Lenacapavir Sodium

吉利德科学（Gilead Sciences）今日宣布，欧洲药品管理局（EMA）人用药品委员会（CHMP）在加速审评程序下对该公司注射用HIV-1衣壳抑制剂lenacapavir作出积极意见，建议其作为暴露前预防（PrEP）药物用于降低成年人与青少年中HIV-1感染风险较高人群因性行为获得HIV-1的风险。欧盟委员会（EC）的最终决定预计将在今年晚些时候公布。根据新闻稿，如获批准，lenacapavir将成为欧盟（EU）首款每年两次的HIV PrEP疗法。艾滋病是由于HIV感染造成的传染性疾病。现代抗病毒药物的发展和“鸡尾酒疗法”的兴起，已经让HIV感染患者能够长期控制病情，并且达到与正常人相当的预期寿命。而近年来的研究发现，在未受到感染时服用抗病毒药物，可以降低被HIV病毒感染的风险。这种预防HIV感染的疗法称为PrEP疗法。Truvada是由吉利德所开发的一款抗病毒药物，获FDA批准用于PrEP超过十年。虽然每日口服Truvada几乎可100%避免HIV感染，然而由于药物成本以及服用与HIV相关药物所面临的污名等因素限制，许多人难以每日服药，口服PrEP疗法的实际效果远低于理论值。因此开发一种能够增加服药依从性的长效HIV  PrEP疗法具有显著需求。这次lenacapavir获CHMP的推荐，主要基于3期PURPOSE 1和PURPOSE 2临床试验数据的结果。在PURPOSE 1研究中，主要分析数据显示，每年两次皮下注射lenacapavir组的2134名参与者中无一人感染HIV，实现了HIV感染的100%预防效果，且在预防HIV感染方面优于每日口服Truvada治疗组。在PURPOSE 2研究中，在lenacapavir组的2179名参与者中，仅有两例HIV感染，表明该组99.9%的参与者未感染HIV，且在预防HIV感染方面优于每日口服Truvada治疗组。两项研究均显示，lenacapavir在预防HIV感染方面优于背景HIV感染发生率（bHIV），且总体耐受性良好，未发现显著或新的安全性问题。两项研究的数据已发表在《新英格兰医学杂志》。图片来源：123RFLenacapavir是一种“first-in-class”长效艾滋病毒衣壳抑制剂，可以干扰艾滋病毒衣壳蛋白的组装和拆卸，在病毒生命周期的多个阶段发挥作用。它曾获得美国FDA授予的突破性疗法认定，用于HIV病毒感染暴露前预防，并在今年6月获FDA批准上市。2024年，《科学》期刊也将lenacapavir评为年度科学突破，表示这一“first-in-class”药物为艾滋病的长效治疗与预防带来了新的曙光。▲Lenacapavir药品（图片来源：参考资料[2]）参考资料：[1] Gilead Receives Positive CHMP Opinions Under Accelerated Review From European Medicines Agency for Twice-Yearly Lenacapavir for HIV Prevention. Retrieved July 25, 2025 from https://www.businesswire.com/news/home/20250724024903/en/Gilead-Receives-Positive-CHMP-Opinions-Under-Accelerated-Review-From-European-Medicines-Agency-for-Twice-Yearly-Lenacapavir-for-HIV-Prevention[2]  Yeztugo® (Lenacapavir) Is Now the First and Only FDA-Approved HIV  Prevention Option Offering 6 Months of Protection. Retrieved June 18,  2025 from  https://www.businesswire.com/news/home/20250520465880/en/Yeztugo-Lenacapavir-Is-Now-the-First-and-Only-FDA-Approved-HIV-Prevention-Option-Offering-6-Months-of-Protection免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。👇 分享，点赞，在看，聚焦全球生物医药健康创新

Europe’s Committee for Medicinal Products for Human Use has reversed its decision on Eli Lilly's Alzheimer's disease treatment Kisunla, giving it a positive opinion in a limited patient population. Four months after giving Eli Lilly’s Alzheimer’s disease treatment a thumbs-down, Europe’s Committee for Medicinal Products for Human Use (CHMP) has recommended it for approval, albeit in a restricted patient population.The positive opinion covers Kisunla's use in patients with early Alzheimer’s symptoms who have one or no copies of the apolipoprotein E ε4 (ApoE4) gene. The drug is not recommended for individuals carrying two copies of the ApoE4 gene. Those patients are more susceptible to amyloid-related imaging abnormalities (ARIA), which are a primary side effect of currently approved Alzheimer’s drugs.The restriction is the same as the one applied by European regulators when it granted marketing authorization to Eisai and Biogen’s Alzheimer’s drug Leqembi three months ago. Those companies went through the same rejection/appeal process as Lilly did with European regulators.Lilly went through the same go-round with regulators in the U.S. before it secured approval of Kisunla in July of last year. Then, earlier this month, the FDA signed off on a new dosing regimen for the treatment, featuring a more gradual titration, which leads to a significantly lower rate of ARIA-related