Placed on a fast track for evaluation by the Food and Drug administration, the free radical scavenger edaravone has been approved for the treatment of amyotrophic lateral sclerosis (ALS) in the United States. By tamping down oxidative stress, edaravone reportedly slows functional decline for patients in early stages of disease. It is the first treatment for ALS to be approved since riluzole in 1995. Edaravone requires intravenous administration, and will be marketed by the New Jersey–based company MT Pharma America under the name Radicava.
Also known as MCI-186, edaravone was originally developed by the Mitsubishi Tanabe Pharma Corporation in Osaka, Japan. A pivotal Phase 3 trial in 2014 led to approval in Japan in 2015, followed by South Korea. In 137 patients with mild disease, edaravone slowed functional decline over six months compared to placebo (see Jan 2016 news).
Treatment consists of daily one-hour injections for two weeks in the first month, followed by 10 injections spread over a 14-day period in subsequent months. Serious risks include hives, swelling, and shortness of breath. The ingredient sodium bisulfite can cause anaphylactic symptoms in some patients. Common side effects were bruising, problems walking, and headaches.
According to the company, edaravone will be available for distribution to U.S. ALS centers in August of 2017. The company says it will cost almost $146,000 each year, compared with a $35,000 price tag in Japan. A company representative said the added cost reflects investments in infrastructure and research and development the company has made in order to gain approval and market the drug in the United States.—Gwyneth Dickey Zakaib
- Sawada H. Clinical efficacy of edaravone for the treatment of amyotrophic lateral sclerosis. Expert Opin Pharmacother. 2017 May;18(7):735-738. PubMed.
- Martinez A, Palomo Ruiz MD, Perez DI, Gil C. Drugs in clinical development for the treatment of amyotrophic lateral sclerosis. Expert Opin Investig Drugs. 2017 Apr;26(4):403-414. Epub 2017 Mar 14 PubMed.