After a long road filled with scrutiny and uncertainty, Amylyx’s amyotrophic lateral sclerosis (ALS) drug, known as AMX0035, has finally scored FDA approval.
The drug, now branded as Relyvrio, won approval for the treatment of ALS in adults. It's the first ALS treatment that showed a significant slowing in both disease progression and functional decline, as well as extended survival, in a randomized clinical trial.
After a first FDA advisory committee meeting in March ended with a negative 6-4 vote for the drug, many companies would have stopped there. But Amylyx was persistent, and the committee reconvened this September to discuss a new analysis the company submitted, resulting in a positive 7-2 vote. The long stretch of time helped the company prepare for an eventual launch, despite not knowing when, or if, its drug would get approval.
“We’ve been planning this a long time,” said Josh Cohen, Amylyx’s co-CEO and co-founder, referring to the drug’s original FDA target decision date of June 29. “We’re fully prepared,” Cohen said in an interview with Fierce Pharma ahead of the approval.
The drug will carry a list price of about $158,000 per year in the U.S., the company said on a post-approval conference call, noting that the price is below the latest FDA-approved ALS product. However, Amylyx is “committed to providing financial assistance” for patients with commercial insurance and will provide the drug at no cost for underinsured or uninsured patients who meet “certain financial eligibility criteria” and who have “exhausted all other options,” said Amylyx’s chief commercial officer Margret Olinger.
The price, before being revealed, was already criticized by U.S. watchdog ICER in June. Using a placeholder price of $169,000 per year, the organization said the cost-effectiveness of the medicine would “far exceed typical thresholds."
The FDA based its approval on data from the phase 2 Centaur trial and biomarker results from a phase 2 of the drug in Alzheimer’s disease. At the recent FDA expert panel meeting, some panelists voiced a desire to wait for the results of the phase 3 Phoenix trial. The company doesn’t expect top-line results from that trial until 2024, Justin Klee, Cohen’s counterpart as co-CEO and co-founder, said in an interview with Fierce Pharma.
“For people with ALS, that’s a long, long time,” Klee said. If the agency were to wait until the results of that trial came out, “a whole generation of ALS patients will die without access,” Cohen added. “It seems against common sense to deny that to people.”
From pricing to insurance to supply, Amylyx is set and covered, Cohen said. But the company wants to do more than “just launch a drug into the market.”
“ALS has a lot of other things that need changing,” Cohen said. From diagnoses that take too long, “woefully underfunded” clinics, and challenges barring access for patients, there are a number of unaddressed, long-standing problems that need addressing, and that Amylyx wants to “put a pretty significant effort” into fixing.
Amylyx isn’t the only one tuned into these issues. The ALS Association is also celebrating the approval. That organization invested $2.2 million of funds raised through 2014’s ALS ice bucket challenge into the development of the drug, the association said in a statement. It also led the years-long advocacy campaign to get the treatment approved, including submitting over 50,000 signatures to the FDA in September 2020 calling on the agency to approve the drug.
A key factor for Relyvrio is its function and survival benefit, which is historically a difficult endpoint to accomplish, and one no other treatment can claim. But to Amylyx’s founders, that’s “just the tip of the iceberg.”
“We also want to invest in research—we don’t want this to be the last drug,” Cohen said. “ALS is an underfunded disease, and we want to be able to dedicate substantial resources to going back into research and drug development, to keep advancing the bar and advancing the needle.”
The drug is already approved in Canada, and Amylyx expects approval in Europe “sometime in roughly the first half of the coming year,” Klee said. But the company knows that ALS is a global disease and is working to “quickly get the drug to every patient that can benefit.” So as for launches in other countries—“stay tuned,” Cohen said.