FDA Approves Oral Form of ALS Medication

The FDA approved edaravone oral suspension for the treatment of adults with amyotrophic lateral sclerosis (ALS).

The same drug was initially approved in 2017 as an intravenous infusion to treat ALS, which is commonly known as Lou Gehrig’s disease.

This new oral form of edaravone is self-administered and can be taken at home. After an individual fasts overnight, edaravone should be taken in the morning orally or through a feeding tube. The oral medication has the same dosing regimen as the IV form: an initial cycle of daily dosing for 14 days, followed by a 14-day drug-free period and subsequent treatment cycles consisting of daily dosing for 10 out of 14-day periods, followed by 14-day drug-free periods.

This approval was based on results from a trial demonstrating that patients with ALS given oral edaravone showed similar levels of the drug in their bloodstream compared to the IV formulation; the drug’s initial approval was based on a 137-patient trial in which individuals receiving edaravone experienced less functional decline versus patients given placebo.

ALS is a rare disease, with an estimated prevalence of 5.0 per 100,000 Americans as of 2014, according to the Centers for Disease Control and Prevention. Besides those with a family history of ALS, individuals who are male, White, non-Hispanic, and over age 60 are more likely to have ALS.

ALS attacks and kills the nerve cells that control voluntary muscles, such as those needed for chewing, walking, breathing, and speaking. These nerves lose the ability to activate specific muscles, which causes those muscles to become weak, leading to paralysis.

ALS becomes progressively more severe over time. Most patients will die from respiratory failure, usually within three to five years of initial symptom onset.

Edaravone’s most common side effects are bruising, problems walking, headaches, and possibly fatigue. Serious side effects of either form include allergic reactions producing hives, rash, and shortness of breath. The FDA cautions that sodium bisulfite in both the oral and IV formulations could cause a life-threatening allergic reaction in patients with sulfite sensitivity.

The prescribing information includes additional information.

FDA granted oral edaravone orphan drug approval, priority review, and fast track designations.

Oral edaravone is marketed by Mitsubishi Tanabe Pharma America Inc. as Radicava ORS.