Edaravone

There were no surprises when results from the PHOENIX study of Amylyx Pharmaceuticals' ill-fated Relyvrio (sodium phenylbutyrate/taurursodiol) were presented at the American Academy of Neurology (AAN) annual meeting this week, but details from the confirmatory trial reveal the extent of how poorly the amyotrophic lateral sclerosis (ALS) drug performed.Relyvrio was granted an accelerated approval by the FDA in 2022 based mainly on results from a small Phase II study that was criticised at the time by some of the agency’s scientists. Earlier this month, Amylyx said it would withdraw Relyvrio in the US – and in Canada where it was sold as Albrioza – making good on a promise to do so if the drug didn't succeed in the much-anticipated Phase III PHOENIX trial.The study randomised 664 patients with clinically definite or clinically probable ALS within two years of symptom onset to receive either Relyvrio or placebo. The primary endpoint, change from baseline in ALS Functional Rating Scale-Revised (ALSFRS-R) total score at 48 weeks, dropped by an average of 14.98 points in the Relyvrio group versus 15.32 for placebo. The difference was not statistically significant.The 137-patient CENTAUR study that supported the drug's approval had shown a difference of 2.32 points on the ALSFRS-R score in favour of Relyvrio versus placebo.Meanwhile, PHOENIX also showed that Relyvrio had no significant benefit in key subgroups, including patients who were not on Mitsubishi Tanabe Pharma's Radicava (edaravone). Further, about a quarter of PHOENIX patients met enrolment criteria that was used in the CENTAUR trial – including having >60% slow vital capacity and being within 1.5 years of symptom onset – and while these patients fared better, the difference on ALSFRS-R was also not significant.Trial investigator Leonard van den Berg said researchers will "continue to learn from PHOENIX to inform future ALS trials." Investigators are also still collecting survival data, but the full results for this secondary endpoint will not be available until 2025 or 2026. Subgroup analyses are also further evaluating "CENTAUR-like" patients in the study and comparing them to results from that trial.For related analysis, see KOL Views Q&A: Leading neurologist outlines where ALS field goes next after Amylyx's Relyvrio failure.

JERSEY CITY, N.J., April 15, 2024 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced the presentation of one abstract in amyotrophic lateral sclerosis (ALS) at the Academy of Managed Care Pharmacy (AMCP) 2024 Annual Meeting, being held in New Orleans, La., Apr. 15-18, 2024. "Understanding healthcare resource utilization is crucial for assessing RADICAVA ORS® (edaravone) cost-effectiveness, optimizing healthcare delivery, improving patient outcomes and guiding healthcare planning," said Gustavo A. Suarez Zambrano, M.D., Vice President of Medical Affairs at MTPA. "Our data demonstrate not only the economic value of RADICAVA ORS, but also its potential to improve efficiency and reduce healthcare costs. These real-world results underscore our commitment to provide innovative treatments that make a meaningful difference in patient care and healthcare outcomes." MTPA's poster will be on display on Tuesday, Apr. 16 from 11 a.m.-6 p.m. and Wednesday, Apr. 17 from 9-11:30 a.m. in the Expo Hall. The presentation will include a preliminary analysis of demographics, characteristics and healthcare resource utilization data, as part of Optum's de-identified Clinformatics® Data Mart (CDM). Healthcare Resource Utilization of Oral Edaravone-Treated Patients with Amyotrophic Lateral Sclerosis Enrolled in an US-Based Administrative Claims Database (Malgorzata Ciepielewska, MS, MTPA) About RADICAVA® (edaravone) and RADICAVA ORS® (edaravone) The U.S. Food and Drug Administration (FDA) approved RADICAVA® (edaravone) on May 5, 2017, and the oral formulation RADICAVA ORS ® (edaravone) on May 12, 2022, for the treatment of amyotrophic lateral sclerosis (ALS). RADICAVA is administered in 28-day cycles by intravenous (IV) infusion. It takes 60 minutes to receive each 60 mg dose. For the initial cycle, the treatment is infused daily for 14 consecutive days, followed by a two-week drug-free period. All cycles thereafter are infused daily 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. For subsequent treatment cycles, RADICAVA ORS is taken for 10 days within a 14-day period followed by a 14-day drug-free period. RADICAVA ORS should be taken in the morning after overnight fasting. Patients