Mitsubishi Tanabe Pharma America’s (MTPA) oral therapy Radicava (edaravone) has shown decreased functional decline and improved survival in long-term amyotrophic lateral sclerosis (ALS) trials.
The analysis is based on four ALS studies, with primary analysis from the post-marketing Phase IIIb MT-1186-A02 (NCT04569084) and its extension study MT-1186-A04 (NCT05151471). Further analysis comes from the Phase III MT-1186-A01 trial (NCT04165824) and its extension study MT-1186-A03 (NCT04577404).
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The analysis compared patients treated with Radicava to Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) historical placebo controls, which included patients who did not receive Radicava but may have received Riluzole in their respective clinical trials.
In the primary analysis cohort, from the A02 and A04 studies, three out of the 78 patients (3.8%) died in the Radicava group compared to 14/78 (17.9%) in the PRO-ACT placebo group.
The baseline risk-adjusted hazard ratio showed an 84% decreased risk of death, indicating a longer survival time in Radicava patients.
While positive trends were observed for the number of deaths and time to death in both the once daily Radicava and on/off Radicava, the approved dosing regimen, when compared to PRO-ACT, neither one individually reached statistical significance for superiority.
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By GlobalDataIn the full cohort, covariate-adjusted restricted mean survival time (RMST) analysis showed a mean prolongation of 7.3 months in survival from 29.6 months in the Radicava group to 22.2 months in the PRO-ACT group. RMST is a metric that estimates the average time a group survives relative to a comparator.
Over 48 weeks of treatment, Radicava was also associated with a slower rate of decline in ALSFRS-R scores. In the primary analysis cohort, patients in both groups had a mean baseline ALS Functional Rating Scale-Revised (ALSFRS-R) score of 41.0 points, with the Radciva group seeing a change of −8.4 points compared to −14.1 points for PRO-ACT patients.
The least squares (LS) mean change in the primary analysis group was −8.4 ± 1.0 points for Radicava compared to −14.1 ± 1.0 points for PRO-ACT patients, a difference of 5.6 points.
In the broader cohort, the LS mean change was −10.5 ± 0.7 points for RADICAVA ORS compared to −12.9 ± 0.6 points for PRO-ACT patients, a difference of 2.4 points.
MTPA medical affairs vice president Dr Gustavo A Suarez Zambrano said: “These findings contribute to the ongoing effort to address unmet needs in ALS treatment by providing insight into the benefits observed with Radicava ORS. By publishing this analysis, we aim to deliver meaningful insights for healthcare providers, patients and caregivers.”
The findings, titled ‘Analysis of Long-term Function and Survival of Edaravone Oral Suspension-Treated Patients With Amyotrophic Lateral Sclerosis Using PRO-ACT Data as Historical Placebo Controls’, were published in Muscle and Nerve.
Oral Radicava was approved by the US Food and Drug Administration (FDA) on 12 May 2022, five years after the IV approval. The therapy was granted Orphan Drug Exclusivity in 2024 due to its oral suspension route, as opposed to most therapies being intravenously (IV) infused.
GlobalData predicts that sales of ALS products will reach $1.28bn by 2029, with growth primarily being led by 12 novel pipeline agents.
GlobalData is the parent company of Clinical Trials Arena.
