FDA approves edaravone for amyotrophic lateral sclerosis.

Abstract

FDA on May 5 announced the approval of edaravone injection for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. ALS is a rapidly progressive, degenerative neurologic disorder that affects the control of motor neurons and results in the atrophy of muscles, including those involved in breathing. About 5,000 people in the United States are diagnosed with the disease annually, and most die from respiratory failure within 3–5 years of diagnosis, according to federal estimates. Edaravone, a member of the 2-pyrazolin-5-one family, is the first new therapy with an FDA-approved indication for ALS since 1995, when the agency approved riluzole, an oral drug product. FDA approved edaravone on the basis of data from a 6-month placebo-controlled clinical trial involving 137 patients in Japan. In that study, the declines in motor, speech, swallowing, and respiratory function were significantly less severe in patients treated with edaravone than in placebo recipients.