FDA Approves Oral Version of Established ALS Drug Radicava

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The U.S. Food and Drug Administration has approved Mitsubishi Tanabe Pharma America's oral alternative to its own treatment for amyotrophic lateral sclerosis (ALS).

Radicava ORS (edaravone) offers a more convenient administration option for patients who cannot or would rather not take the intravenous formulation of Radicava. ORS can be taken orally or through a feeding tube, without having to make dose adjustments.

The product is not any different in composition and efficacy from the IV formulation, which already has FDA approval to help slow the loss of physical function in patients living with ALS, also called Lou Gehrig's disease.

Radicava ORS can be taken at home and is self-administered. Treatment starts with the patient fasting overnight first before the drug is given in the morning by mouth or via a tube. The initial treatment cycle is still daily for 14 days, followed by a 14-day period drug-free and then another cycle of daily dosing for 10 out of 14 days, followed again by a 14-day drug-free phase.

Edaravone was discovered and developed for ALS by MTPC and Mitsubishi Tanabe Pharma Development America in 2001. In 2015, Radicava was approved for ALS in South Korea and Japan. It then received marketing authorizations in Canada in 2018, Switzerland in 2019, China in 2019, Indonesia in 2020, Thailand in 2021 and Malaysia in 2021. In the U.S., Radicava has so far been used to treat more than 6,500 patients.

"At MTPA, patients have been the driving force behind our work as we strive to develop meaningful treatment options for ALS and continue to tackle unmet needs. Five years ago, we proudly launched Radicava as a treatment option for patients with ALS in the U.S. Now, we continue to push the boundaries of innovation with Radicava ORS, an orally administered option allowing patients flexibility in how they take their medicine," Atsushi Fujimoto, president of MTPA, said in a statement.

The FDA's decision is based on positive outcomes from a trial that involved 137 patients who were randomized to receive either Radicava ORS or a placebo. On the 24th week of the study, those who took Radicava saw a 33% decline in the loss of function compared to those who were given a placebo, as measured by the ALS Functional Rating Scale-Revised.

ALS is a rare condition that kills the nerve cells responsible for controlling voluntary muscles, such as chewing, talking, walking and breathing. Muscles weaken over time, leading to paralysis. Most cases result in death due to respiratory failure within three to five years from when the symptoms start to manifest. Around 16,000 Americans have ALS.

"ALS is a progressive disease that, due to its heterogeneous nature, impacts patients at different rates with varying symptoms. 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," Tulio Bertorini, MD, a professor of neurology at The University of Tennesse Health Science Center, commented in the same announcement.

To help make the drug more accessible to patients with ALS, MTPA launched the JourneyMate Support Program, which provides resources to help those living with the disease and their caregivers.