Mitsubishi Tanabe Wins FDA Approval for Radicava, the First New ALS Drug in 22 Years

May 8, 2017
By Alex Keown, BioSpace.com Breaking News Staff

SILVER SPRING, Md. – Mitsubishi Tanabe and its new subsidiary, MT Pharma America, is celebrating after the U.S. Food and Drug Administration approved its amyotrophic lateral sclerosis (ALS) drug Radicava (edaravone), on Friday.

The approval marks the first time a new ALS treatment was approved in 22 years. The approval caused Mitsubishi Tanabe share prices to skyrocket on the Tokyo Stock Exchange, climbing to 2,586 Yen on Friday. Shares are up nearly 10 percent this morning. According to the ALS Association, Radicava will be priced at $1,000 per dosage, which is about $146,000 annually. It is expected to be available for patients by August.

Atsushi Fujimoto, president of MT Pharma America, said Radicava offers new hope for people with ALS.

“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,” Fujimoto said in a statement.

U.S. regulators encouraged the company to seek approval for the drug after learning about its successes in treating ALS in Japan and South Korea. Radicava was granted orphan drug designation. Eric Bastings, deputy director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research, said ALS patients now have an additional treatment option. Sanofi’s riluzole is the other approved ALS treatment in the United States.

Radicava, like Riluzole, is not a cure for ALS, but slows decline in the daily functioning of ALS patients. The drug was approved based on Phase III clinical trials that demonstrated patients taking the drug had a slower decline in physical function than those taking placebo. Data demonstrated patients who received Radicava for six months experienced significantly less decline in physical function—by 33 percent. Despite the slowing of the disease’s progress, patients with ALS typically die within two to five years of diagnosis from respiratory failure.

Also called Lou Gehrig’s disease, ALS is a rare disease that attacks and kills the nerve cells that control voluntary muscles. Voluntary muscles produce movements such as chewing, walking, breathing and talking. The nerves lose the ability to activate specific muscles, which causes the muscles to become weak and leads to paralysis. The Centers for Disease Control and Prevention estimates that approximately 12,000 to 15,000 Americans have ALS, with about 5,000 to 6,000 diagnosed annually.

Radicava will be provided to patients intravenously. It takes about 60 minutes for each dosing. Patients will be dosed for 14 days, which will then be followed by a 14-day drug-free period. Patients will undergo subsequent dosings of 10 of 14 days, followed by another 14 days of drug-free time.

The drug does have some risks. Risks for taking Radicava include hives, swelling, or shortness of breath, and allergic reactions to sodium bisulfite, an ingredient in the drug. Sodium bisulfite may cause anaphylactic symptoms that can be life-threatening in people with sulfite sensitivity. The most common adverse reactions that occurred in greater than 10 percent of patients and greater than placebo were bruising, problems walking and headache.

Other companies are also working on ALS treatments, including Boston-based Biogen .