MDA 2023: Walking, other functions retained longer on Radicava
Study analyzed 360 patients from large U.S. insurance database between 2017-2021
Amyotrophic lateral sclerosis (ALS) patients treated with Radicava (edaravone) tend to go longer before reaching disease milestones such as needing a walking aid or breathing support, according to an analysis of insurance data.
The findings were presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in the poster, “Longer Milestone-free Time in IV Edaravone-treated vs IV Edaravone-naïve Amyotrophic Lateral Sclerosis Patients: An Administrative Claims Analysis.” The work was funded by Mitsubishi Tanabe Pharma America (MTPA), which markets Radicava in the U.S.
Radicava is an approved treatment for ALS that’s thought to work by decreasing oxidative stress, a type of cellular damage that contributes to nerve cell death. It’s administered via infusion into the bloodstream, but an oral formulation called Radicava ORS has been approved in the U.S. MTPA recently announced that more than 10,000 people in the U.S. have received one of the two formulations.
A team of scientists at MTPA and other institutions analyzed data from a large U.S. insurance database between 2017-2021. The analysis only included patients with commercial insurance or Medicare Advantage plans, so it may not represent all ALS patients, the researchers noted.
The researchers examined 360 patients treated with Radicava for a median of 9.1 months and an equal number of patients who weren’t given Radicava. The two groups were matched based on available data so they were comparable for age, race, gender, and disease duration.
Comparing outcomes of Radicava treatment
To compare outcomes between them, the researchers calculated a statistical measure called restricted mean time lost (RMTL), which represents the average amount of time lost due to a certain milestone. For instance, a lower RMTL for death would suggest longer survival times. The researchers specifically assessed the first year after patients were entered into the database.
Results showed, for both commercially insured and Medicare Advantage patients, the RMTL for requiring a walking aid (such as a cane, walker, or wheelchair) was lower for those given Radicava, suggesting treated patients retained their ability to walk independently longer.
Radicava-treated patients also had lower RMTLs for noninvasive ventilation, using artificial nutrition, and hospice care. RMTLs were significantly higher among Radicava-treated patients for invasive ventilation or speech-generating devices, however.
The researchers also compared survival outcomes in the first and second year after patients were enrolled in the database. Results showed, for those who didn’t receive Radicava, mortality rates were 69.9% in the first year and 77.3% in the second. By contrast, mortality rates were 40.4% and 47% in the first and second years respectively for patients on Radicava.
These results “provide an example of real-world impact of [Radicava] in patients with ALS,” the scientists said, adding the findings “may be useful to payers and clinicians in evaluating the use of [Radicava].”
Both Radicava and Radicava ORS are administered in 28-day cycles of 14 days on treatment, followed by 14 days off. MTPA is currently running a Phase 3 clinical trial called MT-1186-A02 (NCT04569084) to compare alternative dosing regimes of Radicava ORS.
Results are expected later this year. Scientists at MTPA and other institutions shared details of the trial’s design at the MDA conference in the poster, “Phase 3b Study to Evaluate Efficacy and Safety of Oral Edaravone Over 48 Weeks in Patients with Amyotrophic Lateral Sclerosis (MT-1186-A02).”
The trial intends to enroll about 380 adults with ALS whose disease began within two years of enrollment. Participants will be randomly assigned to one of two groups and treated for about a year.
In one group, participants will be treated with Radicava ORS (105 mg) every day, rather than taking 14 days off treatment in each 28-day cycle. In the second group, participants will first get Radicava ORS for 14 days, then a placebo for 14 days (mimicking the currently approved schedule) for one 28-day-cycle. For the next 11 cycles, participants will take the therapy for 10 days and a placebo for 18 days.
The main goal is to compare outcomes on the ALS Functional Rating Scale Revised (ALSFRS-R), a standardized measure of ALS symptom severity. Secondary measures include changes in lung function, survival, and quality of life.
After completing the main trial, participants will be able to enter an open-label extension and receive Radicava ORS for an extended period.
“This study will provide important information on the safety, efficacy, and tolerability of 2 dosing regimens for [Radicava ORS] in patients with ALS,” the researchers concluded in their poster.
“These presentations reflect our determination to not only broaden our expertise in ALS, but to continue sharing key insights that have a meaningful impact on the patient and medical communities,” Gustavo A. Suarez Zambrano, MD, vice president of medical affairs at MTPA, said in a company press release.
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