HUTCHMED索乐匹尼获FDA孤儿药资格认定：治原发性免疫性血小板减少症，获批后可享7年美国市场独占期
HUTCHMED（和黄医药，Nasdaq/AIM：HCM；港交所：13）10 月 28 日宣布，美国食品药品监管局（FDA）已授予其在研药物索乐匹尼（sovleplenib）孤儿药资格认定，用于治疗原发性免疫性血小板减少症（ITP）——一种可使出血风险升高的自身免疫性疾病。
索乐匹尼是一种新型、选择性、每日一次口服的脾酪氨酸激酶（Syk）抑制剂。Syk 是 B 细胞受体与 Fc 受体信号通路的主要组成部分，也是多个 B 细胞淋巴瘤亚型与自身免疫性疾病的既定治疗靶点；HUTCHMED 目前保留索乐匹尼的全球全部权利。
FDA 的孤儿药资格认定面向在美国患者不足 20 万人的罕见病药物与生物制品。获得认定的药物若日后获 FDA 批准上市，可享有 7 年美国市场独占期，并可获得合格临床试验的税收抵免、处方药使用者费用法案（PDUFA）费用减免，以及 FDA 在临床开发期间的专门监管指导。
原发性 ITP 的特点是血小板遭到免疫性破坏、生成减少，患者容易出现过度出血与瘀伤，也常伴有疲劳与生活质量下降。公司引用的流行病学数据显示，成人原发性 ITP 发病率约为每年每 10 万人 3.3 例、患病率约为每 10 万人 9.5 例。
在中国，索乐匹尼用于原发性 ITP 与温抗体型自身免疫性溶血性贫血的新药上市申请正由国家药品监督管理局审评，分别有关键性 ESLIM-01 与 ESLIM-02 试验的积极数据支持。公司同时提醒，孤儿药资格认定并不等同于获批上市。
HUTCHMED's Sovleplenib Wins FDA Orphan Drug Designation for Primary Immune Thrombocytopenia
HUTCHMED (China) Limited (Nasdaq/AIM: HCM; HKEX: 13) announced on October 28 that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its investigational drug sovleplenib for the treatment of primary immune thrombocytopenia (ITP), an autoimmune disorder that can lead to an increased risk of bleeding.
Sovleplenib is a novel, selective, once-daily oral inhibitor of spleen tyrosine kinase (Syk), a major component in B-cell receptor and Fc receptor signaling and an established target for multiple subtypes of B-cell lymphomas and autoimmune disorders. HUTCHMED currently retains all worldwide rights to sovleplenib.
The FDA's Orphan Drug Designation program covers drugs and biologics for rare diseases affecting fewer than 200,000 people in the United States. If approved for the designated indication, a designated drug becomes eligible for seven years of U.S. market exclusivity upon approval, tax credits for qualified clinical trials, user fee waivers under the Prescription Drug User Fee Act, and specialized regulatory guidance from the FDA during clinical development.
Primary ITP is characterized by immunologic destruction of platelets and decreased platelet production, leaving patients at increased risk of excessive bleeding and bruising, and is also associated with fatigue and impaired quality of life. The company cited an adult incidence of 3.3 per 100,000 adults per year and a prevalence of 9.5 per 100,000 adults.
In China, New Drug Applications for sovleplenib in primary ITP and warm antibody autoimmune hemolytic anemia are under review by the National Medical Products Administration, supported by positive data from the pivotal ESLIM-01 and ESLIM-02 trials, respectively. The company noted that the designation is not itself an approval.
来源：GSM·美国（原文链接）
图片来源：图：显微镜下的红细胞、血小板与白细胞（中性粒细胞）。照片：Ajay Kumar Chaurasiya，CC BY-SA 4.0，经 Wikimedia Commons。 / Photo: Red blood cells, platelets and a white blood cell under the microscope. Photo by Ajay Kumar Chaurasiya, CC BY-SA 4.0, via Wikimedia Commons.
