FDA Approves First New ALS Drug in Two Decades

Mitsubishi Tanabe Pharma’s drug Radicava (edaravone), has been approved by the FDA to treat patients with amyotrophic lateral sclerosis (ALS). The intravenous infusion treatment is the first to be approved to treat the rare disease in over 20 years.

ALS is a progressive neurodegenerative disease characterized by death of nerve cells controlling voluntary muscles. In a Phase III placebo-controlled clinical trial assessing the safety and efficacy of Radicava, the investigators found that patients taking the drug showed less decline in physical function, as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R).

“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,” said Dr. Eric Bastings, deputy director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “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.”

Data from the MCI186-19 Phase III clinical trial was used to support the FDA’s approval of Radicava. In all, 137 individuals with ALS were enrolled in the study and randomized to receive 60 mg of intravenous Radicava or a placebo, for a six month period.

Patients taking Radicava showed a significantly less decline in physical function of 33 percent, or 2.49 points on the ALSFRS-R. The scale is a validated measure of disability progression in patients diagnosed with ALS.

While Radicava was developed by Japanese pharmaceutical company, Mitsubishi Tanabe Pharma Corporation, it will be commercialized in the US by MT Pharma America. The drug has been approved to treat ALS patients in Japan and South Korea since 2015.

“We believe Radicava offers new hope for people with ALS and exemplifies MT Pharma America’s commitment to innovative therapies for patients in the United States battling life-threatening diseases,” said Atsushi Fujimoto, President, MT Pharma America. “We recognize how important this therapy may be to people with ALS and are committed to helping provide access to this important treatment option, with the goal of keeping out-of-pocket costs at a minimum for eligible patients.”

Between 5,000 and 6,000 Americans are diagnosed with ALS each year, with full diagnosis often taking over a year to confirm. The disease rapidly progresses over time and most patients die within five years of initial diagnosis.

“For people with ALS and their families, having a new therapy which slows the decline of physical ability is incredibly significant,” said Dr. Jonathan S. Katz, ALS Clinic Director, Forbes Norris MDA/ALS Research and Treatment Center at California Pacific Medical Center. “The approval of Radicava brings us into a new era of treatment by evolving how we manage this complex disease. This is an uplifting milestone for the ALS community especially since it’s been so long since we had anything new.”