ALS drug Relyvrio gains FDA approval despite warnings from some scientists:

A controversial new drug for ALS that just received FDA approval could add months to a patient’s life, but some scientists question whether it really works.

Manuel Balce Ceneta / AP

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Manuel Balce Ceneta / AP

A controversial new drug for ALS that just received FDA approval could add months to a patient’s life, but some scientists question whether it really works.

Manuel Balce Ceneta / AP

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

The decision is being praised by patients and their supporters, but is questioned by some scientists.

Relyvriomanufactured by Amylyx Pharmaceuticals of Cambridge, Mass., approved based on a single study only 137 patients. The results show that the drug can prolong patients’ lives by five to six months, or more.

“Six months can someone come to their daughter’s graduation, wedding, the birth of a child,” said Calaneet Balas, president and chief executive officer of the ALS Association. “These are really big, epic things that a lot of people want to make sure they’re around to see and be a part of.”

Balas said the approval was the right decision because patients with ALS usually die within two to five years of being diagnosed and “currently there are not many drugs available.”

But Dr. David RindThe medical director of the Institute for Economic and Clinical Research, isn’t so sure about Relyvrio, which will cost about $158,000 a year.

“I completely understand why people are trying to figure out how to get this to patients,” he said. “There’s just a general concern that maybe the trial went wrong.”

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

Proponents of the drug say that small trials have shown it to work. But FDA scientists and a panel of experts that’s FDA advice, not so sure.

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

In March, the central and peripheral nervous system drug advisory committee concluded that the Amylyx study did not provide “substantial evidence” that their drug was effective. Then in September, in a rare second meeting to consider a drug, the panel reversed course and voted in favor.

The second vote comes after Dr. Billy Dunn, director of the FDA’s Office of Neuroscience, encouraged the committee to exercise “flexibility” when considering a drug that could help people facing certain death.

A much larger study of Relyvrio, Phoenix Test, is in progress. But the result is more than a year off.

A negative outcome from that study would be a blow to Amylyx and ALS patients.

“If you have a drug that extends life by five months,” says Rind, “you have to be able to prove it in a larger trial.”

In the meantime, he said, maybe Amylix should charge less for their drugs.

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.

Klee defended the drug’s price, saying it would allow the company to develop even better treatments. “This is not a cure,” he said. “We need to keep investing until ALS is cured.”

Klee and Cohen have also promised that Amylyx will reevaluate her 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 the patient, including putting the drug on the market on our own.”

But that decision will need backing from investors and the company’s board of directors.


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