Manuel Balce Cenata/AP
The Food and Drug Administration (FDA) has approved a controversial new drug for the deadly condition known as ALS, or Lou Gehrig’s disease.
The decision is being hailed by patients and their advocates, but questioned by some scientists.
reliefmanufactured by Amylyx Pharmaceuticals of Cambridge, Mass., was approved based on a single study of only 137 patients. The results suggested that the drug could prolong the lives of patients by five to six months or more.
“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, monumental things that a lot of people want to make sure they’re around to see and be a part of.”
Balas says the approval was the right decision because ALS patients typically die within two to five years of diagnosis, and “right now there just aren’t a lot of drugs available.”
but dr david rindmedical director of the Institute for Clinical and Economic Review, isn’t so sure about Relyvrio, which will cost about $158,000 a year.
“I totally understand why people would be trying to find a way to get this out to patients,” he says. “There is a general concern that maybe the trial is wrong.”
ALS kills about 6,000 people a year in the US by gradually destroying nerve cells that control voluntary movements, such as walking, talking, eating, and even breathing. Relyvrio, a combination of two existing products, claims to slow down the disease process.
Advocates for the drug say the small trial proved it works. But FDA scientists and a Expert Panel FDA advises, they weren’t so sure.
FDA approval generally requires either two independent studies, each with hundreds of participants, showing effectiveness, or one large study with clearly positive results.
In March, the Central and Peripheral Nervous System Drugs Advisory Committee concluded that the Amylyx study did not provide “substantial evidence” that its drug was effective. Then in September, during a rare second meeting to consider a drug, the panel changed course and voted for approval.
The second vote came 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 deaths.
A much larger study of Relyvrio, the judgment of the phoenix, is running. But the results are more than a year away.
A negative result from that study would be a huge blow to Amylyx and ALS patients.
“If you have a drug that extends life by five months,” says Rind, “you should be able to show it in a larger trial.”
In the meantime, he says, perhaps Amylix should charge less for its 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. “This is not a cure,” she says. “We need to keep investing until we cure ALS.”
Klee and Cohen also promised that Amylyx will re-evaluate its drug based on the results of the Phoenix trial.
“If the Phoenix trial is not successful,” says Klee, “we will do what is right for patients, including voluntarily removing the drug from the market.”
But that decision would require the support of the company’s investors and its board of directors.