Radicava (edaravone) / Mitsubishi Tanabe - LARVOL DELTA

Describing the Amyotrophic Lateral Sclerosis US Population and Use of Supportive Aids and Interventions: Results from a Real-World Survey (ISPOR 2025) - "Of these, 63% were prescribed riluzole, 23% oral edaravone, and 17% IV edaravone. These findings describe the consulting pALS population in the US, highlighting the substantial and varied use of supportive aids/interventions, with a greater number of pALS who consulted in SC using expensive mobility and communication aids."

Clinical • Real-world • Real-world evidence • Amyotrophic Lateral Sclerosis • CNS Disorders • Infectious Disease

Mitsubishi Tanabe Pharma America Announces Publication of Preclinical Research on the Role of Edaravone in Mitigating TDP-43 Mislocalization in ALS (PRNewswire) - "Mitsubishi Tanabe Pharma...announced the publication of new preclinical research in Free Radical Biology and Medicine, highlighting the role of edaravone in mitigating TDP-43 mislocalization, a hallmark feature observed in more than 97% of sporadic amyotrophic lateral sclerosis (ALS) cases...In ALS patient-derived neurons from a patient with ALS harboring a TDP-43 mutation, edaravone significantly reduced the cytoplasmic accumulation of TDP-43, restoring its nuclear localization; Unlike vitamin C, edaravone preserved neurite structures and reduced neurotoxicity, supporting a unique mechanism of action; Edaravone treatment induced significant changes in gene expression as early as six hours post-treatment, with key pathways related to protein degradation and neuroprotection; Edaravone's effects were associated with modulation of the SIRT1-XBP1 pathway, a key regulator of cellular stress responses, suggesting a broader role in ALS neuroprotection."

Preclinical • Amyotrophic Lateral Sclerosis

Long-term Function and Survival of Radicava ORS® (Oral Edaravone)-Treated Patients With ALS vs Propensity Score–Matched PRO-ACT Historical Controls (AAN 2025) - "Analysis of MTPA oral edaravone–treated patients with ALS from MT-1186 studies suggests MTPA oral edaravone significantly increases survival and decreases functional decline vs PRO-ACT placebo."

Clinical

Referral Patterns for Patients With Amyotrophic Lateral Sclerosis Enrolled in a US-Based Administrative Claims Database (AAN 2025) - "There are currently 3 US Food and Drug Administration-approved treatments for patients with ALS: riluzole, edaravone (Radicava® IV [intravenous] and Radicava ORS® oral suspension; Mitsubishi Tanabe Pharma America [MTPA]), and tofersen (for patients with a superoxide dismutase 1 mutation).Design/Patients with ALS continuously enrolled in Optum's de-identified Clinformatics® Data Mart (CDM) from January 2007 to December 2023 were included and grouped based on ALS treatment pattern (ie, untreated, riluzole-only, MTPA edaravone±riluzole)...These real-world data may help clinicians and payers better understand the referral patterns of patients with ALS, which may aid in reducing delays in diagnosis and treatment."

Claims database • Clinical • Amyotrophic Lateral Sclerosis • CNS Disorders • Psychiatry

Extension Study MT-1186-A04 Evaluating Oral Edaravone (Radicava ORS®) Continued Efficacy and Safety up to an Additional 48 Weeks in Patients With ALS (AAN 2025) - P3b | "Similar to the results obtained in MT-1186-A02, in MT-1186-A04, daily MTPA oral edaravone did not show superiority to the FDA-approved on/off regimen (same safety, efficacy and tolerability profile) from the time of the randomization date in Study MT-1186-A02 to ≥12-point decrease in ALSFRS-R or death, whichever happened first, and reinforces the appropriateness of the FDA-approved regimen."

Clinical • Amyotrophic Lateral Sclerosis • CNS Disorders

Treatment of Amyotrophic Lateral Sclerosis in General and Specialist US Healthcare Settings: Results From a Real-World Survey (AAN 2025) - "Of those receiving treatment, 66% were prescribed riluzole formulations (52%, 78%), 36% IV or oral edaravone (45%, 28%), and 5% tofersen (7%, 3%)...These initial findings highlight the complex treatment landscape for pALS in the US, with substantial use of specialist care and diverse pharmacological approaches reflecting the varied needs of patients in managing their disease."

