Triheptanoin（Dojolvi）获批用于治疗患有一组罕见遗传性疾病的成人和儿童
正文 · 中文译文
新闻稿 Triheptanoin（Dojolvi）获批用于治疗患有一组罕见遗传性疾病的成人和儿童 MHRA批准triheptanoin用于治疗患有一组罕见遗传性疾病的成人和儿科患者。 来源： 药品和医疗产品监管局 发布日期： 2026年10月30日 药品和医疗产品监管局（MHRA）已于今日，2026年10月30日，批准triheptanoin（Dojolvi）的口服液体制剂，用于治疗患有长链脂肪酸氧化障碍（LC-FAOD）的成人和儿科患者。该产品获得附条件上市许可，因为需要一项正在进行的儿科研究提供进一步的疗效和安全性证据，才能获得完整上市许可。 LC-FAOD是一组发生于各年龄段成人和儿童的罕见遗传性疾病。 LC-FAOD患者可能难以产生足够的能量，因为其身体无法将长链脂肪酸用作能量来源。这使他们面临发生重大临床事件的风险，从而导致频繁住院。 MHRA运营执行主任Julian Beach表示： “使患者能够获得高质量、安全且有效的医疗产品对我们非常重要。 “患有罕见疾病的患者在获得有效治疗方面往往面临重大障碍，我们确信该药品获批所适用的适当监管标准已得到满足。 “与所有产品一样，我们将持续密切审查triheptanoin的安全性。” Triheptanoin 是一种每日口服的液体，作为严格控制饮食的一部分。它只能凭处方获得，患者应始终严格按照医疗保健专业人员的指示服用此药。 该药物通过帮助防止细胞中的能量危机起作用，这种能量危机可导致肌肉分解、心肌损伤和低血糖。 该药物最常见的不良反应（可能影响超过十分之一的人）包括腹痛、腹泻和呕吐。 任何怀疑自己因该药物出现不良反应的人，都应告知其医生、药师或护士，并直接向黄卡计划报告。 致编辑的说明 新的上市许可于2026年10月30日授予 Ultragenyx Netherlands。 更多信息可在产品特性摘要和患者信息 leaflets 中找到，这些文件将在批准后7天内发布在 MHRA Products 网站上。 药品和医疗保健产品监管局（MHRA）负责通过确保所有药品和医疗器械有效且具有可接受的安全性，来监管英国的所有药品和医疗器械。我们所有的工作都以稳健且基于事实的判断为基础，以确保获益证明任何风险是合理的。 MHRA 是卫生和社会关怀部的执行机构。 媒体垂询，请联系 newscentre@mhra.gov.uk，或致电 020 3080 7651。
正文 · 英文原文
Press release Triheptanoin (Dojolvi) approved to treat adults and children with a group of rare inherited disorders MHRA approves triheptanoin to treat adult and paediatric patients with a group of rare inherited disorders. From: Medicines and Healthcare products Regulatory Agency Published: 30 October 2026 The Medicines and Healthcare products Regulatory Agency (MHRA) has today, 30 October 2026, approved an oral liquid form of triheptanoin (Dojolvi) to treat adult and paediatric patients with long-chain fatty acid oxidation disorders (LC-FAOD). The product has a conditional marketing authorisation because further evidence of its efficacy and safety from an ongoing paediatric study is required for a full marketing authorisation. LC-FAOD is a group of rare inherited disorders in adults and children of all ages. Patients with LC-FAOD may have difficulty producing enough energy because of their body’s inability to use long-chain fatty acids as an energy source. This puts them at risk of major clinical events resulting in frequent hospitalisations. Julian Beach, MHRA Executive Director, Operations, said: “Enabling patient access to high quality, safe and effective medical products is important to us. “Patients living with rare diseases often face significant barriers in accessing effective treatments and we’re assured that the appropriate regulatory standards for the approval of this medicine have been met. “As with all products, we will keep the safety of triheptanoin under close review.” Triheptanoin is a liquid that is taken daily by mouth, as part of a carefully controlled diet. It can only be obtained with a prescription, and patients should always take this medicine exactly as directed by their healthcare professional. The medicine works by helping to prevent energy crises in the cells that can cause muscle breakdown, damage to the heart muscle and low blood sugar. The most common side effects of the medicine (which may affect more than 1 in 10 people) include stomach pain, diarrhoea, and vomiting. Anyone who suspects they are having a side effect from this medicine are encouraged to talk to their doctor, pharmacist or nurse and report it directly to the Yellow Card scheme. Notes to editors The new marketing authorisation was granted on 30 October 2026 to Ultragenyx Netherlands. More information can be found in the Summary of Product Characteristics and Patient Information leaflets which will be published on the MHRA Products website within 7 days of approval. The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe. All our work is underpinned by robust and fact-based judgements to ensure that the benefits justify any risks. The MHRA is an executive agency of the Department of Health and Social Care. For media enquiries, please contact the newscentre@mhra.gov.uk, or call on 020 3080 7651.