Radicava Oral Suspension Earns FDA Approval

Marta Figueiredo, PhD avatar

by Marta Figueiredo, PhD |

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The U.S. Food and Drug Administration (FDA) has approved Mitsubishi Tanabe Pharma America (MTPA)’s oral suspension formulation of edaravone, Radicava ORS, for the treatment of amyotrophic lateral sclerosis (ALS).

The oral therapy is designed to offer the same efficacy as MTPA’s Radicava (edaravone) — an FDA-approved, into-the-vein therapy shown to slow ALS progression — while being more convenient and less burdensome for patients.

Radicava ORS can be self-administered at home and should be taken orally or via feeding tube (5 mL/105 mg) in the morning after overnight fasting. Patients should not consume food (except water) for one hour after administration.

The medication comes in a portable bottle, includes a 5 mL oral syringe for accurate dosing, and does not need refrigeration or reconstitution before administration.

Radicava ORS has the same dosing regimen as Radicava: an initial treatment cycle of daily dosing for 14 days, followed by a 14-day treatment-free period, and subsequent treatment cycles consisting of daily dosing for 10 out of 14-day periods, followed by 14-day treatment-free periods.

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“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,” Atsushi Fujimoto, MTPA’s president, said in a press release.

“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,” Fujimoto added.

“ALS is a progressive disease that, due to its [variable] nature, impacts patients at different rates with varying symptoms,” said Tulio Bertorini, MD, a professor of neurology at the University of Tennessee Health Science Center in Memphis.

“Therefore, it is crucial that patients have treatment and formulation options that accommodate their own unique needs, and Radicava ORS provides [healthcare providers] who have prescribed their ALS patients edaravone with an alternate delivery option,” Bertorini added.

The FDA’s decision comes about four months after the agency granted priority review to MTPA’s application, shortening the review process to up to six months, as opposed to the standard 10 months.

Similar applications are being reviewed by health authorities in Japan — where edaravone’s into-the-vein formulation is sold as Radicut — and, more recently, in Switzerland.

Developed by Japan-based Mitsubishi Tanabe Pharma, edaravone works by reducing oxidative stress, a form of cellular damage that is considered one of the drivers of nerve cell death in ALS.

The into-the-vein formulation, administered through hour-long infusions, was approved for ALS in Japan and South Korea in 2015, in the U.S. in 2017, in Canada the year after, and in Switzerland in 2019. It is not available in the rest of Europe.

Regulatory approvals were based on data from the Phase 3 Study 19 clinical trial (NCT01492686), which showed that six months of Radicava treatment slowed ALS patients’ functional decline by 33% compared with a placebo.

The approval of Radicava ORS was supported by Radicava’s efficacy data and pharmacological findings demonstrating that the oral formulation delivers similar amounts of the active medication to the body.

It also was based on data from an MTPA-sponsored, worldwide Phase 3 trial (NCT04165824) that assessed Radicava ORS’s safety and tolerability in 185 adults with ALS whose symptoms began within three years of enrollment. Participants received 105 mg of the therapy in the same dosing regimen as Radicava for 48 weeks (nearly one year).

Interim results showed that six months of treatment were generally safe and well-tolerated. Exploratory efficacy data also suggested that the oral formulation may slow functional decline to a degree comparable to that seen with Radicava in Study 19.

Those who completed the trial could enter an open-label extension study (NCT04577404), where they are receiving the therapy for 96 weeks (nearly two years). The main goal is to evaluate its long-term safety and tolerability, but exploratory efficacy measures also will be assessed.

Also, two dosing regimens of Radicava ORS are being tested against a placebo in a Phase 3b trial (NCT04569084) that is recruiting up to 380 adults with ALS at 95 locations in the U.S., Canada, Europe, Japan, and South Korea. Participants must have developed ALS symptoms in the two years prior to enrollment.

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The trial’s main goal is assessing changes in ALS Functional Rating Scale Revised scores, a measure of functional disability. Secondary goals include changes in lung function and overall health, as well as the time to death, tracheostomy, or ventilatory support. Tracheostomy is a procedure in which a tube is inserted into the neck to help with breathing.

The study is expected to end in July 2023.

The most commonly reported adverse side effects with Radicava include bruising, walking problems, and headache. Fatigue was reported by 7.6% of patients taking Radicava ORS.

People with a known allergy to edaravone or any of the inactive ingredients in Radicava and/or Radicava ORS should not be given these medications. According to edaravone’s label, patients should notify their healthcare provider if they become pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed.

As a part of MTPA’s commitment to helping patients with ALS access Radicava and Radicava ORS, the company created the JourneyMate Support Program, which provides resources to help patients and caregivers on their ALS treatment journey. It also provides information about insurance coverage and financial support programs for patients prescribed Radicava ORS.

“To address the unmet needs in ALS, we must continue to find new treatments, as well as optimize current treatments and care,” said Calaneet Balas, president and CEO of The ALS Association.

“We thank MTPA and the FDA for working diligently to make this oral formulation possible and are confident it will be a welcome addition to help further support the ALS community,” Balas added.