Clinical • Real-world • Real-world evidence • Amyotrophic Lateral Sclerosis • CNS Disorders • Pain

Evolution of the Treatment Landscape for Patients Diagnosed with Amyotrophic Lateral Sclerosis in the United States (AAN 2025) - "FDA-approved ALS treatments, including riluzole, Mitsubishi Tanabe Pharma America (MTPA) IV (intravenous) and oral edaravone (Radicava® IV and Radicava ORS®), and sodium phenylbutyrate and taurursodiol (withdrawn from the US market following this analysis period), initiated within 6 months of ALS diagnosis and on/after the incident ALS diagnosis were examined by ALS diagnosis year.We observed an increase in the use of riluzole among patients with ALS diagnosed between 2016 and 2022, from 5.9% in 2016 to 24.8% in 2022. During the 2016-2022 period, there was increased use of riluzole and a slow shift toward MTPA IV edaravone. With FDA approval of MTPA oral edaravone in 2022, there was a shift observed toward this newer option. Although many patients still had not received any FDA-approved treatments, that number has decreased, possibly due, in part, to the approval of MTPA oral edaravone."

Clinical • Amyotrophic Lateral Sclerosis • CNS Disorders

Phase 3, Open-Label, Safety Extension Study of Oral Edaravone (Radicava ORS®) Administered Over 96 Weeks in Patients with ALS (MT-1186-A03) (AAN 2025) - P3 | "MTPA oral edaravone showed no new safety concerns and was well-tolerated during the 96-week study period of MT-1186-A03, for a total of 144 weeks when including Study MT-1186-A01."

Clinical • P3 data • Amyotrophic Lateral Sclerosis • CNS Disorders • Constipation • Gastroenterology • Gastrointestinal Disorder • Pulmonary Disease

Mitsubishi Tanabe Pharma America Announces Real-World Data Analysis of Disease Progression with RADICAVA (edaravone) in ALS (PRNewswire) - P=Obs | N=NA | "The observational analysis assessed time-to-progression milestones - such as use of canes, walkers, wheelchairs, artificial nutrition, noninvasive ventilation, speech-generating devices and hospice - for up to 24 months in people with ALS treated with RADICAVA compared to a control group not treated with the therapy....People with ALS treated with RADICAVA had longer milestone-free times for four of six key progression milestones compared to those not treated with RADICAVA, including new use of canes/walkers/wheelchairs, artificial nutrition, noninvasive ventilation and hospice. No milestones were reported in people treated with RADICAVA compared those not treated from 0 to 24 months after the index date....Results from this analysis were published in the Journal of Comparative Effectiveness Research."

Observational data • Real-world • Amyotrophic Lateral Sclerosis

Longer disease progression milestone-free time in people with amyotrophic lateral sclerosis treated versus not treated with intravenous edaravone: results from an administrative claims analysis. (PubMed, J Comp Eff Res) - "Aim: To estimate time-to-progression milestones in people with amyotrophic lateral sclerosis (PALS) treated versus not treated with intravenous (IV) edaravone (Radicava® IV, Mitsubishi Tanabe Pharma America [MTPA], hereafter "IV edaravone") in a real-world setting...Cases treated with IV edaravone and controls not treated with IV edaravone were propensity score matched for: age, sex, race, US region of residence, pre-index disease duration, insurance, riluzole prescription; and pre-index claims for cardiovascular disease, artificial nutrition/gastrostomy tube, noninvasive ventilation and all-cause hospitalization...From 0 to 24 months post index, more cases (n = 129) than controls (n = 103) reported no milestones and more controls (n = 232) than cases (n = 131) reported deaths. In a US-based real-world setting, IV edaravone-treated PALS had a longer time to disease progression milestone events and fewer deaths..."

Journal • Amyotrophic Lateral Sclerosis • Cardiovascular • CNS Disorders

Mitsubishi Tanabe Pharma America to Present New ALS Research at MNDA's 35th International Symposium on ALS/MND (PRNewswire) - "Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced 10 presentations covering advances in amyotrophic lateral sclerosis (ALS) research at the Motor Neurone Disease Association (MNDA) 35th International Symposium on ALS/MND, being held in Montreal, Canada, from December 6-8...Final results from two multi-center, Phase 3b, double-blind, parallel group studies will be presented, including a comparison of daily dosing of RADICAVA ORS (edaravone) with the FDA-approved on/off regimen, assessed over 48 weeks (MT-1186-A02) and up to 96 weeks (MT-1186-A04). Additionally, safety data from a Phase 3 open-label safety extension study (MT-1196-A03) will be shared."

