Catalyst Pharma's FIRDAPSE Approved in Japan for Rare Autoimmune Disorder Lambert-Eaton Myasthenic Syndrome ("LEMS")
Catalyst Pharma's FIRDAPSE Approved in Japan for Rare Autoimmune Disorder Lambert-Eaton Myasthenic Syndrome ("LEMS")
Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (NASDAQ:CPRX), a commercial-stage biopharmaceutical company focused on in-licensing, developing, and commercializing novel medicines for patients living with rare diseases, today announced that its sub-licensee in Japan, DyDo Pharma, Inc., ("DyDo") has reported that the Ministry of Health, Labor and Welfare of Japan has approved DyDo's New Drug Application ("NDA") to commercialize FIRDAPSE (amifampridine) Tablets 10 mg for treatment of patients with Lambert-Eaton Myasthenic Syndrome ("LEMS") in Japan. This approval marks a pivotal advancement in the treatment of LEMS, a rare autoimmune disorder that can severely impact quality of life and represents a significant step forward in addressing the unmet needs of patients affected by LEMS in Japan.
催化制药公司（"Catalyst"或"公司"）（纳斯达克股票代码：CPRX），一家专注于入许可、开发和商业化罕见疾病患者的新药的商业阶段生物制药公司，今日宣布其在日本的子许可方DyDo药业有限公司（"DyDo"）报道日本厚生劳动省已批准DyDo的新药申请（"NDA"），用于商业化FIRDAPSE（阿米氨喹）片10毫克，以治疗日本的兰伯顿-伊顿肌无力综合症（"LEMS"）。该批准标志着LEMS治疗的重要进展，LEMS是一种罕见的自身免疫性疾病，严重影响生活质量，对受到LEMS影响的日本患者未满足的需求构成了显著的进步。
"We are pleased that our partner, DyDo, has secured regulatory approval for FIRDAPSE in Japan. This represents a meaningful milestone, bringing renewed hope to patients and further affirming FIRDAPSE's proven effectiveness in the treatment of LEMS," said Richard J. Daly, Catalyst's President and Chief Executive Officer. "This achievement underscores our unwavering commitment to advancing patient care. We appreciate the collaborative efforts of our partner in securing this approval, and we remain focused on expanding our innovative rare disease product portfolio beyond the U.S. to make a meaningful impact on patients' lives worldwide."
"我们很高兴我们的合作伙伴DyDo在日本取得了FIRDAPSE的监管批准。这代表着一个重要的里程碑，为患者带来了新的希望，并进一步肯定了FIRDAPSE在LEMS治疗中的有效性，"Catalyst制药公司总裁兼首席执行官理查德·J·戴利表示,"这一成就强调了我们对推进患者护理的坚定承诺。我们感谢合作伙伴在获得这一批准方面的协作努力，我们专注于将我们创新的罕见病产品组合扩展到美国境外，对全球患者的生活产生有意义的影响。"
FIRDAPSE (amifampridine) is a leading therapy indicated in the United States for the treatment of LEMS in adults and pediatric patients six years of age and older. LEMS is a rare autoimmune disorder characterized by muscle weakness and fatigue. About 50% of people with LEMS have underlying cancer, as LEMS is observed in approximately 3% of small-cell lung cancer patients. FIRDAPSE is the only U.S. FDA-approved, evidence-based treatment for LEMS. As a cornerstone of Catalyst's commitment to serving those with rare diseases, FIRDAPSE is supported in the U.S. by a comprehensive patient support program to help ensure accessibility and assistance for eligible U.S. patients.
FIRDAPSE（阿米氨喹）是美国批准用于成人和六岁及以上儿童LEMS治疗的主要疗法。LEMS是一种罕见的自身免疫性疾病，特征是肌肉无力和疲劳。约50%的LEMS患者患有潜在癌症，LEMS大约出现在约3%的小细胞肺癌患者中。FIRDAPSE是唯一获得美国FDA批准的、基于证据的LEMS治疗。作为催化制药公司致力于服务罕见疾病患者的支柱，FIRDAPSE在美国有一项全面的患者支持计划，以帮助确保符合条件的美国患者的可及性和援助。
