The US Food & Drug Administration (FDA) has approved the first new treatment for patients with amyotrophic lateral sclerosis (ALS) in 22 years. FDA approval for Radicava (edaravone) was granted following a six-month trial in Japan in which patients given the drug exhibited a slower rate of decline than those given a placebo. 

ALS, also known as Lou Gehrig's disease, is a relatively rare condition affecting up to 15,000 Americans. It is a progressive neurodegenerative disease affecting the nerve cells in the brain and spinal cord. 

Radicava (edaravone), brought to market by the Mitsubishi Tanabe Pharma Corporation, is an intravenous infusion administered by health care professionals. The course of treatment involves patients receiving doses for 14 days, followed by 14 days without. During subsequent treatment cycles, treatment involves daily infusions for ten days within a 14-day period, followed by another two weeks without the medication. The most common adverse reactions experienced by patients on Radicava (edaravone) were bruising and gait disturbance.

The ALS drug won approval under the FDA's orphan drug review program, which encourages pharmaceutical companies to pursues the development of drugs for rare disease by offering incentives, such as tax credits for qualified clinical testing. The last new treatment approved for ALS patients was Rilutek (riluzole). This benzothiazole, used to slow the progression of ALS, was brought to the market in 1995 by Rhone Poulenc Rorer, which is now part of Sanofi. It was released in a generic form in 2013.

Commenting on the approval of Radicava (edaravone), Eric Bastings, MD, deputy director of the division of neurology products in the FDA's Center for Drug Evaluation and Research, said: "[Radicava] 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 ALS Association welcomed the FDA's decision to approve the drug, noting there were other clinical trials in progress and that it was hopeful more therapies would soon be available. "We hope today's announcement signals the beginning of a new chapter in the fight against this terrible disease," said Barbara Newhouse, president and CEO of the organization. 

For more information on Radicava (edaravone), visit:

https://www.radicava.com/patient/