#AAN2022 – US Real-world Data Show Radicava Boosts Survival Rate

Marta Figueiredo, PhD avatar

by Marta Figueiredo, PhD |

Share this article:

Share article via email
PrimeC | ALS News Today | illustration of audience viewing study data

Treatment with Radicava (edaravone) for at least one year significantly reduces the risk of death in people with amyotrophic lateral sclerosis (ALS), prolonging their survival for about six months compared with those not given the therapy, an analysis of U.S. real-life data shows.

“Real-world data may provide additional insight into treatment for rare diseases like ALS, which can sometimes be challenging to research and evaluate in clinical trials,” Gustavo A. Suarez Zambrano, MD, vice president of medical affairs at Mitsubishi Tanabe Pharma America (MTPA), which commercializes Radicava in the U.S., said in a press release.

These findings were presented through an MTPA-funded poster at the 2022 American Academy of Neurology Annual Meeting, being held in Seattle, April 2–7, and virtually, April 24–26.

The poster was titled “Continued Intravenous Edaravone Treatment in Patients with Amyotrophic Lateral Sclerosis (ALS) Increases Overall Survival Compared With No Intravenous Edaravone Treatment: Results from a US Administrative Claims Database.”

Recommended Reading
locked-in ALS | ALS News Today | image of brain

Brain Implant Allows Paralyzed ALS Patient to ‘Speak’ With Family Again

Developed by Japan-based Mitsubishi Tanabe Pharma, Radicava works by lowering oxidative stress, a type of cellular damage thought to be one of the drivers of nerve cell death in ALS. Oxidative stress refers to an imbalance between the production of potentially harmful free radicals and the cells’ ability to detoxify them.

The therapy, administered directly into the bloodstream, was approved for ALS in Japan and South Korea in 2015 and in the U.S. in 2017. The decisions were based on findings from the Phase 3 Study 19 clinical trial (NCT01492686), which evaluated Radicava’s safety and effectiveness in 137 adults with ALS in Japan.

Participants were randomly assigned to receive Radicava or a placebo for 24 weeks (about six months), after which they could choose to receive the therapy for an additional 24 weeks in the study’s open-label extension portion.

Top-line, six-month results showed that Radicava-treated patients had a 33% significantly slower functional decline, as measured by changes in the ALS functional rating scale-revised scores, compared with those on a placebo.

Participants initially assigned to Radicava continued to experience slower disease progression in the extension portion, while those previously on placebo started to benefit from the therapy.

Further analysis showed that patients initially on Radicava in Study 19 had a 52% lower risk of death and a 56% lower cumulative risk of death, tracheostomy, and permanent assisted ventilation, compared with those starting on Radicava six months later — though these differences did not reach statistical significance. Tracheostomy is a surgical procedure that creates an opening in the windpipe for mechanical ventilation.

Given the limited data on Radicava’s benefits in the real-world setting, MTPA analyzed the survival rate of 318 ALS patients receiving the therapy for at least one year and 318 matched patients who were never prescribed Radicava (used as controls).

All patients were enrolled in Optum’s Clinformatics Data Mart, a real-world de-identified database of administrative health claims across the U.S. for people with commercial or Medicare Advantage health insurance plans.

The patients taking Radicava initiated the treatment between Aug. 8, 2017, and March 31, 2020.

Controls were matched for potential influencing factors, including age, sex, race, geographic region, disease duration, insurance, history of cardiovascular disease, riluzole prescription, feeding tube use, artificial nutrition, non-invasive ventilation, and all-cause hospitalization.

Riluzole is an approved ALS therapy sold as Rilutek, Tiglutik, or Exservan, and is also available in generic forms. In both groups, 208 patients (65.4%) had a history of riluzole prescription.

As of March 31, 2021, a lower proportion of patients on Radicava than controls had died from all causes (48.7% vs. 61.6%). This translated into a 27% lower risk of death, with patients on Radicava living a median of six months longer than those not treated with Radicava (median survival of 29.5 months vs. 23.5 months).

“Utilizing rigorous statistical methodology to assess nearly four years of administrative claims data, we found that patients with ALS receiving Radicava … survived longer than patients not treated with the drug in a real-world setting,” said Benjamin Rix Brooks, MD, an ALS specialist and the study’s first author.

Recommended Reading
ALS Association objections | ALS News Today | illustration of multiple thumbs down

Trial Data Not Enough for AMX0035 Approval, FDA Committee Votes

“Findings from this analysis, combined with previously reported pivotal Phase 3 trial data, add to the growing body of knowledge regarding the use of Radicava,” Zambrano added.

MTPA plans to continue assessing Radicava’s benefits in the real-world setting through additional research.

Based on positive interim findings from an international Phase 3 trial (NCT04165824), an oral formulation of Radicava called MT-1186 is now under review for approval by health authorities in Japan and in the U.S., where a decision is expected no later than May 12.

 

Note: The ALS News Today team is providing coverage of the American Academy of Neurology (AAN) 2022 Annual Meeting. Go here to read the latest stories from the conference.