Radicava Prolongs Survival, Small ALS Study Shows
These data add to increasing, but still limited, evidence showing the therapy prolongs the life of ALS patients and reduces the risk of death, further highlighting Radicava’s therapeutic potential in ALS.
Larger studies are needed to confirm the therapy’s survival benefits in this patient population, the researchers noted.
The study, “Improved Long-Term Survival with Edaravone Therapy in Patients with Amyotrophic Lateral Sclerosis: A Retrospective Single-Center Study in Japan,” was published in the journal Pharmaceuticals.
Developed by Mitsubishi Tanabe Pharma, Radicava works by reducing oxidative stress, which is thought to be one of the drivers of nerve cell death in ALS. Oxidative stress is an imbalance between the production of potentially harmful free radicals and a cell’s ability to detoxify them.
The therapy was approved for ALS in Japan and South Korea in 2015 and in the U.S. in 2017, based on findings from the Phase 3 Study 19 clinical trial (NCT01492686), which showed that Radicava effectively slowed disease progression and functional decline by 33%, compared with a placebo.
However, there is limited long-term, real-world data demonstrating Radicava’s safety and effectiveness, and only one prior study reported its effect on survival. While that study showed Radicava-treated patients lived about two times longer than those not receiving the therapy (a median of 61 vs. 32.5 months), mean treatment duration was very short (8.8 months).
Now, researchers in Japan evaluated the long-term survival benefits of Radicava in 45 ALS patients (28 men and 17 women) who first visited their hospital from 2013 to 2018 and were followed-up until December 2020.
A total of 22 patients were treated with Radicava for a mean duration of 26.6 months, or little more than two years (range, 2–64 months), while 23 did not receive the therapy and were used as the control group.
There were no significant group differences in terms of demographic and clinical characteristics. Riluzole, an ALS-approved therapy marketed as Rilutek, Tiglutik, and Exservan, was taken by most patients in both groups (81.8% in the Radicava group and 60.9% in the control group). These differences also were not statistically significant.
The main goal of the study was to assess whether Radicava extended the time until tracheostomy ventilation or death. Tracheostomy is a surgical procedure that creates an opening in the windpipe for mechanical ventilation.
Results showed that Radicava-treated patients lived nearly two times longer than those not given the therapy (median of 49 vs. 25 months). Notably, this survival benefit remained significant after adjusting for potential influencing factors, such as age of onset, riluzole treatment, and disease severity at study’s start.
Two patients in the Radicava group underwent tracheostomy ventilation either at 16 months (nearly 1.5 years) or 62 months (more than five years) after disease onset.
Long-term treatment with Radicava was generally well-tolerated, with no treatment-related serious adverse side effects. No patient discontinued Radicava due to adverse events, with disease worsening as the most common reason to stop treatment (seven of nine patients).
These findings highlighted that Radicava “improves survival and maintains its effect over several years without serious adverse events,” the researchers wrote.
Given the small number of patients included in this study, larger studies are needed to confirm these findings and compare Radicava’s survival effects based on age and site of onset.
Such analyses will be feasible with data from an ongoing post-marketing study that is assessing the five-year safety and effectiveness of Radicava in 700 ALS patients living in Japan.
Mitsubishi Tanabe Pharma America, which commercializes Radicava in the U.S., also is evaluating an oral formulation of Radicava in adults with ALS living in North America, Japan, and certain European countries in a Phase 3 clinical trial (NCT04165824) and its open-label extension study (NCT04577404), expected to end by September 2023.