FDA Approves New Medication for Treatment of ALS
The FDA has approved sodium phenylbutyrate/taurursodiol (Relyvrio) as a treatment option for patients diagnosed with amyotrophic lateral sclerosis (ALS).
The approval of sodium phenylbutyrate/taurursodiol follows a 24-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group study that demonstrated the efficacy of the treatment. During the trial, 137 adult patients with ALS were randomized to either receive the treatment or a placebo. The patients who received sodium phenylbutyrate/taurursodiol showed a slower rate of decline on a clinical assessment of daily functioning when compared to individuals receiving a placebo.
Further, patients who received treatment had a longer overall survival compared to patients who received a placebo.
Sodium phenylbutyrate/taurursodiol is medication that can be taken orally by combining 1 packet in 8 ounces of room temperature water. Additionally, the medication can be administered through a feeding tube. The medication has a recommended dosage of 1 packet (3 grams sodium phenylbutyrate and 1-gram taurursodiol) for the first 3 weeks daily. Physicians will increase the dosage by 1 packet twice a day after the initial 3 weeks.
Common adverse effects include diarrhea, abdominal pain, nausea, and upper respiratory tract infection. Taurursodiol is a bile acid that may cause worsening diarrhea in patients with disorders that interfere with the circulation of bile acid.
“This approval provides another important treatment option for ALS, a life-threatening disease that currently has no cure,” Billy Dunn, MD, director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research said in a press release. “The FDA remains committed to facilitating the development of additional ALS treatments.”
FDA approves new treatment option for patients with ALS. News release. US Food and Drug Administration; September 29, 2022. Accessed October 7, 2022. https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-option-patients-als