MT Pharma Gains FDA Approval for ALS Drug - Drug Discovery and Development

The Food and Drug Administration (FDA) on Friday granted approval to Radicava (edaravone), the second-ever drug approved for amyotrophic lateral sclerosis (ALS).

Japanese pharmaceutical firm Mitsubishi Tanabe Pharma America, Inc.(MT Pharma) developed the drug, which is administered as an intravenous infusion beginning with a 14-day daily dosing cycle followed by another 14-day period of being drug-free. Additional treatment cycles entailed dosing regimens on 10 of 14 days followed by another 14-day drug free period.

The FDA made their decision after reviewing trial data analyzing the drug’s efficacy, according to the official announcement. The FDA granted Radicava orphan drug status.

Investigators performed a randomized six-month clinical trial in Japan where 137 patients were either given Radicava or placebo. Results after 24 weeks indicated patients with ALS declined less on Radicava versus placebo based on a clinical assessment of daily functioning. There was no information given regarding survival rates.

“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 Eric Bastings, M.D., the deputy director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, in a statement. 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.”

The first officially approved drug for ALS was riluzole, which could increase survival rates by a few months.

Common adverse events reported during the trial were bruising and gait disturbance, but the drug was also associated with serious health risks that required serious medical attention like hives, swelling, shortness of breath, and allergic reactions to the sodium bisulfite ingredient.

MT Pharma said each infusion of Radicava would cost $1,086, reported Reuters.

Patients taking Radicava for 12 months according to the dosing and administration label could see costs, before the government discount, reach $145,524.