brain swelling. The new dosing regimen also applies to Thursday’s thumbs-up from the CHMP. Endorsement for Gilead’s PrEP injection  The CHMP has also provided a positive opinion on Gilead Sciences' HIV pre-exposure prophylaxis (PrEP) Yeytuo (lenacapavir), a twice-a-year subcutaneous injection which has been hailed as one of the most important breakthroughs in the effort to end the epidemic.The FDA blessed the shot last month under its commercial name Yeztugo.The CHMP recommendation applies to adults and adolescents and came under an accelerated pathway, “because it is considered to be of major public health interest in the EU and the rest of the world,” the regulator said.It was also evaluated under the EU-Medicines for all program, which included participation from the World Health Organization and regulators from several African and Asian nations. The program helps speed approvals where “regulatory capacity may be limited,” the CHMP said.“Lenacapavir for PrEP has the potential to become a critical tool for public health, helping to expand prevention options for people who face the highest barriers to care,” Dietmar Berger, M.D., Ph.D., Gilead’s chief medical officer, said in a release. Other CHMP decisions A month after Supernus Pharmaceuticals made a $561 million bet on Zurzuvae, buying out its developer Sage Therapeutics, the CHMP has registered a positive opinion on the treatment for postpartum depression. The Biogen-partnered treatment was endorsed by the FDA in August 2023, along with a rejection from the regulator in a much larger patient population, major depressive disorder.The European regulator has also signed off on rare disease specialist IntraBio’s Aqneursa for the treatment of the lysosomal storage disorder Niemann-Pick disease type C (NPC). The endorsement comes 10 months after the FDA approved two treatments for NPC in consecutive weeks, Aqneursa and Zevra Therapeutics’ Miplyffa, which has yet to win a nod in Europe.Less than three weeks after the FDA approved KalVista Pharmaceuticals' Ekterly for acute attacks of hereditary angioedema (HAE), the CHMP has followed suit. This is the first oral treatment for HAE recommended for approval in Europe, reducing the burden of injection on patients and speeding up the time between onset of attack and administration of the medicine, the CHMP said. Ono Pharmaceuticals has won a CHMP nod for Romvimza, for the treatment of adults with symptomatic tenosynovial giant cell tumor (TGCT), a condition where the tissue surrounding the joints and tendons expands abnormally, forming outgrowths of the joint. The drug, which was acquired last year in Ono’s $2.4 billion buyout of Deciphera Pharmaceuticals, was endorsed by the FDA in February of this year, joining Daiichi Sankyo’s Turalio as the only other marketed treatment for TGCT.Ionis Pharmaceuticals' Tryngolza, for the treatment of adults with familial chylomicronemia syndrome, has gotten a thumbs-up from CHMP. The rare, autosomal recessive inherited disorder is characterized by elevated levels of triglycerides in the blood. The FDA approved the treatment in December of last year.Also receiving a CHMP nod is Servier’s Voranigo for low-grade astrocytoma or oligodendroglioma, two rare malignant brain tumors. The FDA blessed the treatment in August of last year.