Trofinetide

SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion following a re-examination procedure, recommending the granting of a marketing authorization for DAYBU® (trofinetide) for the treatment of neurobehavioral symptoms of Rett syndrome in adults and pediatric patients aged five years and older. If granted marketing authorization by the European Commission, DAYBU® would be the first therapy approved for this indication in the European Union (EU). “The CHMP’s positive opinion for DAYBU® is an important milestone in our mission to bring this innovative therapy to the EU, where there are no therapies specifically approved for the neurobehavioral symptoms of this devastating condition," said Catherine Owen Adams, Acadia’s Chief Executive Officer. “Our commitment is to make a meaningful difference in the lives of patients, caregivers, and the wider Rett community by addressing this significant unmet need, and we are very pleased with the outcome of the re-examination process." The CHMP’s recommendation is primarily based on results from the Phase 3 LAVENDER™ study, which demonstrated statistically significant and clinically meaningful improvements in core features of Rett syndrome, as measured by the Rett Syndrome Behaviour Questionnaire (RSBQ) and Clinical Global Impression-Improvement (CGI-I) scale. Importantly, these findings indicate that treatment with DAYBU® can address some of the most impactful aspects of Rett syndrome, which severely impact quality of life for patients and caregivers. "For decades, families in Europe affected by Rett syndrome have had no medicine specifically approved for the neurobehavioral symptoms of this condition, despite the profound impact they have on almost every aspect of daily life,” said Pedro Rocha, President of Rett Syndrome Europe. “The CHMP’s positive opinion represents hope for thousands of European Union individuals living with this devastating condition, their families and caregivers." Following the CHMP recommendation, the European Commission will review the opinion and is expected to issue a final decision in the coming months. If DAYBU® is approved, the marketing authorization would apply to all 27 EU member states, as well as Iceland, Liechtenstein, and Norway. About Rett Syndrome Rett syndrome is a rare, complex, neurodevelopmental disorder and occurs in approximately one of every 10,000 to 15,000 female births worldwide.1-3 A child with Rett syndrome generally exhibits an early period of apparently normal development until six to 18 months, when many of their skills seem to slow down or stagnate. This is typically followed by a regression phase when the child loses acquired communication skills and purposeful hand use. The child may then experience a plateau period in which they could show mild recovery in cognitive interests, but body movements remain severely diminished. As they age, those individuals living with Rett may continue to experience a stage of motor deterioration, which can last the rest of the patient’s life.2 Rett syndrome is typically caused by a genetic mutation on the MECP2 gene.4 In preclinical studies, deficiency in MeCP2 function is thought to lead to impairment in synaptic communication and brain plasticity, and the deficits in synaptic function may be associated with Rett manifestations.4-6 Symptoms of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities.7 Most individuals living with Rett syndrome typically live into adulthood and require intense round-the-clock care.1,8 About DAYBU® DAYBU® (trofinetide) is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor 1. About Acadia Pharmaceuticals