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FDA Approves First New ALS Drug in Years

After its success in Japan, the new treatment for amyotrophic lateral sclerosis be used for U.S. patients to improve their quality of life.


 

A new treatment for amyotrophic lateral sclerosis (ALS), Radicava (edaravone), is on the way for U.S. patients.

“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, MD, deputy director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “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.”

In a 6-month trial, 137 patients were randomly assigned to edaravone or placebo. At week 24, those receiving edaravone had declined less on a clinical assessment of daily functioning.

Edaravon is given intravenously daily for 14 days to start, followed by 14 days drug free. Subsequent treatment cycles consist of dosing on 10 of 14 days, followed by 14 days drug free.

The most common adverse reactions are bruising and gait disturbance. Edaravon also contains sodium bisulfite, which can cause serious allergic reactions in patients with sulfite sensitivity.

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