Just one day after Amylyx Pharmaceuticals’ new amyotrophic lateral sclerosis (ALS) med Albrioza passed muster with Canadian regulators, U.S. watchdog ICER has taken aim at the drug’s cost-effectiveness in a new draft report.
Amylyx has yet to reveal the price for its med, but if the cost were akin to older ALS drug edaravone—around $169,000 a year, according to ICER—then the incremental cost-effectiveness of Amylyx’s med, known as AMX0035 in the U.S., would “far exceed typical thresholds," ICER said Tuesday.
Still, it’s important to note that Albrioza’s true cost-effectiveness will depend on its official price, plus “confirmation of its clinical benefits,” the cost watchdog added. ICER plans to finalize its report in early August following a public comment period, Endpoints News points out. The organization’s CMO told the news outlet it would be tough to truly determine Albrioza’s cost-effectiveness until Amylyx reveals the med’s price.
Amylyx’s drug is up for a decision at the U.S. FDA by Sept. 29.
As far as a potential accelerated approval is concerned, if the med receives a green light while another clinical trial is underway, “policymakers should debate short-term pricing options including a far lower price close to the cost of production until the benefits of treatment can be adequately evaluated,” ICER argued.
On clinical benefit metrics, Albrioza added to standard of care, proved “comparable or better” versus standard of care alone, according to ICER.
Still, that endorsement wasn’t without its caveats, either.
Albrioza was evaluated in the 24-week Centaur trial, plus the open-label extension Centaur-OLE. That study found the drug charted “modest benefits” in slowing ALS progression during the randomization phase, plus a 5-month survival benefit with longer follow-up, ICER noted. That said, the organization “tempered” its rating for the med because the evidence it relied upon was based on a single, small, “fair-quality” randomized trial burdened by “several methodological concerns,” ICER argued.
Putting it simply, “[c]linical experts are divided on whether AMX0035 is effective,” ICER said.
Despite the methodological concerns, ICER noted that the med’s open-label extension “raises the possibility of important survival benefits.” The drug also appears largely safe, ICER said.
ICER noted in its draft report that there’s a “tremendous need” for new ALS therapies. With that context in mind, “pricing at the high end of—or even beyond—traditional cost-effectiveness ranges might be considered,” the organization explained.