Mitsubishi Tanabe Pharma America (MTPA) has announced results from a real-world data analysis indicating that treatment with intravenous edaravone is associated with longer disease progression milestone-free time in people with amyotrophic lateral sclerosis (ALS) compared to those not treated with IV edaravone.
The analysis, funded and conducted by MTPA, leveraged administrative claims data to evaluate the impact of edaravone on ALS disease progression. The findings, detailed in the publication "Longer Disease Progression Milestone-Free Time in People With Amyotrophic Lateral Sclerosis Treated With IV Edaravone vs Non–IV Edaravone-Treated: Results From an Administrative Claims Analysis," provide additional insights into the drug's effectiveness in a real-world setting.
Edaravone for ALS
Edaravone, marketed as RADICAVA IV and RADICAVA ORS (oral formulation), is approved by the U.S. Food and Drug Administration (FDA) for the treatment of ALS. RADICAVA was initially approved in 2017, with RADICAVA ORS gaining approval in 2022. The oral formulation was granted Orphan Drug Exclusivity in 2024, recognizing its contribution to patient care through an innovative oral delivery method.
RADICAVA is administered intravenously in 28-day cycles. The initial cycle involves daily infusions for 14 consecutive days, followed by a two-week drug-free period. Subsequent cycles consist of daily infusions for 10 days within a 14-day period, followed by a two-week drug-free period. RADICAVA ORS is taken daily for 14 consecutive days, followed by a 14-day drug-free period for the initial treatment cycle. Subsequent cycles involve taking RADICAVA ORS for 10 days within a 14-day period, followed by a 14-day drug-free period. It should be taken in the morning after overnight fasting, and patients should refrain from eating or drinking (except water) for one hour after ingestion.
Important Considerations
MTPA emphasized that the results from this real-world data analysis should be interpreted cautiously due to the study's observational and retrospective nature. The absence of randomization introduces potential biases and confounding factors that could influence the outcomes. The company also noted that the study's findings are not generalizable and cannot be used to draw definitive conclusions about treatment effects.
Safety Information
RADICAVA and RADICAVA ORS are contraindicated in patients with a history of hypersensitivity to edaravone or any of the inactive ingredients. Hypersensitivity reactions, including anaphylaxis, have been reported. The products contain sodium bisulfite, a sulfite that may cause allergic-type reactions in susceptible individuals. Common adverse reactions reported in RADICAVA-treated patients include contusion, gait disturbance, and headache. Fatigue has also been observed in patients receiving RADICAVA ORS. Based on animal data, RADICAVA and RADICAVA ORS may cause fetal harm.
To date, RADICAVA and RADICAVA ORS have been used to treat over 16,000 people with ALS in the U.S., with over 2.0-million days of therapy, and have been prescribed by over 2,400 healthcare professionals.
