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FDA approves drug against Amyotrophic Lateral Sclerosis (ALS) despite little evidence on its effectiveness

2022-09-30T02:06:01.422Z

The disease, known in the US as Lou Gehrig, is a condition with no cure and for which there are few treatments. The pharmaceutical company that developed the Relyvrio drug has not yet announced how much it will cost, but it is expected to be in the hundreds of thousands of dollars.



By Berkeley Lovelace Jr. -

NBC News

The United States Food and Drug Administration (FDA) approved this Thursday a controversial new drug designed to slow the progress of Lou Gehrig's disease, also called Amyotrophic Lateral Sclerosis or ALS.

The green light from the federal agency means a victory for patients and activists,

despite limited evidence on the drug's efficacy.

The drug, from Massachusetts-based Amylyx Pharmaceuticals, joins

the few drugs approved by the FDA

to treat this deadly neurodegenerative disease and its symptoms. 

ALS affects the nerve cells needed for activities such as walking, talking, and eating.

There is no known cure, and most people who have it live two to five years after diagnosis, according to the ALS Association.

The FDA has approved a new drug for Lou Gehrig's disease, also called amyotrophic lateral sclerosis or ALS. NurPhoto via Getty Images

The drug, which is taken in pill form, is a combination of two existing products: sodium

phenylbutyrate

, which is prescribed to treat a metabolic disorder, and

taurursodiol

, an over-the-counter supplement used to help prevent liver diseases.

The FDA's decision was based on a single Phase 2 clinical trial, involving 137 ALS patients, which found that people taking the Amylyx drug, to

be sold under the name Relyvrio

, lived about 10 months longer than those that they did not receive it.

The drug also seemed to delay hospitalizations. 

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The drugmaker said it is still determining what it will charge for the drug.

Its price may be similar to that of an older ALS drug, edaravone, which

costs about $170,000 a year

, according to a report by the Institute for Clinical and Economic Review.

Disagreement among neurologists

The approval is likely to cause some disagreement among neurologists who treat ALS.

The FDA

generally requires at least two well-controlled clinical trials

to show that a drug is effective, or a single trial that is "highly statistically persuasive," said Holly Fernandez Lynch, assistant professor of medical ethics at the University of Pennsylvania.

Amylyx's essay, he added, did not meet agency standards.

[Denying a dignified death to Martha Sepúlveda is "disrespectful" and "illegal": the family of the Colombian woman who asks to die by euthanasia reacts]

Concern about the trial results arose in March, when the drug was first presented to an FDA advisory committee.

In briefing papers released before that meeting,

agency scientists questioned the persuasiveness of the Amylyx trial

.

The committee narrowly voted against recommending approval of the drug.

However, the FDA took the unusual step of convening a second advisory committee just six months later, after Amylyx submitted additional analysis of its trial data.

At that meeting, the advisory committee changed its mind and voted to recommend the drug.

After a long battle, a Colombian woman suffering from a degenerative disease dies by euthanasia

Jan. 10, 202201:48

Advocates for more treatments for the disease say that while questions remain about the drug's effectiveness,

patients should at least be allowed to try it.

"We need new treatments as soon as possible if we want to make ALS a liveable disease and eventually cure it," Larry Falivena, a member of the ALS Association, said via email.

[FDA authorizes COVID-19 vaccine booster tailored for omicron variant]

Amylyx is currently conducting a larger Phase 3 clinical trial on the drug, which it expects to complete

in late 2023 or early 2024. 

Source: telemundo

All news articles on 2022-09-30

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