ALS drug Relyvrio gets FDA approval despite warnings from some scientists: Shots


A controversial new drug for ALS that just received FDA approval could add months to the lives of patients, but some scientists question whether it actually works.

Manuel Balce Ceneta/AP


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Manuel Balce Ceneta/AP

ALS drug Relyvrio gets FDA approval despite warnings from some scientists: Shots

A controversial new drug for ALS that just received FDA approval could add months to the lives of patients, but some scientists question whether it actually works.

Manuel Balce Ceneta/AP

The Food and Drug Administration has approved a controversial new drug for the deadly disease known as ALS, or Lou Gehrig’s disease.

The decision is welcomed by patients and their advocates, but questioned by some scientists.

Relyvrio, made by Amylyx Pharmaceuticals of Cambridge, Mass., was approved based on a single study involving just 137 patients. The results suggest that the drug could extend patients’ lives by five to six months or even longer.

“Six months can be someone attending their daughter’s graduation, a wedding, the birth of a child,” says Calaneet Balas, president and CEO of the ALS Association. “These are really big and monumental things that a lot of people want to make sure they’re there to see and be a part of.”

Balas says the approval was the right move because ALS patients typically die within two to five years of a diagnosis, and “right now there just aren’t a lot of drugs available.”

But Dr. David Rind, chief medical officer of the Institute for Clinical and Economic Review, isn’t so sure about Relyvrio, which will cost around $158,000 a year.

“I totally understand why people are trying to find a way to get this to patients,” he says. “There’s just a general concern out there that maybe the lawsuit is bogus.”

ALS kills about 6,000 people a year in the United States by gradually destroying nerve cells that control voluntary movements, such as walking, talking, eating and even breathing. Relyvrio, a combination of two existing products, is intended to slow down the disease process.

Proponents of the drug say the small trial showed it worked. But FDA scientists and a panel of experts advising the FDA weren’t so sure.

Typically, FDA approval requires two independent studies — each with hundreds of participants — showing efficacy, or one large study with clearly positive results.

In March, the Peripheral and Central Nervous System Drugs Advisory Committee concluded that the Amylyx study did not provide “substantial evidence” that its drug was effective. Then in September, in a rare second meeting to review a drug, the panel backtracked and voted in favor of approval.

The second vote came after Dr. Billy Dunn, director of the FDA’s Office of Neuroscience, encouraged the committee to be “flexible” when reviewing a drug that could help people facing a dying certain.

A much larger study of Relyvrio, the Phoenix Trial, is underway. But the results are over a year away.

A negative result of this study would be a blow for Amylyx and ALS patients.

“If you have a drug that prolongs life by five months,” Rind says, “you should be able to demonstrate that in a larger trial.”

In the meantime, he says, maybe Amylix should charge less for his drug.

Relyvrio (marketed as Albrioza in Canada) is the only product made by Amylyx, a company founded less than a decade ago by Joshua Cohen and Justin Klee, who attended Brown University together.

Klee defends the price of the drug, saying it will allow the company to develop even better treatments. “It’s not a cure,” he says. “We must continue to invest until we cure ALS.”

Klee and Cohen also promised that Amylyx would reevaluate its drug based on the results of the Phoenix trial.

“If the Phoenix trial isn’t successful,” Klee said, “we’ll do what’s right for patients, which includes voluntarily removing the drug from the market.”

Cohen and Klee, however, acknowledge that the decision would require the support of the company’s investors and its board of directors.


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