The United States Food and Drug Administration (FDA) has approved a drug to slow the progression of Amyotrophic Lateral Sclerosis (ALS) and potentially delay death caused by the disease.
The medication, which will be marketed under the brand name Relyvrio and belongs to Amylyx Pharmaceuticals, consists of a combination of the generic compounds sodium phenylbutyrate and taurursodiol, which act together to prevent nerve cells in the brain and spinal cord from dying prematurely.
“This approval provides another important treatment option for ALS, a life-threatening disease that currently has no cure,” said Dr. Billy Dunn, director of the Office of Neurosciences at the regulatory agency’s Center for Drug Evaluation and Research. “The FDA remains committed to facilitating the development of additional treatments for ALS,” he added.
ALS is a rare disease that attacks and kills the nerve cells that control the voluntary muscles associated with chewing, walking, breathing and speaking. It is progressive and worsens over time.
It causes the nerves to lose the ability to activate specific muscles, causing them to weaken and leading to paralysis. Most cases cause death from respiratory failure, usually within three to five years after the first symptoms appear. About 5,000 people in the U.S. are diagnosed with ALS each year, and about 20,000 Americans live with the disease.
Relyvrio can be taken by mouth or given through a tube. The recommended dose for the first three weeks is one packet (3 grams of sodium phenylbutyrate and 1 gram of taurursodiol) per day. After three weeks, the dosage increases to one packet twice a day. It is recommended to eat it before a snack or meal.
According to a statement from the FDA, the effectiveness of Relyvrio for the treatment of ALS was demonstrated in a 24-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group study.
In the trial, 137 adult ALS patients were assigned to receive Relyvrio or placebo. Patients treated with Relyvrio experienced a slower rate of decline in daily functioning compared to those receiving a placebo.
Additionally, longer overall survival was observed in a long-term post hoc analysis of patients who originally received Relyvrio versus those who originally received placebo.
The most common adverse reactions experienced with Relyvrio were diarrhea, abdominal pain, nausea, and upper respiratory tract infection.
As the medication contains taurursodiol, a bile acid that can worsen diarrhea in patients with disorders that interfere with the circulation of bile acids, they should consult with a specialist before taking it.
The FDA granted this application Priority Review designation. It also received orphan drug designation, which provides incentives to aid and encourage the development of drugs for rare diseases.
"I think we’re all aware that this drug is not the magic treatment that will cure the disease, but will hopefully slow its progression," Dr. Jinsy Andrews, an associate professor of neurology at the College of Medicine, told Reuters. Columbia University Vagelos Physicians and Surgeons, prior to approval.
Andrews, an investigator in the late-stage Amylyx trial, added that the ALS community agrees that better treatments be developed in the future.
The FDA’s decision also comes after months of complaints from patients, who urged the regulatory body to approve the drug based on its relative safety and its potential to increase survival.
