Nearly 2,000 ALS Patients in US Being Treated With Radicava ORS

Edaravone as an oral suspension seen as equally effective as IV therapy

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by Mary Chapman |

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Nearly 2,000 amyotrophic lateral sclerosis (ALS) patients in the United States have been treated with Radicava ORS (edaravone) since the oral suspension formulation became available there in May, the therapy’s U.S. marketer, Mitsubishi Tanabe Pharma America (MTPA), announced.

This formulation is seen to be as equally effective as Radicava, an intravenous (into-the-vein) treatment that works to slow functional decline in people with the progressive neurodegenerative disorder. With its initial 2017 approval, Radicava became the first new ALS treatment available in the U.S. in 22 years.

Together, the therapies have been used to treat some 8,000 people across the country, with more than 1.1 million days of therapy logged, according to MTPA. Nearly 2,000 U.S. healthcare providers have prescribed the treatments to at least one patient.

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“Our driving focus at MTPA is to ensure we’re making a meaningful difference in the lives of people living with ALS, and this milestone exemplifies our commitment to this goal,” Atsushi Fujimoto, MTPA’s president, said in a company press release.

“We are pleased by the amount of ALS patients that have received Radicava ORS over the past few months, including new patients as well as those who have switched from Radicava, and we look forward to seeing this number rise as more patients gain access to this new oral treatment option,” Fujimoto added.

Radicava as oral suspension can be taken by mouth or feeding tube

The oral therapy, offering patients greater convenience, can be self-administered at home orally or via a feeding tube (5 ml/105 mg). Its bottle packaging includes an oral syringe for precise dosing. The medication needs no refrigeration or prior reconstitution, and it should be taken in the morning after overnight fasting for eight hours with only water consumed for one hour after its administration.

The dosing regimen is the same as for Radicava: daily dosing for 14 days initially, followed by 14 days of no treatment. After that, treatment entails daily dosing for 10 of every 14 days, followed by 14-day treatment-free periods.

Edaravone, the active ingredient in Radicava and Radicava ORS, works by mitigating oxidative stress, a type cellular damage that contributes to nerve cell death in ALS.

Radicava’s initial approval was based on data from a Phase 3 study, called Study 19 (NCT01492686), in which six months of treatment was seen to slow patients’ functional decline by 33%, a significant difference compared to patients randomized to a placebo. Decline was determined using an established scale measuring patients’ abilities in such daily activities as speaking, swallowing, and dressing.

These data also were used to support the oral formulation’s approval in May, along with pharmacological findings demonstrating that Radicava ORS delivers equitable amounts of the active medication to the body.

Radicava ORS’s approval was also based on findings in the global, open-label Phase 3 study (NCT04165824) that evaluated its safety and tolerability in 185 adults whose ALS symptoms started within three years of trial enrollment.

According to interim trial results, six months of treatment were generally safe and well tolerated, and the medication’s use over those months indicated it could slow functional decline to a degree similar to that of Radicava in Study 19.

The most commonly reported adverse events in the Radicava ORS study were muscular weakness (16.2%) and falls (15.7%).

MTPA runs a support program called JourneyMate, providing information and educational resources to patients prescribed Radicava or Radicava ORS and their caregivers.

“ALS affects patients in varying ways, which means their treatment needs are continuously evolving,” said Terry Heiman-Patterson, MD, director of the MDA ALS Center of Hope. “Radicava ORS offers patients the flexibility of an alternate delivery option, and I am eager to continue to see this medication reach more people in the coming years.”