FDA Approves ALS Treatment

The FDA has approved edaravone (Radicava, Mitsubishi Tanabe Pharma America) for the treatment of amyotrophic lateral sclerosis (ALS), the first new ALS therapy approved by the FDA in several years.

Radicava is an intravenous infusion administered with an initial treatment cycle of daily dosing for 14 days, followed by a 14-day drug free period. Subsequent treatment cycles include dosing on 10 of those 14 days, followed by the 14-day drug free period.

The approval is based on a 6-month clinical trial conducted in Japan that demonstrated edavarone’s efficacy as a treatment for ALS. The trial included 137 participants who were randomly assigned to receive either edavarone or placebo. People who received edavarone showed less decline in daily functioning than those receiving a placebo.

The most common adverse effects include bruising and gait disturbance. The drug has also been associated with hives, swelling, shortness of breath, and allergic reactions to sodium bisulfite.

Reference

FDA approves drug to treat ALS [news release]. FDA’s website. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm557102.htm?source=govdelivery&utm_medium=email&utm_source=govdelivery. Accessed May 6, 2017.