CHIESI Farmaceutici SpA

注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需要得到授权。医药圈的4月，迎来一波首家过评药品的“小高潮”，这些药品不仅突破技术壁垒，更有望改写市场格局。根据摩熵医药数据库，本月倍特药业、福元医药、立方制药等22家企业的24款药品（按批文号计）首家过评，共23个品种。014月首家过评药品清单截图来源：摩熵医药数据库，整理自摩熵咨询02单品分析01熊去氧胆酸口服混悬液4月8日，成都赛璟生物研发的熊去氧胆酸口服混悬液作为国内首仿3.1类新药，正式获批上市。该药品是国内企业首个且唯一获批治疗罕见病囊性纤维化相关肝病（CFALD）的药物，填补了国内目前上市的熊去氧胆酸片剂/胶囊剂无此适应症的空白。熊去氧胆酸是一种外源性胆酸药物，通过补充机体缺乏的胆酸，从而调节胆固醇代谢和胆汁的胆碱酸（胆酸的代谢产物）浓度，改善胆汁分泌功能。它主要通过在肝脏中增加胆汁酸的合成和排泄，促进胆盐的肠道循环，从而降低胆固醇的吸收。据摩熵医药-销售数据库显示，2023年熊去氧胆酸全国医院端销售额达25.84亿元，由于纳入集采原因，2024年销售额受到较大影响，2024上半年销售额8.84亿元。截图来源：摩熵医药全国医院（全终端）销售数据库成都赛璟生物获批的熊去氧胆酸口服混悬液，相较于传统的片剂和胶囊剂，更符合儿童生理特征，适用于儿童和吞咽困难的患者，为临床用药提供了更佳的选择。02吸入用丙酸倍氯米松混悬液4月8日，江西艾施特制药的哮喘治疗用药吸入用丙酸倍氯米松混悬液获批上市，该药由济民可信集团创新技术药物研究院研发，江西艾施特制药有限公司落地生产。研究表明，吸入性糖皮质激素可显著降低急性发作风险，当前已成为哮喘长期控制的核心药物。吸入用丙酸倍氯米松混悬液作为国际公认的高效局部抗炎糖皮质激素（ICS），其原研药由意大利凯西制药开发，通过雾化吸入直接作用于气道，可有效控制炎症、缓解症状，并减少全身性激素副作用。哮喘是一种常见的慢性炎症性气道疾病，症状严重时可出现呼吸困难和低氧血症，临床亟需高效、安全的吸入制剂以改善患者预后。据摩熵医药-销售数据库显示，阻塞性气管疾病用药2023年全国医院端销售额达233.12亿元，2024上半年销售额119.95亿元。截图来源：摩熵医药全国医院（全终端）销售数据库此次江西艾施特制药的吸入用丙酸倍氯米松混悬液国内首仿上市，将为国内哮喘患者提供高效、安全的治疗新选择，为基层医疗机构和儿童患者提供更便捷的治疗方案，并有助于降低我国对哮喘药物的进口依赖。END本文为原创文章，转载请留言获取授权近期热门资源获取数据透视：中药创新药、经典验方、改良型新药、同名同方的申报、获批、销售情况-2025042023H2-2024H1中国药品分析报告-2025042024年中国1类新药靶点白皮书-202503中国AI医疗健康企业创新发展百强榜单-202502解码护肤抗衰：消费偏好洞察与市场格局分析-2025022024年FDA批准上市的新药分析报告-2025012024年NMPA批准上市的新药分析报告-2025012024年医保谈判及市场分析报告-2025.012024年中国医疗健康投融资全景洞察报告-202501小分子化药白皮书（上）-2025012024医美注射材料市场发展分析报告-202412中国放射性药物产业白皮书-202410近期更多摩熵咨询热门报告，识别下方二维码领取联系我们，体验摩熵医药更多专业服务会议合作园区服务数据库咨询定制服务媒体合作点击上方图片，即可开启摩熵化学数据查询点击阅读原文，申请摩熵医药企业版免费试用！

