罕见病疗法

Search documents
These Analysts Increase Their Forecasts On PTC Therapeutics
Benzinga· 2025-07-29 15:23
PTC Therapeutics, Inc. PTCT announced it has gained FDA approval for SEPHIENCE on Monday. "We are excited to have reached this important milestone for those affected by PKU," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "The broad labeling reflects the potential of Sephience to meet the significant unmet need of PKU patients. The Sephience clinical data along with our expertise in launching rare disease therapies position Sephience to become the future standard of care. Our expe ...
潜在总金额95亿美元 赛诺菲收购罕见病疗法公司Blueprint
Jing Ji Guan Cha Bao· 2025-06-03 02:02
(原标题:潜在总金额95亿美元 赛诺菲收购罕见病疗法公司Blueprint) 经济观察网讯 据赛诺菲官网消息,当地时间6月2日,赛诺菲与总部位于美国的上市生物制药公司 Blueprint Medicines Corporation(Blueprint)达成协议,赛诺菲将收购专注于系统性肥大细胞增多症 (SM,一种罕见免疫疾病)及其他 KIT 驱动疾病的 Blueprint。 此次收购包括一款罕见免疫疾病药物 Ayvakit/Ayvakyt(avapritinib),已获美国和欧盟批准,以及一条 有前景的晚期和早期免疫学研发管线。此外,Blueprint 在过敏科、皮肤科和免疫学领域的既有影响力 预计将增强赛诺菲不断壮大的免疫学研发管线。 根据收购条款,赛诺菲将在交割时以每股129.00 美元现金支付,代表约91亿美元的股权价值。Blueprint 股东还将获得一份不可交易的或有价值权(CVR),该权利使持有人有权分别因 BLU-808 未来开发和 监管里程碑的达成而获得每份 CVR 2美元和4美元的两笔潜在里程碑付款。包括潜在CVR付款在内的交 易总股权价值,按完全稀释计算约为95亿美元。 Ayvakit 在 ...
赛诺菲斥资超90亿美元收购罕见病疗法公司,创欧洲药企今年收购规模之最
Di Yi Cai Jing· 2025-06-02 09:54
Core Viewpoint - Sanofi has announced a $9.5 billion acquisition of Blueprint Medicines, marking the largest healthcare acquisition by a European pharmaceutical company this year, aimed at expanding its portfolio in rare immune diseases with the drug avapritinib [1][2]. Group 1: Acquisition Details - The acquisition includes avapritinib, the only approved drug globally for advanced systemic mastocytosis, which is a rare immune disease characterized by abnormal mast cell accumulation [1]. - Blueprint's stock surged over 26% following the announcement, while its Chinese partner, Basilea Pharmaceutica, saw a nearly 5% increase in share price [1]. - The deal is expected to enhance Sanofi's research pipeline in rare immune diseases [1]. Group 2: Financial Performance - Avapritinib generated $479 million in net revenue for Sanofi in 2024, with nearly $150 million in revenue in Q1 2025, reflecting a year-over-year growth of over 60% [2]. - In China, avapritinib has been approved and recommended as a first-line treatment in the first "Systemic Mastocytosis Diagnosis and Treatment Guidelines" [2]. Group 3: Future Prospects - The acquisition will also provide Sanofi with the next-generation systemic mastocytosis drug elenestinib and the highly selective oral wild-type KIT inhibitor BLU-808, which has potential applications in various immune diseases [2]. - Sanofi's CEO Paul Hudson indicated that this acquisition complements recent purchases of early-stage drugs and that the company retains significant capacity for further acquisitions [2].