Acadia is committed to turning scientific promise into meaningful innovation that makes the difference for underserved neurological and rare disease communities around the world. Our commercial portfolio includes the first and only FDA-approved treatments for Parkinson’s disease psychosis and Rett syndrome. We are developing the next wave of therapeutic advancements with a robust and diverse pipeline that includes mid- to late-stage programs in Alzheimer’s disease psychosis and Lewy body dementia psychosis, along with earlier-stage programs that address other underserved patient needs. At Acadia, we’re here to be their difference. For more information, visit us at acadia.com and follow us on LinkedIn and X. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements other than statements of historical fact and can be identified by terms such as “may,” “will,” “should,” “could,” “would,” “intends,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “projects,” “predicts,” “potential,” “guidance,” “continue” and similar expressions (including the negative thereof) intended to identify forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the expected or potential approval of trofinetide or DAYBU® by the European Commission, including the potential timing of such approval, and if approved, the potential benefits of trofinetide as a treatment of the neurobehavioral symptoms of Rett Syndrome and the importance of trofinetide to patients, caregivers and the Rett community. Forward-looking statements are subject to known and unknown risks, uncertainties, assumptions and other factors that may cause our actual results, performance or achievements to differ materially and adversely from those anticipated or implied by our forward-looking statements. Such risks, uncertainties and other factors include, but are not limited to, the inherent uncertainty regarding development of product candidates, including approval of trofinetide by the European Commission; our dependency on the continued successful commercialization of our products and our ability to maintain or increase sales of our products; our ability to obtain necessary regulatory approvals to commercialize our products and product candidates; if and when approved, market acceptance of our products and our ability to continue to stay in compliance with applicable laws and regulations. Given the risks and uncertainties, you should not place undue reliance on these forward-looking statements. For a discussion of these and other risks, uncertainties and other factors that may cause our actual results, performance or achievements to differ, please refer to our quarterly report on Form 10-Q for the period ended March 31, 2026, filed on May 7, 2026, as well as our subsequent filings with the Securities and Exchange Commission from time to time. The forward-looking statements contained herein are made as of the date hereof, and we undertake no obligation to update them after this date, except as required by law. References 1 Fu C, Armstrong D, Marsh E, et al. Consensus guidelines on managing Rett syndrome across the lifespan. BMJ Paediatrics Open. 2020;4:e000717. 2 Kyle SM, Vashi N, Justice MJ. Rett syndrome: a neurological disorder with metabolic components. Open Biol. 2018; 8:170216. 3 May DM, Neul JL, Satija A, et al. Real-world clinical management of individuals with Rett syndrome: a physician survey. J Med Econ. 2023;26(1):1570-1580. 4 Amir RE, Van den Veyver IB, Wan M, et al. Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2. Nat Genet. 1999; 23(2):185-188. 