IV edaravone may slow disease-progression milestones in ALS
Key takeaways:
- The treatment group logged longer times to four disease-progression milestones compared with controls.
- At 2 years, more untreated controls died than those treated with IV edaravone.
Treatment with IV edaravone for ALS was linked to a slowing of disease-progression milestones compared with no IV edaravone treatment, results of an administrative claims analysis show.
"Since ALS has no cure, it is important to further explore the benefit of FDA-approved treatments related to the impact in reducing disease progression,” Gustavo A. Suarez Zambrano, MD, vice president of medical affairs at Mitsubishi Tanabe Pharma America, which funded the study, told Healio in an email regarding the study, which was published in the Journal of Comparative Effectiveness Research.
James D. Berry, MD, MPH, clinical researcher at the Healey Center for ALS at Massachusetts General Hospital, and colleagues sought to estimate the time-to-progression milestones in people with ALS (PALS) treated with IV edaravone (Radicava, Mitsubishi Tanabe Pharma America) in a real-world setting compared with those not given the treatment.
From an initial cohort of more than 15,000 adults, their retrospective, observational cohort study included 395 individuals with ALS (mean age, 63.2 years; 57.5% men) who were continuously enrolled in Optum's Clinformatic Data Mart between August 2017 and December 2021. All were propensity score matched to an equal number of controls (mean age, 63.7 years; 60.5% men) by age, sex, race, region of United States residence, pre-index disease duration, insurance, riluzole prescription, as well as pre-index claims for a range of physical maladies and all-cause hospitalization.
The index date for PALS was the first IV edaravone claim, while the index date was randomly assigned after IV edaravone market availability for controls.
Restricted mean time lost was calculated for six milestones of ALS disease progression: new use of gait assistance devices, need for artificial nutrition, noninvasive ventilation, progression to invasive ventilation, speech-generating devices and hospice care.
Results showed that PALS given IV edavarone recorded a smaller amount of restricted mean time lost compared with controls, indicating longer disease progression milestone-free time across all six milestones.
The researchers also reported that, between 0 to 24 months post-index date, more PALS (n = 129) vs. controls (n = 103) reported no milestone achievements, while more deaths were reported for controls (n = 232) than PALS (n = 131).
"These data provide real-world evidence that complements findings from randomized controlled trials and offers additional insights into how treatments like Radicava may impact the day-to-day lives of people living with ALS,” Zambrano told Healio.
Reference:
Mitsubishi Tanabe Pharma America announces real-world data analysis of disease progression with Radicava (edaravone) in ALS. https://www.mt-pharma-america.com/media/news/press-releases/2025/01/29/mitsubishi-tanabe-pharma-america-announces-real-world-data-analysis-of-disease-progression-with-radicava-edaravone-in-als. Published Jan. 29, 2025. Accessed Jan. 31, 2025.