Radicava ORS Shows Safety, Tolerability Over Nearly Year of Use

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Radicava ORS, an oral formulation of edaravone recently approved to treat amyotrophic lateral sclerosis (ALS) in the U.S., was safe and generally well-tolerated over almost a year of treatment in a Phase 3 clinical trial, new data highlight.

The results were presented by the therapy’s developer, Mitsubishi Tanabe Pharma America (MTPA), at the European Network to Cure ALS (ENCALS) Meeting 2022 held this month in Edinburgh, Scotland.

Radicava ORS, edaravone as an oral suspension, works to help reduce the oxidative stress that damages cells. A formulation of the molecule delivered via infusion into the bloodstream, marketed by MTPA as Radicava, has been approved in the U.S. since 2017 and in Canada since 2018.

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Radicava ORS is designed to offer the same efficacy as Radicava in a more convenient form. The oral version was approved by the U.S. Food and Drug Administration in May, supported in part by data from a Phase 3 clinical trial (NCT04165824).

That trial enrolled 185 adults with ALS whose symptoms began in the three years prior to enrollment, and who were given Radicava ORS for 48 weeks (nearly one year). Interim results, announced late last year after about six months of treatment, showed the therapy to be well-tolerated.

Data presented at ENCALS now show that edaravone in an oral formulation was generally well-tolerated over 48 weeks, MTPA announced in a press release. The most common adverse events were falls (22.2%), muscular weakness (21.1%), constipation (17.8%), shortness of breath (10.8%), trouble swallowing (10.3%), and back pain (10.3%).

No serious adverse events were deemed to be related to treatment with Radicava ORS. Sixteen patients (8.6%) discontinued the trial due to adverse events, for reasons mostly attributed to ALS progression. There were 12 deaths during the study, with none related to the use of Radicava ORS.

“I’m encouraged by the findings from the Phase 3 trial, which provide important information on the safety and tolerability of this new formulation of edaravone in patients with ALS. These data reinforce the potential significance of RADICAVA ORS and its important role in the treatment of this devastating disease,” said Gary Pattee, MD, a neurologist and ALS specialist in Nebraska.

During the trial, ALS severity was measured using the ALS Functional Rating Scale–Revised (ALSFRS-R), with lower scores indicating more severe symptoms. At the study’s start, the average ALSFRS-R score was 40 points, and over nearly a year on Radicava ORS, the average score decreased by 11.3 points. According to MTPA, the typical rate of decline in the ALSFRS-R is slightly more than 1 point per month.

MTPA is running an ongoing extension study (NCT04577404) where participants from the original trial can continue treatment with Radicava ORS for up to two years (96 weeks).

The company also recently announced that the specialty pharmacy Soleo Health will be one of four distributors of Radicava ORS in the U.S. MTPA and Soleo had previously partnered for distribution of the original formulation of Radicava.

“We look forward to continuing our partnership with Soleo Health, which is one of the specialty pharmacies that has the specific knowledge and experience to best support the ALS community,” Atsushi Fujimoto, president of MTPA, said in a press release.

“Soleo Health’s five-year partnership with MTPA for delivering infused Radicava … will continue with the successful introduction of Radicava ORS. Our team continues to demonstrate value in providing therapy and care for infused Radicava patients, which will afford those patients a smooth, seamless transition to Radicava ORS, should they and their prescriber make this treatment decision,” said Drew Walk, CEO of Soleo.

“We strongly believe in the potential of the oral formulation of edaravone to help people with ALS,” said Gustavo A. Suarez Zambrano, MD, vice president of medical affairs at MTPA.