JERSEY CITY, N.J., June 17, 2024 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced the presentation of final results from the global, multi-center, double-blind, Phase 3b MT-1186-A02 study of oral edaravone in amyotrophic lateral sclerosis (ALS). Final results showed the daily dosing regimen of RADICAVA ORS® (edaravone) did not show superiority to the U.S.

新泽西州泽西市（JERSEY CITY，N.J.），2024年6月17日/PRNewswire/--三菱田边制药美国公司（MTPA）今天宣布了全球多中心双盲3b期MT-1186-A02口服依达拉奉治疗肌萎缩侧索硬化症（ALS）的最终结果。最终结果显示，RADICAVA ORS®（依达拉奉）的每日给药方案没有显示出优于美国的优势。

Food and Drug Administration (FDA) approved on/off dosing regimen based on the primary endpoint of Combined Assessment of Function and Survival (CAFS) at 48 weeks. Both dosing regimens provided comparable change in ALSFRS-R from baseline to week 48. These findings suggest that the current on/off regimen of RADICAVA ORS is the most appropriate regimen for people living with ALS based on efficacy and safety findings from studies MT-1186-A02 and MCI186-19.

美国食品和药物管理局（FDA）根据48周时功能和生存综合评估（CAFS）的主要终点批准了开/关给药方案。从基线到第48周，两种给药方案均提供了ALSFRS-R的可比变化。这些发现表明，根据MT-1186-A02和MCI186-19研究的疗效和安全性发现，目前的RADICAVA-ORS开/关方案是ALS患者最合适的方案。

ALSFRS-R changes observed at week 48 in Study MT-1186-A02 of RADICAVA ORS were similar to the post hoc assessment of the previous Japanese, Phase 3 Study MCI186-19 of intravenous (IV) RADICAVA® (edaravone).1,2 The MT-1186-A02 study found no new safety concerns and reinforces edaravone as a safe product.

在RADICAVA ORS研究MT-1186-A02的第48周观察到的ALSFRS-R变化与之前的日本静脉注射（IV）RADICAVA®（依达拉奉）的第3阶段研究MCI186-19的事后评估相似[1,2]。MT-1186-A02研究没有发现新的安全问题，并加强了依达拉奉作为安全产品的作用。

The full results are included in a poster presentation at the European Network for a Cure for ALS (ENCALS) 2024 meeting taking place in Stockholm, Sweden..

在瑞典斯德哥尔摩举行的2024年欧洲ALS治愈网络（ENCALS）会议上，完整的结果包括在海报上。。

'The safety and efficacy of the on/off dosing regimen of oral edaravone in this Phase 3b post-marketing study was similar to the safety and efficacy of oral edaravone,' said Gustavo A. Suarez Zambrano, M.D., Vice President of Medical Affairs at MTPA. 'We extend our gratitude to the study participants, investigators, caregivers and clinical trial staff for completing this journey with us.'.

MTPA医疗事务副总裁Gustavo A.Suarez Zambrano医学博士说：“在这项3b期上市后研究中，口服依达拉奉开/关给药方案的安全性和有效性与口服依达拉奉的安全性和有效性相似。”我们感谢研究参与者，研究人员，护理人员和临床试验人员与我们一起完成这段旅程。”。

The MT-1186-A02 study, which was a post-marketing commitment following the FDA approval of intravenous (IV) RADICAVA® (edaravone), was designed to evaluate the superiority of an investigational once-daily dosing regimen of oral edaravone (105 mg) vs. the FDA-approved on/off dosing regimen administered in 28-day cycles, in people with ALS over 48 weeks.

MT-1186-A02研究是FDA批准静脉注射（IV）RADICAVA®（依达拉奉）后的上市后承诺，旨在评估口服依达拉奉（105 mg）每日一次给药方案与FDA批准的28天周期开/关给药方案在48周以上ALS患者中的优越性。

A pre-planned futility analysis, conducted after 50% of the planned study population (N=190) reached 48 weeks, assessed the study's primary endpoint and the probability of the study results changing if all participants completed the 48-week study period. Through that interim analysis, the Independent Data Monitoring Committee (IDMC) concluded that there is a low statistical probability for the investigational once-daily dosing regimen to show superiority to the current on/off dosing regimen as measured by the ALS Functional Rating Scale Revised (ALSFRS-R) score at study completion; therefore, study discontinuation was recommended by the IDMC..

