ALS drug, the company said Tuesday.
- Amylyx said it was informed the Committee for Medicinal Products for Human Use, or CHMP, “is trending toward a negative opinion” of the medicine, which is sold in the U.S. as Relyvrio. The committee next meets in June.
- Amylyx, for its part, plans to request a formal re-examination if the CHMP issues a negative verdict, a process that could take up to four months. The company “disagree[s] with the current view” of the committee and “remain[s] confident” in the data from the key clinical trial of Relyvrio, said Tammy Sarnelli, its head of regulatory affairs and clinical compliance, in a statement.
The Food and Drug Administration approved Relyvrio last September, but not without controversy. Prior to the drug’s clearance, the FDA had requested the company start another large trial, but eventually approved the drug before Amylyx got the results.
That approval was largely based on the findings from a smaller study that found patients who took the treatment declined slower than those who’d received a placebo. Those prescribed the drug also lived slightly longer as well.
Sales of the treatment, which costs $158,000 per year in the U.S., have taken off since then, surpassing the expectations of Wall Street analysts. In the first quarter of this year, the drug generated more than $71 million for Amylyx.
Yet further growth could be harder to achieve if Amylyx can’t bring the drug to market in Europe.
The FDA and EMA have had opposing views of new drugs before. Just last week, the European regulator recommended pulling the approval for Novartis’ sickle cell disease drug Adakveo, which the FDA cleared for use in 2019.
The EMA could still eventually end up issuing a positive verdict for Relyvrio. Statistics cited by Evercore ISI analyst Michael DiFiore in a client note Tuesday show the CHMP has changed its opinion about 39% of the time when drug applications have been re-examined. That history “makes us believe that [Amylyx] still has a shot” in Europe, DiFiore wrote.
“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,” said Stéphanie Hoffmann-Gendebien, Amylyx’s head and general manager of international markets.
Amylyx shares fell about 2% in trading on Tuesday.