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

The Food and Drug Administration has approved a new drug for a fatal disease.

Patients and their advocates are applauding the decision, but some scientists are questioning it.

A single study of 137 patients was used to approve Relyvrio. The results suggested the drug could prolong patients' lives.

Calaneet Balas, president and CEO of theALS Association, says that six months can be anything from a daughter's graduation to the birth of a child. These are big, monumental things that many people want to be a part of.

There aren't a lot of drugs available right now so approval was the right decision.

The Institute for Clinical and Economic Review's chief medical officer isn't so sure about Relyvrio.

He understands why people are trying to find a way to get this to patients. There is a general concern that the trial is flawed.

In the US, about 6,000 people a year are killed by amyotrophic lateral sclerosis, also known as Lou Gehrig's disease. The goal is to slow down the disease process.

The small trial proved that the drug works. FDA scientists and an expert panel were unsure.

One large study with clearly positive results is usually required for FDA approval.

The committee concluded that the Amylyx study didn't provide enough evidence that the drug was effective. The panel voted in favor of approval during a rare second meeting in September.

The FDA's Office of Neuroscience director encouraged the committee to be flexible when considering a drug that might help people facing certain death.

There is a large study of Relyvrio. Results are off for a long time.

A negative result from that study would hurt patients.

Rind says you should be able to show that the drug prolongs life by five months in a larger trial.

He thinks Amylix could charge less for their drug.

The only product made by Amylyx is Relyvrio, which is marketed as Albrioza inCanada.

Klee says the price will allow the company to develop better treatments. He says this isn't a cure. We need to invest until we cure the disease.

According to Klee and Cohen, Amylyx will re-evaluate its drug after the Phoenix trial.

Klee says they will take the drug off the market if the trial doesn't go well.

The board of directors would have to support the decision.