HomeHealthAmylyx's hotly debated drug for Lou Gehrig's disease gets FDA approval

Amylyx’s hotly debated drug for Lou Gehrig’s disease gets FDA approval


A hotly debated drug for Lou Gehrig’s disease won approval from the US Food and Drug Administration on Thursday.

The go-ahead represents a long-desired victory for patients, though it is likely to renew doubts about the scientific rigor behind government reviews of experimental drugs, as the Associated Press reported.

The FDA approved the drug from Amylyx Pharmaceuticals based on the results of a small mid-stage study, the AP reported.

FDA PANEL VOTES TO RECOMMEND AMYLYX ALS DRUG

In the small clinical study, patients with the debilitating disease seemed to progress more slowly and survive several months longer.

Test tubes are seen in front of the Amylyx logo in this illustration, July 24, 2022.
(REUTERS/Dado Ruvic/Illustration)

“This approval provides another important treatment option for ALS, a life-threatening disease that currently has no cure,” Dr. Billy Dunn, director of neurology drugs at the FDA, said in a statement.

The FDA generally requires two large studies or one study with “very persuasive” survival results for approval.

Third drug approved for ALS

The newly approved drug is called Relyvrio.

It is the third drug approved in the US for the treatment of amyotrophic lateral sclerosis, or ALS, which destroys nerve cells needed for basic functions like walking, talking and swallowing.

“I think it shows the FDA’s ability to be easy and I think it shows a lot of tenacity on the part of ALS patients and advocates,” said Dr. Catherine Lomen-Hoerth, an ALS specialist at the University of California, San Francisco. , the AP reported.

“The company really tried to do everything they could to get this potentially promising drug to patients,” he added.

Amylyx’s drug is the latest in a series of neurological drugs that have won FDA approval despite questionable effectiveness data, according to the AP.

The agency still faces two government investigations into its approval of the Alzheimer’s drug Aduhelm last year, which has yet to be shown to slow the disease, the AP noted.

In early September, FDA advisers, reversing an earlier decision, voted to recommend approval of the drug.

But in an online memo summarizing its decision, the FDA said “regulatory flexibility” was appropriate to approve Relyvrio, “given the serious and life-threatening nature of ALS and the substantial unmet need.”

In early September, FDA advisers, reversing an earlier decision, voted to recommend approval of the drug.

The Central and Peripheral Nervous System Drugs Advisory Committee vote was 7-2 in favor of approval after hours of debate over the strength and reliability of the company’s analysis.

This 2018 photo provided by Amylyx shows company co-founders Joshua Cohen, left, and Justin Klee in Cambridge, Massachusetts.

This 2018 photo provided by Amylyx shows company co-founders Joshua Cohen, left, and Justin Klee in Cambridge, Massachusetts.
(Amylyx via AP)

In March, the panel declined to recommend FDA approval for the drug, due to what the group said was a lack of “substantially persuasive” data.

Recently, however, the FDA has faced great pressure from ALS patients, advocates and members of Congress, the AP noted.

The agency received more than 1,300 written comments from the ALS community in support of the treatment.

The company did not immediately disclose the price on Thursday, but said it planned to announce the information in a call with investors on Friday morning.

The FDA approval is expected to force insurers to cover Amylyx’s drug.

Cambridge, Massachusetts-based Amylyx did not immediately disclose the price on Thursday, but the company said it planned to announce the information in a call with investors on Friday morning, the AP reported.

In Canada, where the drug received approval in June, the company has proposed a price equivalent to $165,000.

AMX0035, as the drug is also known, is reportedly a combination of two existing products. One is a dietary supplement called taurursodiol, which can be purchased online. The other is a prescription drug called sodium phenylbutyrate, which is used to treat a rare type of metabolic disorder.

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The combination is meant to slow down ALS.

ALS is a progressive neurodegenerative disease that affects nerve cells and the spinal cord. There are two types of ALS: sporadic, which is the most common form in the US; and familial, meaning it is inherited, according to ALS.org. Scientists have yet to determine the exact cause of the disease.

Julia Musto of Fox News Digital, as well as the Associated Press and Reuters, contributed to this report.



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