P3 data • Amyotrophic Lateral Sclerosis • CNS Disorders

Clinical trial of bosutinib for amyotrophic lateral sclerosis: induced pluripotent stem cell-based Drug Repurposing for Amyotrophic Lateral Sclerosis Medicine (iDReAM) study (ALS-MND 2024) - "Efficacy was assessed with the total ALSFRS-R compared with the placebo arm or edaravone arm in edaravone Study MCI186-19 and with registry data from a multicenter ALS cohort study, the Japanese Consortium for Amyotrophic Lateral Sclerosis Research (JaCALS). This is a Phase 2 open-label clinical trial. No new safety concerns were identified on 24-week treatment of bosutinib in patients with ALS. Three out of four efficacy endpoints were met in the efficacy analysis."

Clinical • Amyotrophic Lateral Sclerosis • Chronic Myeloid Leukemia • CNS Disorders • Hematological Malignancies • Leukemia • Oncology • ABL1 • Plasma NfL

Preliminary analysis of treatment combinations in patients with amyotrophic lateral sclerosis enrolled in an US-based administrative claims database (ALS-MND 2024) - "Sodium phenylbutyrate and taurursodiol (PB-TURSO) was FDA-approved in 2022, but voluntarily discontinued in 2024. Tofersen was FDA-approved for patients with ALS with a superoxide dismutase 1 mutation in 2023... Patients with ALS continuously enrolled in Optum's de-identified ClinformaticsV R Data Mart (CDM) from 1 August 2017, through 30 September 2023, were included (intravenous) and Radicava ORSV and grouped based on ALS treatment combination... Patients were grouped based on use of riluzole only (n = 2193) vs. other FDA approved treatment/treatment combinations (n = 967) including Mitsubishi Tanabe Pharma America (MTPA) edaravone, PB-TURSO, riluzole ± edaravone, edaravone ± PB-TURSO, riluzole ± PB-TURSO, or riluzole þ edaravone ± PB-TURSO. Patients were predominantly male (53.5–53.6%), White (72.2–74.4%) and covered by Medicare (68.6–77.8%) rather than commercial insurance."

Claims database • Clinical • Amyotrophic Lateral Sclerosis • CNS Disorders

Evolution of the treatment landscape for patients diagnosed with ALS in the United States (ALS-MND 2024) - "FDA-approved ALS treatments, including riluzole, RadicavaV R (edaravone) IV (intravenous), Radicava ORSV (edaravone) oral suspension, and sodium phenylbutyrate and taurursodiol (which was withdrawn from the US market after the assessment period in this analysis), initiated within 6 months of ALS diagnosis and on/after the incident ALS diagnosis were examined by ALS diagnosis year. We observed a consistent increase in the use of riluzole among patients with ALS diagnosed from 2016 to 2022, rising from 5.9% in 2016 diagnoses to 24.8% in 2022. Mitsubishi Tanabe Pharma America (MTPA) IV edaravone was introduced in 2017 and saw a consistent increase from 1 to 4.8% between 2017 and 2021. However, with the introduction of oral edaravone in 2022, the use of IV edaravone decreased to 1%, while the use of oral edaravone increased to 8.9%."

Clinical • Amyotrophic Lateral Sclerosis • CNS Disorders

Canadian regulatory framework and flexibility in the context of rare diseases with unmet medical need such as amyotrophic lateral sclerosis (ALS) (ALS-MND 2024) - "Access to ALS Therapies in Canada: There are currently two available ALS treatments in Canada: RADICAVA (edaravone) and RILUTEK (riluzole)... HC follows rigorous scientific and regulatory processes for drug approval, while exercising flexibility within the regulatory framework to respond to the pressing need for rare diseases therapies, including ALS, for Canadian patients."

