FDA批准Givosiran治疗急性肝卟啉症
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FDA批准Givosiran治疗急性肝卟啉症
笔记 2019-11-20 FDA On November 20, 2019, the Food and Drug Administration approved givosiran (GIVLAARI, Alnylam Pharmaceuticals, Inc.) for adults with acute hepatic porphyria (AHP)【FDA批准首例治疗遗传性罕见病的疗法】 Efficacy was evaluated in ENVISION (NCT03338816), a randomized, double‑blind, placebo‑controlled, multinational trial enrolling 94 patients with AHP. Patients were randomized (1:1) to receive once monthly subcutaneous injections of givosiran 2.5 mg/kg or placebo during a 6‑month double‑blind period. The primary efficacy outcome measure was the rate of porphyria attacks requiring hospitalizations, urgent healthcare visit, or intravenous hemin administration at home. The mean rates of attacks over a 6-month time period were 1.9 (95% CI:1.3,2.8) for patients receiving givosiran and 6.5 (95% CI:4.5, 9.3) for those on placebo. On average, patients with AHP on givosiran experienced 70% (95% CI: 60%, 80%) fewer porphyria attacks compared to placebo. The most common adverse reactions (>20% of patients) included nausea and injection site reactions. The label contains warnings for anaphylactic reactions, hepatic and renal toxicities, and injection site reactions. Hepatic toxicity was mostly transaminase elevation. Renal toxicity was mostly serum creatinine elevation and decreases in estimated glomerular filtration rate. The recommended givosiran dose is 2.5 mg/kg once monthly by subcutaneous injection. View full prescribing information for GIVLAARI. FDA granted this application priority review and orphan product and breakthrough therapy designations. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics. Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088. For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov. Follow the Oncology Center of Excellence on Twitter @FDAOncology 岗位必读建议
文件适用范围本文适用于美国FDA监管下的药品和生物制品,包括创新药、生物类似药、原料药等。主要针对治疗严重或危及生命疾病的药物,包括化学药、生物制品、疫苗等。适用于Biotech、大型药企、跨国药企、CRO和CDMO等各类企业。 文件要点总结1. 加速审批程序概述
2. 突破性疗法资格标准
3. 优先审评资格标准
4. 加速审批的条件和后市场要求
5. 沟通与合作
以上仅为部分要点,请阅读原文,深入理解监管要求。 |