FDA Approves Oral Form of ALS Therapy

The FDA has approved Radicava ORS (edaravone) oral suspension for the treatment of adults with amyotrophic lateral sclerosis (ALS). Radicava ORS, developed by Mitsubishi Tanabe Pharma America, is an orally administered version of Radicava, which was originally approved in 2017 as an intravenous (IV) infusion to treat ALS.

Tulio Bertorini, M.D.

"ALS is a progressive disease that, due to its heterogenous nature, impacts patients at different rates with varying symptoms," Tulio Bertorini, M.D., Professor of Neurology, The University of Tennessee Health Science Center, said in a press release. "Therefore, it is crucial that patients have treatment and formulation options that accommodate their own unique needs, and RADICAVA ORS provides HCPs who have prescribed their ALS patients edaravone with an alternate delivery option.

Radicava ORS can be taken orally or administered through a feeding tube. The oral medication has the same dosing regimen as Radicava—an initial treatment 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.

The effectiveness of Radicava ORS is based on a study that showed comparable levels of Radicava ORS in the bloodstream to the levels from the IV formulation of the therapy. The efficacy of Radicava for the treatment of ALS was previously demonstrated in a six-month clinical trial that served as the basis for approval in 2017. In that trial, 137 participants were randomized to receive Radicava or placebo. At Week 24, individuals receiving Radicava declined less on a clinical assessment of daily functioning compared to those receiving placebo.

ALS is a rare disease that attacks and kills the nerve cells that control voluntary muscles. Voluntary muscles produce movements such as chewing, walking, breathing, and talking. ALS causes the nerves to lose the ability to activate specific muscles, which causes the muscles to become weak and leads to paralysis. ALS is a progressive disease that continues to get worse over time.