Amyotrophic Lateral Sclerosis • CNS Disorders • Rare Diseases

Phase 3, open-label, safety extension study of oral edaravone (Radicava ORSV R ) administered over 96 weeks in patients with ALS (MT-1186-A03) (ALS-MND 2024) - P3 | "In Study MT-1186-A03, oral edaravone was well tolerated with no new safety concerns. The most common treatment-emergent adverse events (TEAEs) were fall, muscular weakness, dyspnea, constipation, and dysphagia. These TEAEs were consistent with the safety profile for edaravone from previous clinical trials."

Clinical • P3 data • Amyotrophic Lateral Sclerosis • CNS Disorders • Constipation • Gastroenterology • Gastrointestinal Disorder • Pulmonary Disease

Phase 3b study MT-1186-A02 to investigate the superiority of daily dosing vs the FDA-approved on/off regimen of oral edaravone (Radicava ORSV R )in patients with ALS (ALS-MND 2024) - P3b | "At week 48, combined assessment of function and survival (CAFS), including change in ALSFRS-R score and time to death, indicated daily dosing did not show a statistically significant difference vs the FDA-approved on/off dosing. Oral edaravone was well tolerated and no new safety concerns were identified in either group in Study MT-1186-A02. Daily oral edaravone did not show superiority to the FDA-approved on/off regimen in the CAFS, and was comparable in safety, efficacy, and tolerability to the on/off dosing regimen, therefore reinforcing the appropriateness of the FDA-approved regimen."

Clinical • Head-to-Head • P3 data • Amyotrophic Lateral Sclerosis • CNS Disorders

Phase 3b Study MT-1186-A04 Extension Study to Evaluate the Continued Efficacy and Safety of Radicava ORSV R (Oral Edaravone) for up to an Additional 48-Weeks in Patients With Amyotrophic Lateral Sclerosis (ALS-MND 2024) - P3b | "Over 96 weeks including the treatment period of Study MT-1186-A02, results for the primary endpoint indicated daily dosing did not show a statistically significant difference vs the FDA-approved on/off dosing. Oral edaravone was well tolerated and no new safety concerns were identified in either group in Study MT-1186-A04. Similar to the results obtained in MT-1186-A02, in MT-1186-A04, daily Radicava ORSV R did not show superiority to the FDA-approved on/off regimen (same safety, efficacy and tolerability profile) from the time of the randomization date in Study MT-1186-A02 to at least a 12-point decrease in ALSFRS-R or death, whichever happened first, and reinforces the appropriateness of the FDA-approved regimen."

Clinical • P3 data • Amyotrophic Lateral Sclerosis • CNS Disorders

Treatment patterns and survival benefit of edaravone-treated people with ALS in the ALS/MND Natural History Consortium (ALS-MND 2024) - "Radicava ORSV R (edaravone) oral suspension was FDA approved in 2022... Patients receiving edaravone ± riluzole (n 1⁄4 176) were matched to those receiving riluzole only (n 1⁄4 176) on sex, age, body mass index, race; and pre-index non-invasive ventilation, artificial nutrition, and disease duration; baseline mean ± SD ALS Functional Rating Scale-Revised score (39.5 ± 4.8 and 39.3 ± 4.8, respectively) and baseline forced vital capacity %–predicted (79.3%±23.5 and 79.4%±21.4%, respectively). Matched variables had a standardized mean difference 0.1. After baseline covariate adjustment, RMST analyses over 50 months suggested a survival benefit for patients receiving edaravone ± riluzole (30.5 months) vs."

Amyotrophic Lateral Sclerosis • CNS Disorders

Efficacy of Radicava® IV (intravenous edaravone) in subjects with differing trajectories of disease progression in amyotrophic lateral sclerosis: Use of a novel statistical approach for post hoc analysis of a pivotal phase 3 clinical trial. (PubMed, J Neurol Sci) - "This post hoc analysis suggests MTPA IV edaravone treatment results in slower ALSFRS-R decline vs placebo in most predicted nonlinear trajectories. LCA is a novel approach that may benefit future trial analyses."

