BY MATTHEW PERRONE
WASHINGTON (AP) — The maker of a
drug for Lou Gehrig’s disease that recently failed in a large study said
Thursday it will pull the medicine from the market, acknowledging it didn’t
help patients with the deadly neurological condition.
Amylyx Pharmaceuticals announced it will voluntarily halt
sales and marketing of the drug in the U.S. and Canada, where new patients will
no longer be able to get a prescription.
“While this is a difficult moment for the ALS community, we
reached this path forward in partnership with the stakeholders who will be
impacted and in line with our steadfast commitment to people living with ALS,”
company co-founders said in a statement. Patients already taking the therapy
who wish to continue will be able to enroll in a program to receive it for
free.
The Food and Drug Administration approved the much-debated
drug, Relyvrio, in September 2022, following a years-long advocacy campaign by
patients with amyotrophic lateral sclerosis, or ALS.
The drug’s failure is a bitter disappointment for patients
and advocates, who have pressed the FDA and other federal agencies to fund and
approve more experimental therapies for the fatal muscle-wasting disease.
Relyvrio’s withdrawal leaves just three ALS medicines
available to U.S. patients, only one of which has been shown to extend survival
by several months.
Cambridge, Massachusetts-based Amylyx also said Thursday it
will lay off 70% of its more than 350 employees as part of a major
restructuring effort. Company executives said they plan to continue studying
Relyvrio and another experimental drug for several rare diseases, including
Wolfram syndrome, which causes childhood diabetes and blindness.
Company shares climbed more than 7.5% in trading Thursday
morning.
Amylyx said last month it was considering pulling its drug
after a clinical trial in 600 patients failed to show any improvements in
survival or other health measures, such as muscle strength or walking ability.
The company’s voluntary action resolves what could have been
a major dilemma for the FDA. The agency’s regulators would not have had a clear
path to quickly force the drug from the market if the company had refused to
remove it. That’s because the FDA granted the drug full approval, despite the
preliminary nature of the company’s data on effectiveness.
The 2022 approval was mainly based on results from one
small, mid-stage study that was criticized by some of the agency’s own internal
scientists. Normally the agency requires two large, late-stage studies that
show a clear benefit before granting approval. But at the time FDA officials
explained that “regulatory flexibility” was appropriate when reviewing
Relyvrio, “given the serious and life-threatening nature of ALS and the
substantial unmet need.”
The medication is part of a string of drugs for deadly,
degenerative diseases that have won FDA approval in recent years despite
questionable evidence they work.
ALS gradually destroys the nerve cells and connections
needed to walk, talk, speak and breathe. Most patients
die within three to five years of a diagnosis.
Relyvrio is a powder that combines two older drugs: a
prescription medication for liver disorders and a dietary supplement associated
with traditional Chinese medicine.
Amylyx faced criticism for pricing the drug at $158,000 for
a year’s supply. Sales were disappointing, with some patients
discontinuing the medicine after only a few months.
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The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute’s Science and Educational Media Group. The AP is solely responsible for all content.