Payers May Balk at Newly Approved ALS Drug’s $158K Price

by Tim Casey   The FDA on Sept. 29 approved Relyvrio (sodium phenylbutyrate/taurusodiol), making it only the third medication to treat patients with amyotrophic lateral sclerosis (ALS). While ALS advocates and some clinicians applauded the agency’s decision, other providers have questioned the drug’s effectiveness, and some experts believe the drug is not cost-effective with its approximately $158,000 annual wholesale acquisition cost (WAC).     Industry reacts to high price  The nonprofit Institute for Clinical and Economic Review (ICER) found that Relyvrio would be cost-effective if it was priced at between $9,100 and $30,600 per year. And Bruce Booth, a former scientist who’s now a partner at the Atlas Venture biotechnology venture capital firm, wrote on Twitter that the $158,000 price for Relyvrio was “even more egregious than I thought!”   Relyvrio’s manufacturer, Amylyx Pharmaceuticals, Inc., noted the WAC is lower than the most recent ALS drug to hit the market: the oral form of Radicava (edaravone), which the FDA approved in May.  Amylyx expects the drug will be available by late October or early November. The company has been in negotiations with health insurance companies and PBMs for months even before the FDA approval.   Approval process was closely monitored  Relyvrio’s approval process was closely watched, particularly because the FDA made the unusual move to hold two advisory committee meetings before making its decision.   Adam Quick, M.D., a neurologist at the Ohio State University Wexner Medical Center who specializes in ALS, tells AIS Health that the FDA typically only approves drugs that have shown to be effective in two placebo-controlled trials. But in this case, the agency made an exception because of the disease’s severity and lack of treatment options on the market.   Quick says he would prescribe Relyvrio to all of his patients when it’s available to use with the other ALS medications “with the hope that since these are medicines that work differently from one another that they could each have some effect on the disease, and maybe that effect is greater than using one medicine by itself.”  Drug reminds some of Alzheimer’s controversy  The lack of clarity about the drug’s effectiveness, its high cost and the severity of the disease it treats has caused many people to compare Relyvrio with Aduhelm (aducanumab), an Alzheimer’s medication that the FDA approved in June 2021.    In April, CMS said it would cover Aduhelm only for people enrolled in randomized, controlled trials conducted either through the FDA or the National Institutes of Health. And UnitedHealthcare, the U.S.’s largest health insurer, said in May that it would restrict access to Aduhelm across all of its channels.    After the FDA approved Relyvrio late last month, Booth hinted that Amylyx could have a similar coverage issue if the WAC remains anywhere close to its current $158,000. He wrote on Twitter that “As Aduhelm demonstrated, insurers/providers may disconnect their approval processes from the FDA’s drug approval process where [a] huge gap in price vs. proven value [exists]. A disconnect that is a bad precedent for the sector.”     From Radar on Drug Benefits   Subscribers may read the in-depth article online. Learn more about subscribing to AIS Health's publications.