Sanofi’s SAR446268 earns US fast track designation for the treatment of non-congenital myotonic dystrophy type 1 Designation earned for one-time AAV gene therapy SAR446268, designed to silence DMPK expressionMyotonic dystrophy type 1 (DM1) is a rare, genetic disorder that causes progressive muscle weakness and wasting, with no currently approved medicines Paris, September 23, 2025. The US Food and Drug Administration (FDA) has granted fast track designation to SAR446268, Sanofi's one-time AAV gene therapy f ...

Key Takeaways CHMP recommends EU approval of Dupixent for chronic spontaneous urticaria in patients 12 and above.Phase III studies showed Dupixent reduced itch, hives and improved disease control.Dupixent sales hit EUR 7.3B in H1 2025, with Sanofi projecting about EUR 22B in sales by 2030.Sanofi (SNY) and partner Regeneron (REGN) announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending the approval of Dupixent (dupilumab) in ...

Key Takeaways SNY's NDA for tolebrutinib in nrSPMS is under FDA priority review with a decision pushed to Dec. 28, 2025.Extension follows submission of new analyses, deemed a major amendment to SNY's application by the FDA.In phase III studies, tolebrutinib delayed confirmed disability progression versus placebo and Aubagio.Sanofi (SNY) announced that the FDA has extended the target action date for its new drug application (NDA) for tolebrutinib by three months. The NDA is seeking approval of tolebrutinib, ...

一场席卷英国的生命科学版图的暴风雨，仍在呼啸着。 9月10日，默沙东宣布关闭伦敦药物研发中心。这已不再是缩减规模或暂缓投资，而是一次彻底的清 场。所有研发业务转移至美国，只留下空壳般的注册存在。 紧随其后，礼来宣布叫停原定在英国的生物技术孵化器项目，声称要"等待生命科学环境更加明朗"。 这还不够，当本土巨头阿斯利康也加入队列，冻结了剑桥研究中心的2亿英镑投资时，信号已异常清 晰：英国正在愈发远离全球制药的舞台中心。 9月15日，法国赛诺菲也公开表态，将对英国采取类似立场，加入撤退阵营。 而这场风暴，或许远未结束…… MNC被迫出逃 站在MNC角度，这波撤退绝非主动选择，而是在英国层层叠叠的税收重压下的被迫出逃。 长期以来，药企在英国开展研发和商业活动，需要面对公司税、研发税以及药品销售返款等多重税收负 担。 提起英国NHS，大概是很多英国人颇为自豪的一项公共事业，常常被视为"免费医疗"的典范。然而，在 老龄化加剧、资金紧张重重困境下，NHS的动作正在变形，压力进一步给到药企身上。 根据英国法定定价方案和"品牌药定价、获取与增长自愿计划"（VPAG），药企必须将一定比例的销售 额返还英国国家医疗服务体系NHS， ...

Update on the US regulatory review of tolebrutinib in non-relapsing, secondary progressive multiple sclerosis Paris, September 22, 2025. The US Food and Drug Administration (FDA) has extended by three months the target action date of its review of the new drug application (NDA) of tolebrutinib, an oral and brain-penetrant investigational Bruton's tyrosine kinase (BTK) inhibitor to treat non-relapsing, secondary progressive multiple sclerosis (nrSPMS) and to slow disability accumulation independent of relaps ...