5 Fukuda T, Itoh M, Ichikawa T, et al. Delayed maturation of neuronal architecture and synaptogenesis in cerebral cortex of Mecp2-deficient mice. J Neuropathol Exp Neurol. 2005; 64(6):537-544. 6 Asaka Y, Jugloff DG, Zhang L, et al. Hippocampal synaptic plasticity is impaired in the Mecp2-null mouse model of Rett syndrome. Neurobiol Dis. 2006; 21(1):217-227. 7 Neul JL, Kaufmann WE, Glaze DG, et al. Rett syndrome: revised diagnostic criteria and nomenclature. Ann Neurol. 2010; 68(6):944-950. 8 Tarquinio DO, Hou W, Neul JL, et al. The changing face of survival in Rett syndrome and MECP2-related disorders. Pediatr Neurol. 2015; 53(5):402-411.

喜欢我的分享，点击上方 关注我吧！ 2026 年 6 月 22–25 日 人用医药产品委员会（CHMP）会议要点 2026 年 6 月人用医药产品委员会（CHMP）会议议程已发布于欧洲药品管理局（EMA）官网；本次会议纪要将于未来数周内对外公示。 2026 年 6 月统计数据 ——CHMP 审评意见及撤回申请月度、累计数据 新药正面审评意见：6 份 其中普通新药 3 款、罕见病药 1 款、生物类似药 2 款； 2026 年累计：42 份 新药否定审评意见：3 份 2026 年累计：6 份 适应症拓展正面审评意见：13 份 2026 年累计：72 份 新药上市申请撤回：0 份 2026 年累计：5 份 6款新药获上市推荐许可 欧洲药品管理局人用医药产品委员会（CHMP）在 2026 年 6 月会议上，推荐六款药品获批上市。 委员会建议授予Aujemflu（灭活流感病毒表面抗原：血凝素 + 神经氨酸酶）上市许可，该疫苗用于 50 周岁及以上人群流感预防。 CHMP 建议授予Hopledo（左旋多巴 / 卡比多巴）上市许可，用于成人帕金森病，且经口服左旋多巴 - 多巴脱羧酶抑制剂方案治疗后病情控制不佳、伴中重度运动波动（运动功能异常）患者。 Onswik（艾夫西托阿法胰岛素）获 CHMP 正面审评意见，适应症为成人 2 型糖尿病。 委员会对2款生物类似药出具支持意见： Denosumab Ascend（地舒单抗）：用于伴骨转移晚期实体瘤成人患者骨相关并发症预防；以及成人、骨骼发育成熟青少年骨巨细胞瘤治疗。 Nylaspeg（培非格司亭）：用于缩短化疗所致中性粒细胞减少症（中性粒细胞为一类白细胞）持续时间，降低发热性中性粒细胞减少症发生率。 复审结果 经复审后，委员会建议授予Daybu（曲非奈肽）上市许可，用于 5 周岁及以上儿童与成人雷特综合征神经行为相关症状治疗。相关神经行为症状包括手部重复刻板动作、躁动、情绪异常、焦虑、睡眠障碍及沟通障碍。 CHMP 最初曾驳回本品用于雷特综合征的上市申请；本次复审后同意批准上市，但适应症范围受限。 如需了解本次复审详细结论，可查阅下方表格配套问答文件。 三款药品获否定审评意见 委员会对三款药品出具不予批准意见： Tacquell（自体黑色素瘤来源肿瘤浸润淋巴细胞，体外扩增）：用于晚期黑色素瘤（已发生全身转移的皮肤恶性肿瘤）。 Yartemlea（那索普利单抗）：用于 2 周岁及以上造血干细胞移植（HSCT）相关血栓性微血管病成人及儿童患者。血栓性微血管病是造血干细胞移植后可危及生命的严重并发症；造血干细胞移植指替换患者自身骨髓，植入供者干细胞重建可生成健康血细胞的骨髓。 Xervyteg（混合异体粪便微生物制剂）：用于急性移植物抗宿主病（aGvHD）。该病为移植术后高危致命病症，骨髓 / 干细胞移植后供体细胞攻击受者机体。 如需了解上述三款药品不予批准的详细说明，可查阅下方表格配套问答文件。 、 12 款药品获批拓展治疗适应症 CHMP 出具正面意见，建议修订Imvanex上市许可条款。本品为天花、猴痘及痘苗病毒（痘病毒科另一毒株）预防疫苗，本次建议适用人群最低年龄下调至 2 周岁及以上（原 12 周岁及以上）。 委员会同时建议欧盟境内已上市的另外 11 款药品新增适应症，分别为：Datroway、Jaypirca、Leqvio、Menquadfi、Opzelura、Orladeyo、Rezolsta、Rinvoq、Stelara、Symtuza、Tecvayli。 两项适应症拓展申请撤回 两款复方制剂拓展适应症申请已撤回：DuoResp Spiromax、BiResp Spiromax（布地奈德 / 福莫特罗）原申请新增 “仅用于轻度哮喘急救缓解治疗” 的适应症。 启动欧盟法规第 31 条集中审评程序 委员会启动对利福平口服混悬液 / 糖浆（20 毫克 / 毫升） 的专项审评。本品含抗生素利福平，用于结核病及其他重度感染治疗。 启动审评原因是监管方担忧辅料二乙醇胺（DEA）含量存在安全隐患；啮齿动物长期高剂量暴露试验提示二乙醇胺可能具有致癌性。 本次审评由荷兰药品管理局依据《欧盟指令 2001/83/EC》第 31 条提请启动，相关启动通告详见下方表格。 集中审评程序收尾结论 CHMP 完成对Tavneos（阿伐可泮） 的全部审评工作，建议欧盟撤销该药品上市许可，现有证据无法证实其临床获益大于用药风险。本品用于成人重度活动性肉芽肿性多血管炎（GPA）、显微镜下多血管炎（MPA），二者均为罕见血管炎症。本次专项审评启动，是因新出现信息对支撑本品欧盟获批的核心临床试验数据完整性提出重大质疑。 其他更新事项 CHMP 批准Fluenz（鼻喷减毒活流感疫苗）可由非医疗专业人员在医护人员监督下完成接种。本品为鼻喷剂型，用于 2 周岁至 18 周岁以下儿童青少年季节性流感预防。 委员会建议修订Ixchiq（减毒基孔肯雅疫苗）上市许可条款，限定仅适用于 12 周岁及以上、基孔肯雅病毒高感染风险人群。 CHMP 完成Mounjaro（替尔泊肽） 新增适应症申请审评：原申请拓展适应症为合并心血管疾病的成人 2 型糖尿病，用于降低主要不良心血管事件（心、循环系统严重病变）风险。委员会未批准该新增适应症，但同意将申报配套研究数据纳入药品说明书，确保临床医师可查阅替尔泊肽在合并心血管疾病 2 型糖尿病人群中的完整研究数据。 【关键专业术语注解】 marketing authorisation：上市许可 positive/negative opinion：正面审评意见 / 不予批准（否定）意见 biosimilar medicines：生物类似药 re-examination：上市申请复审 restricted indication：受限适应症 referral：欧盟集中专项审评程序 excipient：药用辅料 possible carcinogen：潜在致癌物 product information：药品说明书（欧盟统一称谓） febrile neutropenia：发热性中性粒细胞减少症 autologous /allogeneic：自体 / 异体 graft-versus-host disease (GvHD)：移植物抗宿主病 cardiovascular events：心血管不良事件 reliever-only treatment：仅急救缓解治疗 live nasal influenza vaccine：鼻喷减毒活流感疫苗 Agenda：会议议程 minutes：（官方）会议纪要（药监机构固定专用表述，非普通分钟） 原文延伸： https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-22-25-june-2026 内容来源于EMA,翻译仅供参考学习 希望搭建一个理性交流的平台，与志同道合者共同探讨化工的未来图景。    ——专注化工领域   懂您的需求—— 化工综合服务提供商 企业官网建设|产品国内外推广|撮合交易|行情资讯|供应链金融 业务联系：潘经理    手     机：13958177281(微信同号） 邮     箱：472097288@qq.com 加我微信，获取更多福利 化工潘说——化工一起说  关注我，获取更多信息！