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.
催化製藥公司（"Catalyst"或"公司"）（納斯達克股票代碼：CPRX），一家專注於入許可、開發和商業化罕見疾病患者的新藥的商業階段生物製藥公司，今日宣佈其在日本的子許可方DyDo藥業有限公司（"DyDo"）報道日本厚生勞動省已批准DyDo的新藥申請（"NDA"），用於商業化FIRDAPSE（阿米氨喹）片10毫克，以治療日本的蘭伯頓-伊頓肌無力綜合症（"LEMS"）。該批准標誌着LEMS治療的重要進展，LEMS是一種罕見的自身免疫性疾病，嚴重影響生活質量，對受到LEMS影響的日本患者未滿足的需求構成了顯著的進步。
"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."
"我們很高興我們的合作伙伴DyDo在日本取得了FIRDAPSE的監管批准。這代表着一個重要的里程碑，爲患者帶來了新的希望，並進一步肯定了FIRDAPSE在LEMS治療中的有效性，"Catalyst製藥公司總裁兼首席執行官理查德·J·戴利表示,"這一成就強調了我們對推進患者護理的堅定承諾。我們感謝合作伙伴在獲得這一批准方面的協作努力，我們專注於將我們創新的罕見病產品組合擴展到美國境外，對全球患者的生活產生有意義的影響。"
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）是美國批准用於成人和六歲及以上兒童LEMS治療的主要療法。LEMS是一種罕見的自身免疫性疾病，特徵是肌肉無力和疲勞。約50%的LEMS患者患有潛在癌症，LEMS大約出現在約3%的小細胞肺癌患者中。FIRDAPSE是唯一獲得美國FDA批准的、基於證據的LEMS治療。作爲催化製藥公司致力於服務罕見疾病患者的支柱，FIRDAPSE在美國有一項全面的患者支持計劃，以幫助確保符合條件的美國患者的可及性和援助。
譯文內容由第三人軟體翻譯。