在50%的计划研究人群（N=190）达到48周后进行的预先计划的徒劳分析评估了研究的主要终点以及如果所有参与者都完成了48周的研究期，研究结果发生变化的可能性。通过该中期分析，独立数据监测委员会（IDMC）得出结论，研究性每日一次给药方案的统计概率较低，以ALS功能评定量表修订版（ALSFRS-R）测量的当前开/关给药方案在研究完成时的得分；因此，IDMC建议停止研究。。

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). In 2024, the FDA recognized RADICAVA ORS with Orphan Drug Exclusivity based on the major contribution to patient care of the innovative oral formulation.

关于RADICAVA®（依达拉奉）和RADICAVA ORS®（依达拉奉），美国食品和药物管理局（FDA）于2017年5月5日批准了RADICAVA®（依达拉奉），并于2022年5月12日批准了口服制剂RADICAVA ORS®（依达拉奉），用于治疗肌萎缩侧索硬化症（ALS）。2024年，FDA基于创新口服制剂对患者护理的主要贡献，认可了具有孤儿药排他性的RADICAVA ORS。

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通过静脉（IV）输注以28天的周期给药。每服用60毫克需要60分钟。对于初始周期，治疗连续14天每天输注，然后是两周的无药期。此后的所有周期在14天内每天输注10天，然后是两周的无药期。

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.

RADICAVA ORS连续14天每天服用，随后在初始治疗周期中有14天的无药期。对于随后的治疗周期，RADICAVA ORS在14天内服用10天，然后是14天的无药期。应在禁食过夜后的早晨服用RADICAVA ORS。

Patients should not eat or drink (except water) within one hour after taking RADICAVA ORS.3.

患者服用RADICAVA ORS后1小时内不应进食或饮用（水除外）。

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.

依达拉奉是由三菱田边制药公司（MTPC）为ALS发现和开发的，并由三菱田边制药美国公司（MTPA）在美国商业化。MTPC集团公司于2001年开始通过13年的迭代临床平台研究ALS。2015年，依达拉奉被批准为RADICUT®，用于治疗日本和韩国的ALS。

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.

随后在加拿大（2018年10月），瑞士（2019年1月），印度尼西亚（2020年7月），泰国（2021年4月），马来西亚（2021年12月）和巴西（2024年2月）授予了营销授权。加拿大（2022年11月）和瑞士（2023年5月）批准了RADICAVA®口服混悬液的上市许可，日本于2022年12月批准了2.1%的RADICUT®口服混悬液。

To date, in the U.S., RADICAVA and RADICAVA ORS have been used to treat over 16,000 people with ALS, with over 1.9-million days of therapy, and have been prescribed by over 2,400 HCPs.4-6.

迄今为止，在美国，RADICAVA和RADICAVA ORS已被用于治疗16000多名ALS患者，治疗时间超过190万天，并已被2400多名HCP开出处方。

IMPORTANT SAFETY INFORMATION

重要安全信息

Hypersensitivity ReactionsRADICAVA (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..

超敏反应RADICAVA（依达拉奉）和RADICAVA ORS（依达拉奉）禁用于对依达拉奉或本产品的任何非活性成分有超敏反应史的患者。RADICAVA发生了超敏反应（发红，风团和多形红斑）和过敏反应（荨麻疹，血压下降和呼吸困难）。。

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.

应仔细监测患者的超敏反应。如果发生超敏反应，停用RADICAVA或RADICAVA ORS，按照标准护理进行治疗，并监测直至病情消退。

Sulfite Allergic ReactionsRADICAVA 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..

亚硫酸盐过敏反应RADICAVA和RADICAVA ORS含有亚硫酸氢钠，亚硫酸氢钠可能引起过敏性反应，包括过敏症状和易感人群的危及生命或不太严重的哮喘发作。一般人群中亚硫酸盐敏感性的总体患病率尚不清楚，但在哮喘患者中发生频率更高。。

Adverse ReactionsThe 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.