Journal • P3 data • Retrospective data • Amyotrophic Lateral Sclerosis • CNS Disorders

Protocol for a phase 2 study of bosutinib for amyotrophic lateral sclerosis using real-world data: induced pluripotent stem cell-based drug repurposing for amyotrophic lateral sclerosis medicine (iDReAM) study. (PubMed, BMJ Open) - P1/2 | "Efficacy using the ALSFRS-R score will be compared with the external published data from an edaravone study (MCI186-19) and registry data from a multicentre ALS cohort study, the Japanese Consortium for Amyotrophic Lateral Sclerosis Research. The findings will be disseminated in peer-reviewed journals and at scientific conferences. jRCT2051220002; Pre-results, NCT04744532; Pre-results."

Clinical protocol • Journal • P2 data • Real-world • Real-world evidence • Amyotrophic Lateral Sclerosis • CNS Disorders • ABL1

Mitsubishi Tanabe Pharma America Unveils New ALS Research Findings at NEALS 2024 Annual Meeting (PRNewswire) - "Mitsubishi Tanabe...announced that twelve presentations following expansive research in amyotrophic lateral sclerosis (ALS) will be shared as part of the Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) 2024 Annual Meeting...A presentation will examine the impact of RADICAVA ORS on survival and function in ALS patients, using data from the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database. For this analysis, patients were propensity score-matched with participants from studies MT-1186 A01-A04. Additionally, multiple presentations will explore RADICAVA ORS through multi-center, phase 3b, double-blind, parallel group studies, including a comparison of daily dosing to the U.S. Food and Drug Administration (FDA)-approved on/off regimen, evaluated over 48 weeks (MT-1186-A02) and up to 96 weeks (MT-1186-A04)....Two preclinical studies will be presented, showcasing the effects of edaravone."

Clinical data • Preclinical • Amyotrophic Lateral Sclerosis • CNS Disorders

Phase 3b Study MT-1186-A02 to Investigate the Superiority of Daily Dosing vs the FDA-approved On/Off Regimen of Oral Edaravone in Patients with Amyotrophic Lateral Sclerosis (ANA 2024) - P3b | "Introduction: An on/off dosing regimen of Radicava IV (edaravone) and Radicava ORS (edaravone) oral suspension was approved by the US Food and Drug Administration (FDA) for the treatment of amyotrophic lateral sclerosis (ALS) in 2017 and 2022, respectively. Daily oral edaravone did not show a statistically significant difference, and therefore did not show superiority, to the FDA-approved on/off regimen in the CAFS and reinforces the appropriateness of the FDA-approved regimen."

Clinical • Head-to-Head • P3 data • Amyotrophic Lateral Sclerosis • CNS Disorders

Phase 3, Open-Label, Safety Extension Study of Oral Edaravone Administered Over 96 Weeks in Patients with ALS (MT-1186-A03) (ANA 2024) - P3 | "This support was funded by Mitsubishi Tanabe Pharma America, Inc., Jersey City, NJ, in accordance with Good Publication Practice Guidelines 202 Introduction: Radicava (intravenous [IV] edaravone) and Radicava ORS (oral suspension edaravone) were approved by the US Food and Drug Administration (FDA) for the treatment of amyotrophic lateral sclerosis (ALS) in 2017 and 2022, respectively, and studies have demonstrated these approved formulations have similar pharmacokinetics. Oral edaravone showed no new safety concerns and was well-tolerated during the 96-week study period. Sponsorship This study was sponsored by Mitsubishi Tanabe Pharma America, Inc.Acknowledgments: The authors thank Irene body, VMD, PhD, of p-value communications, Cedar Knolls, NJ, for providing medical writing support. Editorial support was also provided by p-value communications."

Clinical • P3 data • Amyotrophic Lateral Sclerosis • CNS Disorders

Mitsubishi Tanabe Pharma America to Showcase RADICAVA ORS (edaravone) Presentations at 2024 Neuromuscular Study Group Annual Meeting (PRNewswire) - "An updated analysis from the ALS/Motor Neuron (MND) Natural History Consortium (NHC) registry will be presented, focusing on treatment patterns among individuals with ALS treated with RADICAVA. Additionally, a preliminary analysis will examine the demographics, clinical characteristics and treatment patterns in individuals with ALS using a repository database. Another ongoing analysis will explore healthcare resource utilization among individuals with ALS treated with RADICAVA ORS using administrative claims data using Optum's Clinformatics Data Mart (CDM)."

Real-world • Amyotrophic Lateral Sclerosis • CNS Disorders