should not eat or drink (except water) within one hour after taking RADICAVA ORS.1 Edaravone was discovered and developed for ALS by Mitsubishi Tanabe Pharma Corporation (MTPC) and commercialized in the U.S. by Mitsubishi Tanabe Pharma America, Inc. (MTPA). The MTPC group companies began researching ALS in 2001 through an iterative clinical platform over a 13-year period. In 2015, edaravone was approved as RADICUT® for the treatment of ALS in Japan and South Korea. Marketing authorizations were subsequently granted in Canada (October 2018), Switzerland (January 2019), Indonesia (July 2020), Thailand (April 2021), Malaysia (December 2021) and Brazil (February 2024). Marketing authorization for RADICAVA® Oral Suspension was granted in Canada (November 2022) and Switzerland (May 2023), and RADICUT® Oral Suspension 2.1% was granted regulatory approval in Japan in December 2022. To date, in the U.S., RADICAVA and RADICAVA ORS have been used to treat over 14,600 people with ALS, with over 1.8-million days of therapy, and have been prescribed by more than 2,300 HCPs.2-4 IMPORTANT SAFETY INFORMATION Hypersensitivity Reactions RADICAVA (edaravone) and RADICAVA ORS (edaravone) are contraindicated in patients with a history of hypersensitivity to edaravone or any of the inactive ingredients of this product. Hypersensitivity reactions (redness, wheals, and erythema multiforme) and cases of anaphylaxis (urticaria, decreased blood pressure, and dyspnea) have occurred with RADICAVA. Patients should be monitored carefully for hypersensitivity reactions. If hypersensitivity reactions occur, discontinue RADICAVA or RADICAVA ORS, treat per standard of care, and monitor until the condition resolves. Sulfite Allergic Reactions RADICAVA and RADICAVA ORS contain sodium bisulfite, a sulfite that may cause allergic-type reactions, including anaphylactic symptoms and life-threatening or less severe asthmatic episodes in susceptible people. The overall prevalence of sulfite sensitivity in the general population is unknown but occurs more frequently in asthmatic people. Adverse Reactions The most common adverse reactions (≥10%) reported in RADICAVA-treated patients were contusion (15%), gait disturbance (13%), and headache (10%). In an open label study, fatigue was also observed in 7.6% of patients receiving RADICAVA ORS. Pregnancy Based on animal data, RADICAVA and RADICAVA ORS may cause fetal harm. To report suspected adverse reactions or product complaints, contact Mitsubishi Tanabe Pharma America, Inc., at 1-888-292-0058. You may also report suspected adverse reactions to the FDA at 1-800-FDA-1088 or . INDICATION RADICAVA and RADICAVA ORS are indicated for the treatment of amyotrophic lateral sclerosis (ALS). For more information, including full Prescribing Information, please visit . About Mitsubishi Tanabe Pharma America, Inc. Based in Jersey City, N.J., Mitsubishi Tanabe Pharma America, Inc. (MTPA) is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC). It was established by MTPC to develop and advance our pipeline as well as commercialize approved pharmaceutical products in North America. For more information, please visit or follow us on X (formerly Twitter), Facebook and LinkedIn. About Mitsubishi Tanabe Pharma Corporation Mitsubishi Tanabe Pharma Corporation (MTPC), the pharma arm of Mitsubishi Chemical Group (MCG), is one of the oldest pharmaceutical companies in the world, founded in 1678. MTPC is headquartered in Doshomachi, Osaka, the birthplace of Japan's pharmaceutical industry. MCG has positioned health care as its strategic focus in its management policy, "Forging the future". MTPC sets the MISSION of "Creating hope for all facing illness". To that end, MTPC is working on the disease areas of central nervous system, immuno-inflammation, diabetes and kidney, and cancer. MTPC is focusing on "precision medicine" to provide drugs with high treatment satisfaction and additionally working to develop "around the pill solutions" to address specific patient concerns based on therapeutic medicine, including prevention of diseases, pre-symptomatic disease care, prevention of aggravation and prognosis. For more information, go to . Media inquiries: [email protected] 1 RADICAVA and RADICAVA ORS Prescribing Information. Jersey City, NJ: Mitsubishi Tanabe Pharma America, Inc.; 2022. 2 Data on file. Mitsubishi Tanabe Pharma America, Inc. 3 Data on file. Mitsubishi Tanabe Pharma America, Inc. 4 Data on file. Mitsubishi Tanabe Pharma America, Inc. SOURCE Mitsubishi Tanabe Pharma America