The US Food and Drug Administration (FDA) approved the drug to treat a rare neurological disease, amyotrophic lateral sclerosis (ALS). The development of the drug was partly funded by the Ice Bucket Challenge in 2014.
The drug Relvyrio, also known as AMX0035, was developed by Amylyx Pharmaceuticals in Massachusetts. The drug is an oral combination of sodium phenylbutyrate and taurursodiol.
The FDA relied on a single phase 2 clinical trial that included 137 adult patients with ALS to approve the drug. During the 24-month trial, participants received either Relyvrio or a placebo. The patients treated with Relyvrio experienced a slower decline in daily functioning compared to those receiving a placebo. Moreover, the drug improved survival in ALS patients.
The development of Relyvrio was funded by the money collected during ALS Ice Bucket Challenge. In 2014, millions of social media users posted videos in which they pledged to donate money to the ALS Association and poured a bucket of ice-cold water on themselves. The challenge raised $2.2 million.
The ALS Association, a patient advocacy group, praised the FDA approval, saying that this new treatment is "a significant step" in the fight with ALS, but there is still "a lot of work to do" to cure the disease.
In March, the FDA Peripheral and Central Nervous System Drugs Advisory Committee voted 6-4 to recommend against approval of Relvyrio, saying there was not enough data to prove that the drug was effective. Nevertheless, due to the submission of additional analyses and advocacy, the Committee voted 7-2 in favor of approval in September.
Dr. Jinsy Andrews, associate professor of neurology and Director of Neuromuscular Clinical Trials at Columbia University Irving Medical Center, says that the data from the phase 2 clinical trial is sufficient to allow access to people living with ALS who have limited options for treatment.
She explains that in the trial, taking Relyvrio resulted in a statistically significant reduction in the loss of physical function and had a positive impact on survival. Moreover, the trial suggests that starting the drug as early as possible may provide better benefits.
“These data, along with a favorable safety and tolerability profile, provide sufficient support to allow access while the confirmatory trial is ongoing,” Andrews said to Healthnews.
The Institute for Clinical and Economic Review (ICER), a non-profit organization evaluating the cost of medicines, recommended the Relyvrio price for $9,100 – $30,600 per year. Amylyx Pharmaceuticals, however, set the price at $158,000 for a year’s supply but said ALS patients would not need to pay the full price.
Andrews says she sees Relyvrio as a medication that can be used in combination with already approved treatments for ALS.
“For a serious life-threatening condition such as ALS, having treatments that can slow the loss of functional decline and increase survival is valuable to those living with ALS,” she said.
What is ALS?
ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, according to the ALS Association.
The disease affects the motor neurons, most of which reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. ALS causes these neurons to degenerate over time until they die. When it happens, the brain can no longer initiate and control muscle movement, and people may lose the ability to speak, eat, move, and breathe.
According to the Centers for Disease Control and Prevention (CDC), ALS is age-related and is usually diagnosed between 55 and 75 years of age. The disease is slightly more common among women than men. ALS patients typically live between two to five years after symptoms develop, but some people live ten years or even longer.
Approximately 5,000 people in the US are diagnosed with ALS annually, and about 20,000 Americans are currently living with the disease, the FDA says.
The New England Journal of Medicine. Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis.
ALS Association. Understanding ALS.
Amylyx Pharmaceuticals. Amylyx Pharmaceuticals Announces FDA Approval of RELYVRIO™ for the Treatment of ALS.
ALS Association. FDA Approves First ALS Treatment Funded by Ice Bucket Challenge.
Institute for Clinical and Economic Review. ICER Publishes Evidence Report on Treatments for Amyotrophic Lateral Sclerosis.