FDA approves medication to treat Lou Gehrig’s Disease

The FDA approved Radicava for the treatment of ALS, the first new treatment approved “in many years,” according to an FDA director.

“After learning about the use of edaravone to treat ALS in Japan, we rapidly engaged with the drug developer about filing a marketing application in the United States,” Eric Bastings, MD, deputy director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said in a press release. “This is the first new treatment approved by the FDA for ALS in many years, and we are pleased that people with ALS will now have an additional option.”

Health care professionals can deliver Radicava (edaravone, Mitsubishi Tanabe Pharma Corporation) through an intravenous infusion, with an initial treatment cycle of 14 daily doses followed by a 2-week drug-free timeframe. This treatment repeats with dosing 10 out of 14 days and another 14-day drug-free period, according to the FDA.

A clinical trial in Japan including 137 participants treated with edaravone showed clinically less decline on assessment of daily functioning compared to those receiving placebo.

According to the press release, the most common adverse effects reported in the trial were bruising and gait disturbance. Additionally, some effects require immediate attention, such as hives, swelling, shortness of breath and allergic reaction to sodium bisulfite.

Disclosures: Bastings reports being an employee of the FDA.