Palvella罕见病新药QTORIN雷帕霉素凝胶获FDA受理并授予优先审评：有望成微囊型淋巴管畸形首个获批疗法
总部位于 Pennsylvania 州 Wayne 的临床阶段生物制药公司 Palvella Therapeutics（Nasdaq：PVLA）10 月 28 日宣布，美国食品药品监管局（FDA）已受理其 QTORIN 3.9% 雷帕霉素无水凝胶的新药申请（NDA），并授予优先审评资格，用于治疗微囊型淋巴管畸形（microcystic lymphatic malformations，简称 microcystic LMs）；处方药使用者付费法案（PDUFA）目标行动日期定为 2027 年 2 月 28 日。
微囊型淋巴管畸形是一种由 PI3K/mTOR 通路失调驱动的严重、终身疾病，美国确诊的儿童与成人患者超过 3 万人。病灶可出现渗液、出血、反复感染与功能损害，目前 FDA 尚无批准的治疗药物；QTORIN 雷帕霉素若获批，将成为该病的首个获批疗法。
支持本次申请的 3 期 SELVA 研究达到全部六项预设疗效终点（均 p<0.001）：在完成疗效评估期的 6 岁及以上受试者中，95% 在主要终点 mLM-IGA 上获得改善。药物耐受性良好，未出现药物相关的严重不良事件，且所有受试者在所有测量时间点的全身雷帕霉素浓度均低于 2 ng/mL。申请还得到 2 期研究结果及已发表的雷帕霉素超说明书使用文献支持。
QTORIN 雷帕霉素此前已获得 FDA 的突破性疗法认定、快速通道资格与孤儿药认定，研发还得到 FDA 孤儿产品开发办公室的经费资助。首席执行官 Wes Kaupinen 称，NDA 获受理并取得优先审评是公司的一项重大里程碑。公司正筹备在 2027 年上半年作为独立商业化公司在美国上市该药；其管线还包括用于皮肤静脉畸形与临床显著性棘状角化瘤的 QTORIN 雷帕霉素，以及用于播散性浅表光化性汗孔角化症的 QTORIN 匹伐他汀。
FDA Accepts Palvella's QTORIN Rapamycin NDA With Priority Review for Microcystic Lymphatic Malformations; PDUFA Date Set for Feb. 28, 2027
Palvella Therapeutics, Inc. (Nasdaq: PVLA), a clinical-stage biopharmaceutical company based in Wayne, Pennsylvania, announced on October 28 that the U.S. Food and Drug Administration (FDA) has accepted for filing and granted Priority Review to the New Drug Application (NDA) for QTORIN 3.9% rapamycin anhydrous gel for the treatment of microcystic lymphatic malformations (microcystic LMs), with a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027.
Microcystic LMs are a serious, lifelong condition driven by PI3K/mTOR dysregulation, affecting more than 30,000 diagnosed children and adults in the United States. The lesions can leak, bleed, cause recurrent infections and impair function, and there are currently no FDA-approved treatments; if approved, QTORIN rapamycin would be the first FDA-approved therapy for the disease.
The NDA is supported by the Phase 3 SELVA study, which met all six prespecified efficacy endpoints (all p<0.001): 95 percent of participants aged six and older who completed the efficacy evaluation period improved on the mLM-IGA primary endpoint. The drug was well tolerated, with no drug-related serious adverse events, and systemic rapamycin levels remained below 2 ng/mL at all measured timepoints for all participants. Phase 2 results and published literature on off-label rapamycin use also support the application.
QTORIN rapamycin previously received Breakthrough Therapy, Fast Track and Orphan Drug designations, and the program received grant funding from the FDA's Office of Orphan Products Development. Chief Executive Officer Wes Kaupinen called the acceptance and Priority Review a significant milestone for the company. Palvella is preparing for a potential standalone U.S. commercial launch in the first half of 2027; its pipeline also includes QTORIN rapamycin for cutaneous venous malformations and clinically significant angiokeratomas, and QTORIN pitavastatin for disseminated superficial actinic porokeratosis.
来源：GSM·美国（原文链接）
图片来源：图：美国食品药品监管局（FDA）位于 Maryland 州的白橡树园区一号大楼。照片：美国食品药品监管局，公共领域，经 Wikimedia Commons。 / Photo: Building 1 of the U.S. Food and Drug Administration's White Oak campus in Maryland. Photo by the U.S. Food and Drug Administration, public domain, via Wikimedia Commons.
