Radicava ORS (edaravone oral) / Tanabe Pharma, Shionogi - LARVOL DELTA

Shionogi Recognizes the Four-Year Anniversary of RADICAVA ORS (edaravone) for Treatment of ALS (Shionogi Press Release) - "To date, RADICAVA ORS has been prescribed by approximately 1,640 physicians in the U.S., with about 810 physicians prescribing it monthly to more than 4,500 people for the treatment of ALS."

Commercial • Amyotrophic Lateral Sclerosis

Exploratory Post Hoc Analysis of Neutrophil-to-lymphocyte Ratio as a Novel Response Biomarker for Edaravone Oral Suspension-treated Patients with Amyotrophic Lateral Sclerosis vs Untreated Propensity Score-matched PRO-ACT Historical Placebo Controls (AAN 2026) - P3 | "Background The US Food and Drug Administration (FDA) approved an on/off dosing regimen of Radicava ORS® (edaravone) oral suspension for patients with ALS...MT-1186-A01/A02/A03/A04 patients were propensity score-matched 1:1 on 11 baseline variables with historical, PRO-ACT controls (patients given placebo may have received riluzole)...These analyses were non-randomized comparisons, and there was a potential time bias between matched groups. Further studies are needed to validate NLR as a prognostic biomarker across ALS populations."

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

Shionogi to Present RADICAVA (edaravone) ORS and Digital Health Analyses in ALS at AAN 2026 (Businesswire) - "An analysis will be presented assessing neutrophil-to-lymphocyte ratio (NLR) changes in response to edaravone oral suspension-treated patients with ALS and examining the predictive value of baseline NLR compared with propensity score-matched PRO-ACT placebo controls. These results further examine the potential of NLR as a prognostic biomarker in ALS....A study will be presented evaluating smartphone-based telespirometry as an at-home tool for ALS monitoring. In a six-month prospective study of patients receiving edaravone, ALSFRS-R decline differed based on non-invasive ventilation (NIV) status."

Biomarker • Clinical data • Amyotrophic Lateral Sclerosis

Shionogi Expands Rare Disease Presence with Planned Acquisition of Global Rights to Tanabe Pharma Corporation’s RADICAVA ORS (edaravone) and IV RADICAVA (Businesswire) - "Tanabe Pharma plans to form this new entity, and Shionogi intends to purchase 100% of its shares. The agreement between Shionogi and Tanabe Pharma was signed this afternoon. As part of the transaction, a new business company established by Tanabe for RADICAVA in the U.S. will become a wholly owned subsidiary of Shionogi Inc....Under this agreement, Shionogi will pay a lump sum of USD 2.5 billion to Tanabe Pharma through Shionogi Inc., upon completion of the procedures. Additionally, Shionogi may pay a royalty on future sales, subject to certain conditions."

M&A • Amyotrophic Lateral Sclerosis

Radicut Suspension Approved in Korea for the Treatment of ALS (Mitsubishi Tanabe Press Release)

Korea approval • 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

Mitsubishi Tanabe Pharma Canada, Inc. Announces Publication of Long-Term Function and Survival Analysis of RADICAVA Oral Suspension (edaravone) -Treated Patients with ALS (Canada Newswire) - "The primary analysis, based on data from clinical studies MT-1186-A02/A04, evaluated long-term survival and functional outcomes of 78 propensity score-matched patients treated with RADICAVA Oral Suspension compared to 78 matched historical placebo controls from the PRO-ACT database....The post-hoc analysis, based on data from clinical studies MT-1186-A01/A02/A03/A04, expanded the evaluation to a broader ALS population (n=210 RADICAVA Oral Suspension patients vs. 210 PRO-ACT placebo patients) to confirm robustness of the initial results."

Retrospective data • Amyotrophic Lateral Sclerosis

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

MT-1186-A02: Efficacy and Safety Study of Oral Edaravone Administered in Subjects With ALS (clinicaltrials.gov) - P3 | N=384 | Terminated | Sponsor: Mitsubishi Tanabe Pharma America Inc. | Phase classification: P3b ➔ P3

Phase classification • Amyotrophic Lateral Sclerosis • CNS Disorders

MT-1186-A04: Efficacy and Safety Extension Study of Oral Edaravone Administered in Subjects With ALS (clinicaltrials.gov) - P3 | N=202 | Terminated | Sponsor: Mitsubishi Tanabe Pharma America Inc. | Phase classification: P3b ➔ P3

Phase classification • Amyotrophic Lateral Sclerosis • CNS Disorders

RADICAVA ORS (edaravone) Studies (PRNewswire) - "Final efficacy and safety 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 at 48 weeks (MT-1186-A02) and its extension study (MT-1186-A04). Safety data from a Phase 3 open-label safety extension study for up to 96 weeks (MT-1196-A03) will also be shared. An additional presentation will examine the long-term function and survival of patients treated with RADICAVA ORS using data from propensity score-matched patients in the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database."

P3 data • Retrospective data • Amyotrophic Lateral Sclerosis

Mitsubishi Tanabe Pharma America to Showcase New Research Findings in ALS at 2025 MDA Clinical and Scientific Conference (PRNewswire) - "Preclinical Data: Data from a preclinical study examining the effects of edaravone on spinal motor neurons derived from an ALS patient carrying the A382T mutation in TDP-43, as analyzed through a comprehensive transcriptomic approach, will be presented."

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

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 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

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

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