Illustration: Natalie Peeples/Axios
The FDA's expedited approval of a new ALS treatment priced at $158,000 a year, has touched off another debate over balancing regulation with patient access.
Why it matters: ALS, or Lou Gehrig's disease, is a dreaded neurological condition whose victims usually die within three years of the onset of symptoms. But there's limited evidence the newly approved drug works, or that its price is justified.
Driving the news: The FDA on Thursday announced the approval of Relyvrio, an experimental drug combination from Amylyx Pharmaceuticals. It's the first ALS drug to win approval in five years and only the third in the agency's history.
- The approval was granted without the clinical trial evidence the agency usually requires. The FDA originally recommended Amylyx apply for approval after the Phase 3 trial was completed in 2024, but backed off after intense campaigning by patient advocacy groups, per the NYT.
- "Given the serious and life-threatening nature of ALS and the substantial unmet need, this level of uncertainty is acceptable in this instance and consideration of these results in the context of regulatory flexibility is appropriate," the FDA wrote in a benefit-risk assessment.
- Amylyx has said the list price of Relyvrio will be $158,000 a year.
Between the lines: Advocates and experts quickly criticized the price as excessive. An analysis by the Institute for Clinical and Economic Review had previously said that the drug would be cost-effective if it was priced between $9,100 and $30,600 per year.
- "$158,000 annually for a medicine that is not a biologic or complicated delivery device or new molecule, but rather the combination of two drugs that already exist and have for a while," tweeted Walid Gellad, a professor of medicine and health policy at the University of Pittsburgh.
- "The issue in the US will be the sky high cost combined with residual uncertainty about level of efficacy," Gellad added.
The intrigue: The company previously told the agency that it would voluntarily withdraw Relyvrio from the market if its shown to be ineffective in its Phase 3 trial. Its co-CEO said on Friday that he expected regulators to order such a withdrawal if circumstances merit, and that the company "will always do what is right for patients."
The big picture: The entire episode recalls last year's controversial FDA approval of Aduhelm, an Alzheimer's treatment that also had limited evidence that it worked.
- But Aduhelm has serious side effects, raising the question of whether it was ethical to put the drug in patients' hands before it was even clear it could help them.
- Such safety questions aren't part of the debate over Relyvrio, which the FDA says has "no significant safety signals of concern."
Be smart: Federal law now has the power to limit the prices of old drugs or the price increases of drugs already on the market, but can do nothing to constrain the launch prices of new drugs.
- Eye-popping launch prices are now the norm, and in some cases experts largely agree they may even be warranted.
- But it's against this backdrop that the drug pricing debates of the future will play out.
- Meanwhile, the FDA continues to use expedited approvals for pricey cancer drugs and other treatments.
Editor's note: This story has been updated to incorporate a more comprehensive account of the Amylyx co-CEO’s remarks on an investor call. It removed a reference to another media outlet's report that didn't give a full account of what was said.