The funding will be used to advance GLM101 into a Phase IIb trial: Credit: Malte Mueller via Getty Images. Glycomine has raised $115m in Series C financing to advance its lead investigational therapy, GLM101, into a Phase IIb clinical trial for an ultra-rare disorder. GLM101 is being developed as a treatment for phosphomannomutase-2 congenital disorder of glycosylation (PMM2-CDG), a rare and life-threatening genetic disorder with no approved treatments. Glycomine is currently conducting an open-label Phase II clinical trial (NCT06657859) at sites in Europe and the US, with 20 patients enrolled to date. The company recently initiated dosing in paediatric patients. Interim results from nine adult and adolescent patients showed an average 11.9-point improvement over 24 weeks on the international cooperative ataxia rating scale, indicating potential clinical benefit in one of the most debilitating features of the disease. The candidate is a mannose-1-phosphate replacement therapy aimed at correcting the metabolic deficiency caused by mutations in the PMM2 gene. These mutations impair the synthesis of N-linked glycans, which are critical for the normal function of proteins and lipids in the body. The resulting condition manifests with a wide range of symptoms, like ataxia (poor muscle control), developmental delays, vision loss, and multi-organ involvement. Current management is limited to supportive care such as physiotherapy, assistive equipment, and symptom-targeting interventions. The latest investment builds on the company’s previous $35m raise in June 2021, which supported its Phase I programme for GLM101. In the 16 April announcement, Glycomine’s CEO Steve Axon said: “This financing will enable us to advance GLM101 into a randomised, placebo-controlled trial later this year – an important step toward bringing the first disease-modifying therapeutic to patients with PMM2-CDG.” CTI Life Sciences Fund and Advent Life Sciences co-led the funding round, with participation from Novo Holdings, Sanofi Ventures, Abingworth, RiverVest Venture Partners, Sanderling Ventures, Chiesi Ventures, Remiges Ventures, and Asahi Kasei Ventures. In parallel to Glycomine’s programme, a separate effort is underway to assess the potential of a repurposed therapy for PMM2-CDG. Maggie’s Pearl – a joint venture between the rare disease biotech Perlara, the non-profit organisation Maggie’s Cure, and Mayo Clinic – is sponsoring a Phase III trial of oral epalrestat (NCT04925960) in 40 paediatric patients. The US Food and Drug Administration (FDA) cleared the Phase III study in December 2021, and the study is now fully enrolled and expected to complete by YE. Epalrestat is an aldose reductase inhibitor approved in Japan for the treatment of diabetic neuropathy. This recent update comes amid growing uncertainty around the future of incentives for rare disease drug development in the US. In particular, the potential discontinuation of the FDA’s paediatric priority review voucher (PRV) programme is raising concerns across the sector.

Roche devastated the Angelman syndrome community in 2023, when it ended clinical development of rugonersen, a treatment candidate for the rare neurodevelopmental condition. But after losing a potential acquirer for the drug last year, the Swiss pharma giant has found new life for the drug. Oak Hill Bio is picking up rugonersen and aims to dose the first patient in a Phase 3 trial in the first quarter of 2026, the small biotech exclusively told Endpoints News . Oak Hill and Roche declined to disclose terms of the deal. “This is a positive step forward for patients living with Angelman syndrome and their families,” a Roche spokesperson said in an emailed statement. Angelman leads to severe developmental delays, leaving many unable to speak. The rare condition is thought to impact about 500,000 people worldwide, and is the result of a mutation in a gene inherited from a person’s mother. With rugonersen, an antisense oligonucleotide, Roche hoped to boost the paternal version of the gene, called UBE3A, in the central nervous system. But the pharma company stopped development of the treatment in early 2023 because it “did not meet Roche’s internal criteria.” At least 10 other Angelman treatment candidates have been stopped in recent years. Roche continues to develop a different type of drug, a small molecule called alogabat, for the condition. “When we looked at the data,” Oak Hill CEO Josh Distler said in an interview, “we concluded very rapidly that there was really promising data and it was desperately needed.” The Cambridge, MA-based biotech will be at least a few quarters behind Ionis and Ultragenyx in the development of an antisense oligonucleotide for Angelman. Ultragenyx dosed the first patient with its candidate, called GTX-102, in December, and Ionis plans to start its own Phase 3 for its candidate ION582 in May, according to the study entry in the federal trials database. “We benefit from a couple companies that have gone before us. We’ve been able to look at what they’ve done, and there’s some advantages of not being the first mover,” Distler said. The company is working with clinical investigators and patient groups and will share its Phase 3 design later this year, Distler said. Late-stage development may be a tall task for Oak Hill, which has said little about its funding to date. The company emerged in 2022 with rare disease drug candidates from Takeda . The company is backed by a family office, but Distler declined to disclose the investor’s name. Oak Hill is also working off of money it’s receiving as part of a license and development agreement with Chiesi for an experimental drug for complications of extremely premature birth. The partners didn’t disclose terms of that January 2024 deal.