Radicava (edaravone) / Mitsubishi Tanabe - LARVOL DELTA

Tanabe Pharma America to Present Preclinical Research at MNDA on Edaravone's Potential Mechanisms in ALS (PRNewswire) - "...Treatment with edaravone was observed to preserve neurite structures and reduce neuronal cell death in ALS iPSNs in a concentration-dependent manner, whereas the control treatment (vitamin C) did not produce similar effects. Edaravone also reduced indicators of oxidative stress and was associated with changes in TDP-43 localization."

Preclinical • Amyotrophic Lateral Sclerosis

Real-world data on disease-modifying drugs for ALS in Japan (ALS-MND 2025) - "Edaravone was previously administered as an intravenous injection, but on 17 April 2023, Radicut oral suspension was launched (the U.S. product name, RADICAVA ORSV, was approved in the U.S. in May 2022 and in Canada in November)... The 10th NDB Open Data covers the period including the launch of Edaravone oral suspension, and compares the number of prescriptions before and after the launch of Edaravone oral suspension. After the launch of Edaravone oral suspension, the number of prescriptions increased every month, while the number of outpatient prescriptions for Edaravone injection decreased. The number of prescriptions for Riluzole also gradually decreased."

Clinical • Real-world • Real-world evidence

Exploratory post hoc analysis of neutrophil-tolymphocyte ratio as a novel responsiveness biomarker for edaravone oral suspension–treated patients with amyotrophic lateral sclerosis vs untreated propensity score–matched PRO-ACT historical placebo controls (ALS-MND 2025) - P3 | "Sponsorship: Study sponsored by Mitsubishi Tanabe Pharma America, Inc., Jersey City, NJ, USA..Background: The US Food and Drug Administration (FDA) approved an on/off dosing regimen of Radicava ORSV(edaravone) oral suspension for patients with amyotrophic lateral sclerosis (ALS)...Study MT-1186-A01/A02/A03/A04 patients were propensity score matched 1:1 on 11 baseline variables with historical, PRO-ACT controls (placebo-arm participants not administered investigational treatment but may have received riluzole)... Relative to baseline NLR, patients from Studies MT-1186-A01/02/03/04 (n = 221) showed a numeric trend toward suppressing NLR at 48 weeks (least squares mean difference= −0.2851) vs. matched PRO-ACT controls (n = 221) (p = 0.065). Patients were grouped according to baseline NLR <3 or NLR ≥3."

Biomarker • Clinical • Retrospective data • Amyotrophic Lateral Sclerosis • CNS Disorders

At-home telespirometry identifies significantly slower decline of ALS functional rating scale-revised total score but not erect/supine slow vital capacity in edaravone-treated ALS subjects not requiring noninvasive ventilation [NCT05106569] (ALS-MND 2025) - P=N/A | "There was no statistically significant effect of IV edaravone or edaravone oral suspension on the: (1) mean delta eSVC/sSVC overall or according to NIV treatment status (2) mean delta ALSFRS-R total score overall. Analysis according to NIV treatment status identified a statistically significant (p = 0.037) slowing in the mean delta ALSFRS-R total score (−0.9 units/month [Q1, Q3= −1.2, −0.4]) in edaravone-treated subjects vs mean delta ALSFRS-R total score (−1.5 units/ month [Q1, Q3= −2.1, −0.8]) in ALS subjects not treated with edaravone that did not use NIV during the study. Strengths include number of subjects uniformly studied, higher ALSFRS-R progression rate compared with certain published rates, and NIV treatment initiation stratification."

Clinical • Non-invasive

Acceptance of Clinical Studies Conducted in Nonus Settings by the FDA Based on the Analysis of Selected Novel Drug Approvals (ISPOR-EU 2025) - "In 2017, only 2 (4%) novel drugs met the criterion, including Radicava, evaluated exclusively in Japan with no US patient representation... Cases show an increasing rate of products accepted based on foreign data. If studies are conducted under Good Clinical Practice (GCP) and there are no significant regional or ethnic differences in outcomes, they could be accepted without further requirements. However, sometimes post-marketing studies are needed, and recent US policy changes could increase the FDA's foreign data scrutiny."

