ALS drug Relyvrio nets FDA approval despite some scientists' warnings : Shots - Health News A controversial new drug for ALS could add months to patients' lives – if it actually works

ALS drug's approval draws cheers from patients, questions from skeptics

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The Food and Drug Administration has approved a controversial new drug for the fatal condition known as ALS, or Lou Gehrig's disease. NPR's Jon Hamilton reports that the decision is being hailed by patients and advocates but questioned by some scientists.

JON HAMILTON, BYLINE: The FDA approved the drug, named Relyvrio, based on a study of just 137 patients. But Calaneet Balas, president of The ALS Association, says the decision is very good news for people with the disease.

CALANEET BALAS: Right now there just aren't a lot of drugs available.

HAMILTON: And they offer only a modest benefit. As a result, people with ALS typically die within two to five years of a diagnosis. Balas says Relyvrio may give them an extra five to six months or more.

BALAS: Six months can be someone attending their daughter's graduation, a wedding, the birth of a child. These are really big, monumental things that many people want to make sure that they're around to see and be a part of.

HAMILTON: But there's an ongoing debate about the drug's effectiveness. Experts who advise the FDA voted against approval in March. Then they reversed their position during a rare second meeting a few weeks ago. Dr. David Rind of the Institute for Clinical and Economic Review says it's hard to interpret the results of a single small drug trial.

DAVID RIND: I totally understand why people would be trying to figure out a way to get this to patients. There's just a general concern out there that maybe the trial is wrong.

HAMILTON: A much larger trial of the drug is underway. Rind says a negative result would be a major blow.

RIND: If you've got a drug that's extending life by five months, you ought to be able to show that in a larger trial.

HAMILTON: In the meantime, Rind says perhaps the company should charge less than the announced price of $158,000 a year. Relyvrio is the only product made by Amylyx Pharmaceuticals. The Massachusetts company was founded by Josh Cohen and Justin Klee, who attended Brown University together. Klee says the drug is priced to allow the company to develop even better treatments.

JUSTIN KLEE: This is not a cure. We need to keep investing until we cure ALS.

HAMILTON: Klee also promised that Amylyx will reevaluate its drug based on the results of a larger trial called PHOENIX.

KLEE: If the PHOENIX trial is not successful, we will do what's right for patients, which includes taking the drug voluntarily off the market.

HAMILTON: Cohen and Klee, though, acknowledge that the decision isn't theirs alone. It would require support from the company's investors and its board of directors. Jon Hamilton, NPR News.

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