FDA批准ORLADEYO颗粒剂型用于成人与青少年：遗传性血管性水肿口服预防药添新选择
总部位于美国北卡罗来纳州 Research Triangle Park 的 BioCryst Pharmaceuticals 公司10月21日宣布，美国食品药品监管局（FDA）已批准其补充新药申请，ORLADEYO（berotralstat）150毫克口服颗粒剂型可用于12岁及以上成人与青少年患者，预防遗传性血管性水肿（HAE）发作。
新获批的颗粒剂型为吞咽困难、不愿服用胶囊、或希望在12岁以后继续使用颗粒剂型的患者提供了另一种每日一次的口服选择。公司介绍，颗粒外观与大小类似撒粉，可直接倒入口中用水或牛奶送服，也可撒在一勺柔软的非酸性食物上服用。此次批准依据的是颗粒剂型与胶囊剂型的化学、生产与质量控制信息，以及安全性与耐受性数据。
ORLADEYO 是首个、也是目前唯一一个面向2岁及以上 HAE 患者的靶向口服预防疗法，通过降低血浆激肽释放酶活性预防发作。其胶囊剂型于2020年12月首次获 FDA 批准用于12岁及以上患者，现已在全球45多个国家获批；颗粒剂型曾于2025年12月获批用于2至未满12岁儿童患者。公司援引的两项临床研究显示，150毫克胶囊在96周时平均发作率降低91%，急救药物使用减少88%，最常见的不良反应为胃肠道反应与背痛，多数在治疗初期出现并随时间减轻。
BioCryst 总裁兼首席执行官 Charlie Gayer 表示，此次批准让医生能以新剂型提供 ORLADEYO 已证实的疗效，方便吞咽困难或不愿服胶囊的患者用药。公司同时说明，ORLADEYO 未获批用于治疗急性 HAE 发作，高于每日一次150毫克的剂量不推荐使用，因可能延长 QTc 间期。
FDA Approves ORLADEYO Oral Pellets for Adults and Adolescents: New Formulation Option for Hereditary Angioedema Prevention
BioCryst Pharmaceuticals, based in Research Triangle Park, North Carolina, announced on October 21 that the U.S. Food and Drug Administration (FDA) has approved its supplemental New Drug Application for the 150 mg oral pellet formulation of once-daily ORLADEYO (berotralstat) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older.
The new pellet formulation gives patients who have difficulty swallowing, prefer not to take a capsule, or wish to remain on pellets beyond age 12 another once-daily oral option. The pellets are sprinkle-like in size and can be poured directly into the mouth and swallowed with water or milk, or sprinkled over a spoonful of soft, non-acidic food. The approval was based on chemistry, manufacturing and controls information and data on the safety and tolerability of the pellets compared with the capsules.
ORLADEYO is the first and only targeted oral prophylactic therapy for HAE patients aged 2 and above, working by decreasing plasma kallikrein activity. Its capsule form was first approved by the FDA in December 2020 for patients 12 and older and is now approved in more than 45 countries; the pellet form was approved in December 2025 for children aged 2 to under 12. Across two clinical studies cited by the company, 150 mg capsules produced a mean attack-rate reduction of 91% at 96 weeks and an 88% reduction in rescue medication use, with gastrointestinal reactions and back pain the most frequently reported adverse reactions.
BioCryst President and Chief Executive Officer Charlie Gayer said the approval strengthens the company's ability to offer personalized treatment choice with the proven efficacy of ORLADEYO in a formulation that is easier for patients who have difficulty swallowing or prefer not to take a capsule. The company noted ORLADEYO is not established for treating acute HAE attacks, and doses above 150 mg once daily are not recommended because of the potential for QTc interval prolongation.
发布：GSM·美国（发布链接）
图片来源：图：处方药胶囊（资料图）。美国国家过敏与传染病研究所 NIAID 摄，CC BY 2.0，取自维基共享资源。Photo: Prescription capsules (file photo). NIAID, CC BY 2.0, via Wikimedia Commons.
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