不良反应RADICAVA治疗患者报告的最常见不良反应（≥10%）为挫伤（15%），步态障碍（13%）和头痛（10%）。在一项开放标签研究中，7.6%接受放射治疗的患者也观察到疲劳。

PregnancyBased on animal data, RADICAVA and RADICAVA ORS may cause fetal harm.

怀孕根据动物数据，RADICAVA和RADICAVA ORS可能会导致胎儿伤害。

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 www.fda.gov/medwatch.

要报告疑似不良反应或产品投诉，请致电1-888-292-0058联系三菱田边制药美国公司。。

INDICATIONRADICAVA and RADICAVA ORS are indicated for the treatment of amyotrophic lateral sclerosis (ALS).

适应症RADICAVA和RADICAVA ORS适用于治疗肌萎缩侧索硬化症（ALS）。

For more information, including full Prescribing Information, please visit www.RADICAVA.com.

有关更多信息，包括完整的处方信息，请访问www.RADICAVA.com。

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.

关于位于新泽西州泽西市的三菱田边制药美国公司，三菱田边制药美国公司（MTPA）是三菱田边制药公司（MTPC）的全资子公司。它由MTPC成立，旨在开发和推进我们的管道，并在北美将经批准的药品商业化。

For more information, please visit www.mt-pharma-america.com or follow us on X (formerly Twitter), Facebook and LinkedIn..

。。

About Mitsubishi Tanabe Pharma CorporationMitsubishi 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.

关于三菱田边制药公司三菱田边制药公司（MTPC）是三菱化学集团（MCG）的制药子公司，成立于1678年，是世界上最古老的制药公司之一。MTPC总部位于日本制药业的发源地大阪市的Doshomachi。

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.

MCG已将医疗保健定位为其管理政策“打造未来”的战略重点。MTPC设定了“为所有面临疾病的人创造希望”的使命。为此，MTPC正在研究中枢神经系统、免疫炎症、糖尿病和肾脏以及癌症等疾病领域。MTPC专注于“精准医学”，以提供高治疗满意度的药物，并致力于开发“围绕药丸的解决方案”，以解决基于治疗医学的特定患者问题，包括预防疾病，症状前疾病护理，预防恶化和预后。

For more information, go to https://www.mt-pharma.co.jp/e/..

有关更多信息，请访问https://www.mt-pharma.co.jp/e/..

Media inquiries:Media_MTPA@mt-pharma-us.com

媒体查询：Media_MTPA@mt-pharma-us.com

1 RADICAVA and RADICAVA ORS Prescribing Information. Jersey City, NJ: Mitsubishi Tanabe Pharma America, Inc.; 2022.2 Edaravone (MCI-186) ALS 19 Study Group. Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial.

1 RADICAVA和RADICAVA ORS规定信息。新泽西州泽西市：三菱田边制药美国公司。；2022.2依达拉奉（MCI-186）ALS 19研究组。依达拉奉在明确定义的肌萎缩侧索硬化症患者中的安全性和有效性：一项随机，双盲，安慰剂对照试验。

Lancet Neurol. 2017;16(7):505-512.3 RADICAVA and RADICAVA ORS Prescribing Information. Jersey City, NJ: Mitsubishi Tanabe Pharma America, Inc.; 2022.4 Data on file. Mitsubishi Tanabe Pharma America, Inc.5 Data on file. Mitsubishi Tanabe Pharma America, Inc.6 Data on file. Mitsubishi Tanabe Pharma America, Inc..

柳叶刀神经学。2017年；16（7）：505-512.3 RADICAVA和RADICAVA ORS规定信息。新泽西州泽西市：三菱田边制药美国公司。；2022.4存档数据。Mitsubishi Tanabe Pharma America，Inc.5存档数据。Mitsubishi Tanabe Pharma America，Inc.6存档数据。三菱田边制药美国公司。。

SOURCE Mitsubishi Tanabe Pharma America

来源：三菱田边制药（美国）