监管进展 - 欧洲药品管理局人用药品委员会采纳积极意见 推荐批准Dupixent用于治疗慢性自发性荨麻疹 覆盖12岁及以上中重度患者群体[1] - 最终批准决定预计在未来几个月内做出[1] - 若获批 Dupixent将成为欧盟十余年来首个针对慢性自发性荨麻疹的靶向药物[10] 临床试验数据 - 支持意见的数据来自LIBERTY-CUPID三期项目中的两项研究(NCT04180488 A研究和C研究)[2] - 研究显示Dupixent在24周时相比安慰剂显著减少瘙痒和荨麻疹症状[2][10] - 项目中的B研究在不同患者群体中提供了额外安全性数据[2] - 安全性结果与已知安全性特征基本一致 成人及青少年患者中发生率≥5%的不良事件包括注射部位反应、COVID-19感染、高血压、慢性自发性荨麻疹和意外过量[3] 产品特性与适应症 - Dupixent是全人源单克隆抗体 通过抑制白细胞介素4和白细胞介素13信号通路发挥作用 非免疫抑制剂[6] - 目前已在包括日本和美国在内的多个国家获批用于特定成人和青少年慢性自发性荨麻疹患者[4] - 全球超过60个国家批准用于一种或多种适应症 包括特应性皮炎、哮喘、慢性鼻窦炎伴鼻息肉、嗜酸性食管炎、结节性痒疹、慢性自发性荨麻疹、慢性阻塞性肺病和大疱性类天疱疮[7] - 全球正在接受治疗的患者超过100万人[7] 疾病背景 - 慢性自发性荨麻疹是慢性炎症性皮肤疾病 部分由2型炎症驱动 导致突发性虚弱性荨麻疹和反复发作的瘙痒[5] - 标准治疗为靶向H1受体的抗组胺药 但许多患者症状仍无法控制[5] 研发合作与管线 - Dupilumab由赛诺菲和再生元根据全球合作协议共同开发[8] - 迄今已在60多项临床研究中涉及超过10,000名患有2型炎症相关慢性疾病的患者[8] - 除已批准适应症外 正在进行三期研究的疾病包括未知原因慢性瘙痒和慢性单纯性苔藓[9] 公司背景 - 再生元(NASDAQ: REGN)是领先的生物技术公司 专注于为严重疾病患者开发变革性药物[11] - 利用VelociSuite等专有技术生产优化全人源抗体和新型双特异性抗体[12] - 赛诺菲(EURONEXT: SAN, NASDAQ: SNY)是研发驱动型生物制药公司 专注于免疫系统相关药物和疫苗开发[14][15]

Sanofi and Regeneron’s Dupixent to treat chronic spontaneous urticaria advances in EU with positive CHMP opinion Recommendation for adults and adolescents based on phase 3 studies showing Dupixent significantly reduced itch and hives at 24 weeks compared to placebo If approved, Dupixent would be the first targeted medicine in over a decade indicated for CSU in the EU Paris and Tarrytown, September 22, 2025. The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a p ...

Recommendation for adults and adolescents based on Phase 3 trials showing Dupixent significantly reduced itch and hives at 24 weeks compared to placebo If approved, Dupixent would be the first targeted medicine in over a decade indicated for CSU in the European Union (EU) TARRYTOWN, N.Y. and PARIS, Sept. 22, 2025 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted ...

糖尿病是一种慢性健康状况，以长期高血糖为特征的代谢紊乱。它影响了全世界人群的很大比例。 久坐的生活方式以及不健康的饮食，增加了糖尿病的患病率。根据国际糖尿病联合会（IDF）提供的最 新数据，2021年，有5.37亿年龄在20岁-79岁的成年人被诊断出患有糖尿病。据估计，到2030年，这个 数字将达到6.43亿。 糖尿病的越来越流行，增加了对治疗药物的需求。全球糖尿病药物市场规模在2024年的价值为883.2亿 美元，预计该市场将从2025年的10014.8亿美元，增长到2032年的2338.4亿美元，在预测期间的复合年增 长率为12.7％。 这进一步激发了药企对治疗糖尿病药物的开发。2025年糖尿病药物市场，可谓暗流涌动，GLP-1类药物 持续领跑，SGLT2抑制剂竞争白热化，胰岛素赛道加速洗牌。 No.1 礼来 自2025年10月1日起，礼来中国将免疫事业部与抗肿瘤事业部合并为"免疫与肿瘤事业部"，由现任免疫 事业部副总裁忻晨芸女士担任新部门负责人。同时，抗肿瘤事业部副总裁尹航先生决定于9月30日离 职，寻求新的职业发展机会。 据媒体报道，诺和诺德面向全球的公告发布两天后，诺和诺德中国迅速跟进。流出的内部邮 ...

Ariel Investments, an investment management company, released its “Ariel Global Fund” second-quarter 2025 investor letter. A copy of the letter can be downloaded here. The second quarter marked a period of extremes. The stocks fell after the “Liberation Day” tariff announcement in early April and rebounded following the pause in tariff implementation. Enthusiasm for artificial intelligence (AI) themed stocks and robust corporate earnings results lifted global and U.S. indices to new highs. Against this back ...