Recommendation based on a Phase 3 trial showing a significantly greater proportion of children on Dupixent achieved histological remission compared to placebo, consistent with improvements seen in adults and adolescents If approved, Dupixent would be the first and only medicine in the EU indicated for EoE in this age group Sept. 20, 2024 --- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the European Medicines Agency’s Committee for Medicinal Products for Human U

Sept. 20, 2024 -- Firefly Neuroscience, Inc. (“Firefly,” “we,” or the “Company”) (NASDAQ: AIFF), an Artificial Intelligence (“AI”) company developing innovative solutions that improve brain health outcomes for patients with neurological and mental disorders, today announced advancing its artificial intelligence-powered Brain Network Analytics (BNA™) technology after successful research collaborations with Takeda Pharmaceutical Company Limited (“Takeda”) and Novartis, two global leaders in pharm

Sept. 23, 2024 -- Samsung Bioepis Co., Ltd. today presented the “totality-of-the-evidence” supporting extrapolation of atypical hemolytic uremic syndrome (aHUS) indication on EPYSQLI™ (SB12), a biosimilar to Soliris1 (eculizumab), at the 56th European Society for Paediatric Nephrology (ESPN 2024). The congress will be taking place from September 24 to 27, 2024 in Valencia, Spain. SB12, a biosimilar to reference eculizumab (ECU-RP), was approved by the European Commission (EC) in May 2023 for t

上周（9月16日~9月22日），全球生物制药领域有至少4家致力于创新药研发的公司宣布完成新一轮融资。这些最新获得资本市场青睐的新锐公司主要致力于神经系统疾病小分子疗法、癌症细胞疗法、癌症疫苗、新一代干细胞疗法等领域。 9月19日，GC Therapeutics宣布成立，并已完成6500万美元的A轮融资，由Cormorant Asset Management领投，Mubadala Capital、Andreessen Horowitz （a16z） Bio + Health、Medical Excellence Capital、Cercano Management和Pear VC参投，使该公司的总筹资金额达到7500万美元。 GC Therapeutics致力于推进并解锁下一代细胞疗法。该公司的诱导多能干细胞（iPSC）编程平台TFome（转录因子组）由哈佛医学院和Wyss研究所的教授George Church博士的实验室开发，该平台旨在通过一步式工艺以比传统方法快100倍的速度提供基于iPSC的现货型细胞疗法，以提高效力、效率、质量并降低成本，从而彻底改变细胞疗法。该公司最初的管线

RIM-TD open-label extension (OLE) study showed long-term improvement of Tardive Dyskinesia (TD) symptoms from treatment with deutetrabenazine over three years in European patients1 TD is an involuntary movement disorder that develops in around 15%-25% of patients taking antipsychotic medications for conditions such as schizophrenia, bipolar disorder, and major depressive disorder2,3 Data presented at the European College of Neuropsychopharmacology Congress (ECNP) in Milan 21-24 September 2024 Se

9月20日，华东医药宣布其全资子公司华东医药（杭州）有限公司与四环医药控股集团有限公司旗下非全资附属公司惠升生物制药股份有限公司，就其已上市创新产品惠优静（脯氨酸加格列净片）在中国大陆的商业化权益达成独家战略合作。 惠优静是惠升生物在中国获批上市的治疗2型糖尿病的SGLT-2抑制剂。根据协议条款，华东医药将负责惠优静在中国大陆的商业化市场推广，惠升生物作为MAH持有人将负责惠优静的注册、临床开发、生产和供应。 惠优静（脯氨酸加格列净片）是一种SGLT-2抑制剂，通过不依赖胰岛素的降糖机制，减少肾小管对葡萄糖的重吸收，从而增加尿糖排泄，达到降低血糖水平的效果，为惠升生物具有自主知识产权的1类新药，其独特的分子结构确保了出色的疗效和优秀的安全性。惠优静已经获得中国、美国、欧洲、日本、韩国、香港等多个国家和地区专利权。2024年1月19日，惠优静获得国家药品监督管理局（NMPA）正式批准上市，用于单药治疗或与盐酸二甲双胍联合使用，改善成人2型糖尿病患者的血糖控制。 内容来源于网络，如有侵权，请联系删除。

美国FDA宣布批准Zevra Therapeutics旗下口服疗法Miplyffa（arimoclomol）与酶抑制剂miglustat联合使用，以治疗成人和2岁及以上儿童的C型尼曼匹克病（NPC）相关神经系统症状。根据新闻稿，arimoclomol是美国FDA批准的首个NPC药物。 NPC是一种极为罕见、不可逆转的神经退行性溶酶体贮积病，其特点是胆固醇在细胞内的转运出现缺陷，胆固醇不能正常地离开内吞途径进入内质网从而积聚在溶酶体内，最终导致这些物质在包括脑组织在内的各种组织中积累。该疾病由NPC1或NPC2基因的突变所引起，这些基因负责制造溶酶体蛋白。患者的主要神经损伤表现为语言、认知、吞咽、行走和精细运动技能方面的障碍。 一项为期12个月的随机、双盲、安慰剂对照试验评估了arimoclomol的安全性和有效性，试验对象为2至19岁、经分子生物学检验确诊为NPC的患者。50例患者按2：1的比例随机接受根据体重调整的arimoclomol（31至124毫克）或安慰剂治疗，每日口服三次。在这50例患者中，39名（78%）在试验中接受了miglustat作为基础治疗。 R4DNPCC

Sept. 20, 2024 -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has confirmed its June 2024 negative opinion on the marketing authorization application of intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration. “We are deeply disappointed by this outcome, which leaves millions of Europeans with GA without a treatment

Sept. 19, 2024 -- ACELYRIN, INC. (Nasdaq: SLRN), a late-stage clinical biopharma company focused on accelerating the development and delivery of transformative medicines in immunology, today announced that positive results from the company’s global Phase 3 clinical trial of izokibep in moderate-to-severe hidradenitis suppurativa (HS) patients will be shared as a late-breaking oral presentation at the 2024 European Academy of Dermatology and Venereology taking place September 25-28, 2024 in Amst

MVA-BN approved for use against mpox and smallpox in adolescents 12-17 years of age after expedited review with EMA Represents the second EMA approval of an MVA-BN-based vaccine for a younger population COPENHAGEN, Denmark, September 19, 2024 – Bavarian Nordic A/S (OMX: BAVA) announced today that the European Commission has adopted the Committee for Medicinal Products for Human Use (CHMP) recommendation for the approval of a type II variation for IMVANEX® (MVA-BN) smallpox and mpox vaccine, exte