Clinical

Edaravone for amyotrophic lateral sclerosis. (PubMed, Aust Prescr) - No abstract available

Journal • Review • Amyotrophic Lateral Sclerosis • CNS Disorders

EVALUATION OF LONG-TERM PROGNOSIS OF EDARAVONE IN ALS PATIENTS: A REAL-WORLD COMPARATIVE STUDY USING SUNRISE JAPAN AND JACALS REGISTRY DATA (PACTALS 2025) - "Additionally, comparisons were conducted within patient populations comparable to those in the clinical trial MCI186-19... This investigation, which examines the long- term prognostic impact of edaravone under real- world conditions using existing registry data from JaCALS, may provide a valuable reference for studies on other neurodegenerative diseases."

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

CLINICAL TRIAL OF BOSUTINIB FOR AMYOTROPHIC LATERAL SCLEROSIS: IDREAM STUDY (PACTALS 2025) - "Efficacy was assessed with the total ALSFRS-R compared to the placebo arm or edaravone arm in edaravone Study MCI186- 19 and with ALS registry data from JaCALS. This is a Phase 2 open-label clinical trial. No new safety concerns were identified in the 24-week bosutinib treatment of 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 • ABL1

ALS Treatment Update RADICAVA (PACTALS 2025) - "Sponsored by Teva Pharma Australia"

Real-world safety and tolerability of intravenous edaravone in patients with amyotrophic lateral sclerosis. (PubMed, Neurodegener Dis Manag) - "Treatment discontinuation was primarily related to ALS disease progression/death, rather than safety or tolerability. This study representative of real-world patients with ALS suggests that edaravone showed consistent safety and tolerability profiles with previous studies."

Journal • Real-world evidence • Amyotrophic Lateral Sclerosis • CNS Disorders

Development and Validation of a Stability-Indicating RP-HPLC Method for Edaravone Quantification. (PubMed, Molecules) - "Currently, it is available as an IV infusion (Radicava®, Jersey City, NJ, USA) and an oral liquid suspension (Radicava ORS®, Jersey City, NJ, USA)...Successful separation of edaravone from void signals and degradant products was achieved. The method was precise and accurate at the concentration range of 6.8-68.6 µg/mL and was recommended to use without methyl hydroxybenzoate (MHB) as an internal standard."

Journal • Amyotrophic Lateral Sclerosis

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. (PubMed, Muscle Nerve) - "This suggests edaravone oral suspension significantly increases survival time and decreases physical functional decline versus PRO-ACT placebo patients."

Journal • Amyotrophic Lateral Sclerosis • CNS Disorders

Safety Extension Study of Edaravone Oral Suspension in Patients With Amyotrophic Lateral Sclerosis for up to an Additional 96 Weeks of Treatment. (PubMed, Muscle Nerve) - P3 | "These results help establish the long-term safety and tolerability profile of edaravone oral suspension."

Journal • Amyotrophic Lateral Sclerosis • CNS Disorders • Constipation • Gastroenterology • Gastrointestinal Disorder • Pulmonary Disease

Efficacy and Safety of Once Daily Dosing vs. Approved On/Off Dosing of Edaravone Oral Suspension Up to 48 Weeks in Patients With Amyotrophic Lateral Sclerosis (Study MT-1186-A02). (PubMed, Muscle Nerve) - P3 | "Daily edaravone oral suspension did not show superiority and had equivalent safety and tolerability vs. the approved On/Off regimen, reinforcing the appropriateness of the approved dosing regimen."

Clinical • Journal • Amyotrophic Lateral Sclerosis • CNS Disorders

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

Biomarkers for Postoperative Intimal Hyperplasia. (PubMed, Int J Angiol) - "In Group edaravone, free-radical scavenger, edaravone (Radicut ® , Mitsubishi Tanabe Pharma Corp., Osaka Japan) was administered, instead of saline...The serum IL-8 levels in Group edaravone were appeared to be suppressed but not significant (132 ± 24 pg/mL, p  = 0.72). It is very important to detect the biomarker such as the serum IL-8, before the establishment of postoperative intimal hyperplasia."

Biomarker • Journal • CXCL8

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