The Food and Drug Administration on Thursday approved a new medication for Amyotrophic Lateral Sclerosis (A.L.S), the debilitating and deadly neurological disorder also known as Lou Gehrig’s Disease. On Friday, the drug’s manufacturer, Amylyx Pharmaceuticals, announced the list price of the drug will be $158,000.
That list price, Pam Belluck of The New York Times notes, is far higher than the annual price of $9,100 to $30,700 recommended by the Institute for Clinical and Economic Review, a nonprofit that evaluates the value of drugs.
“Amylyx officials predicted that most patients would pay little or nothing for the treatment because the company expects insurers, both private and public, to cover it. Amylyx plans to provide it free to uninsured patients experiencing financial hardship,” Belluck reports.
She adds that the new drug, to be marketed under the name Relyvrio, was approved by the FDA “even though the agency’s analysis concluded there was not yet sufficient evidence that the medication could help patients live longer or slow the rate at which they lose functions like muscle control, speaking or breathing without assistance.”
The agency said the approval was merited even without additional evidence of the drug’s effectiveness because the medication is considered safe and “given the serious and life-threatening nature of A.L.S. and the substantial unmet need, this level of uncertainty is acceptable in this instance.”
Amylyx says that there are about 29,000 people living with A.L.S. in the United States and at least 200,000 people with the disease worldwide.
On a related note, the Office of Inspector General at the Department of Health and Human Services said this week that the F.D.A.’s accelerated approval process for drugs raised some concerns.
In a study of the agency’s accelerated approvals, the HHS watchdog found that 34% have at least one confirmatory trial past its planned completion date and four of the 278 drugs granted accelerated approval are five years or more past their original completion dates.
“The accelerated approval pathway holds promise for patients who face serious illnesses where adequate treatments are lacking,” the watchdog report said. “However, for a variety of reasons, sponsors do not always complete trials promptly. This can result in drugs staying on the market-and being administered to patients-for years without the predicted clinical benefit being verified. And insurers-including Medicare and Medicaid-paying billions for treatments that are not verified to have clinical benefit.”
The Office of Inspector General estimated that Medicare and Medicaid spent more than $18 billion from 2018 to 2021 on drugs that had been granted accelerated approval but still had incomplete confirmatory trials past their original completion dates.
Read more about the F.D.A approval process at Kaiser Health News.