The U.S. Food and Drug Administration (FDA) announced the approval of Relyvrio, an oral drug to treat amyotrophic lateral sclerosis, or ALS, Thursday. The drug, developed about a decade ago by CEOs of Amylyx Pharmaceuticals Josh Cohen and Justin Klee, gained approval despite concerns over whether the drug effectively slows the progression of the disease and helps patients live longer.
ALS, commonly referred to as Lou Gehrig’s disease, affects up to 30,000 people in the U.S., the majority being those over the age of 60. The fatal neurodegenerative disease compromises the nerve cells in the brain and spinal cord, leading to a progressive loss of muscle control that causes difficulty speaking, moving, breathing, and eating. The debilitating disease gained major attention following the viral ALS Ice Bucket Challenge—which raised $115 million for the ALS Association in 2014—and helped fund the development of Relyvrio.
Most people with ALS die within three to five years after diagnosis. The FDA cites the brutal progression of the disease as the reason for stamping its seal of support on the drug before the completion of the Phase 3 trial.
“There are limitations to these findings that result in a degree of residual uncertainty about the evidence of effectiveness that exceeds that which might typically remain following a conclusion that substantial evidence of effectiveness has been demonstrated,” a statement in the FDA approved summary reads. “However, given the serious and life-threatening nature of ALS and the substantial unmet need, this level of uncertainty is acceptable in this instance and consideration of these results in the context of regulatory flexibility is appropriate.”
The FDA’s approval resides largely on the drug’s Phase 2 trial. In March, an FDA advisory committee voted no on whether the trial data established a conclusion that the drug is effective in the treatment of ALS. However, CNN reports that Amylyx said it would potentially take the drug off the market if Phase 3 results failed and it was approved, a move that, along with “patient testimonials” and the previous results, led the FDA to approve the drug this week.
Phase 2, a randomized six-month trial, included 137 people who were diagnosed with ALS and progressing in the disease. Over two-thirds took Relyvrio and the others took a placebo; after 24 weeks, the group taking the drug saw a 25% decline in the progression of the disease (including trouble speaking, walking, breathing, and eating) compared to those on the placebo. In a more extended analysis, those who received the drug lived longer than those who took the placebo.
The drug may not work miracles for everyone, says Hande Ozdinler, associate professor of neurology at Northwestern University Feinberg School of Medicine.
“Patients develop the disease due to different underlying causes. Waiting for a drug that is going to cure everyone is like waiting for Godot. It is not going to come,” she says. “This drug will be very effective in some patients and maybe not so much effective in some, and that is okay. We have an obligation to those patients who are going to benefit tremendously from this drug.”
A number of ALS advocacy groups applauded the FDA’s approval.
“We still have a lot of work to do to cure ALS, but this new treatment is a significant step in that fight,” says Calaneet Balas, president and CEO of the ALS Association, in a statement Thursday.
A group of physicians who work with ALS patients penned a letter to the FDA director in May, pushing for the drug’s approval. The drug is “safe, well-tolerated, and provides a clinically meaningful benefit,” they wrote.
ALS’s devastating effects on human lives persists, leaving doctors who treat those diagnosed largely at a loss when it comes to treatment. For them, Relyvrio could provide patients even a slight bit of relief.
“While these effects may seem incremental, they are meaningful to us as ALS specialists and to our patients. It can mean a difference between walking up the stairs or being restricted to a floor of your home,” the physicians write. “It can mean being able to feed yourself versus needing help to just cut your food. This means more time for our patients with the people they love.”
The side effects of the drug include diarrhea, abdominal pain, nausea, and upper respiratory tract infection, according to Amylyx’s press release Thursday, citing the need to speak with your doctor before considering using the drug to check drug interactions with other medications you may be taking.
Doctors will be able to prescribe the drug immediately and patients should receive access to it in four to six weeks, the company says. Phase 3 study results are expected in early 2024.
Editor’s note: This story has been updated to amend some earlier factual errors
