FDA受理Revtorpyk扩大适用申请：PIK3CA突变型乳腺癌患者有望获二线新选择，2027年6月25日出结果
总部位于明尼苏达州 Minneapolis 的生物科技公司 Celcuity（纳斯达克：CELC）11月9日宣布，美国食品药品监管局（FDA）已受理其乳腺癌药 Revtorpyk（gedatolisib）的补充新药申请（sNDA）。这次申请要把该药的适用人群扩大到带 PIK3CA 突变的激素受体阳性（HR+）、人表皮生长因子受体2阴性（HER2-）局部晚期或转移性乳腺癌患者，FDA 给出的审评目标日期是2027年6月25日。
按申请内容，目标患者是在转移阶段接受过至少一线内分泌治疗后病情仍进展的人群。申请依据是三期临床试验 VIKTORIA-1 中 PIK3CA 突变队列的数据，该药此前已凭早期临床数据获得 FDA 的突破性疗法认定与快速通道资格。
Celcuity 首席执行官兼联合创始人 Brian Sullivan 说，FDA 受理这份申请，意味着把 Revtorpyk 带给更多 HR+/HER2- 局部晚期或转移性乳腺癌患者又近了一步；如果获批，该药将可作为二线治疗用于患者，不再区分肿瘤的 PIK3CA 状态。
Revtorpyk 今年7月14日已先获 FDA 批准，联合氟维司群、并可加用哌柏西利，用于未检出 PIK3CA 突变的同类患者。公司介绍，HR+/HER2- 是最常见的乳腺癌亚型，约占全部乳腺癌的70%，其中约40%的患者肿瘤带有 PIK3CA 突变。
Revtorpyk 是一种同时作用于 PI3K 多个亚型与 mTOR 复合物的激酶抑制剂。公司在公告中同时列出重要安全信息，其中口腔炎是较常见的不良反应，联合用药组发生率最高达72%，用药期间需按说明书监测与处理。FDA 的受理不等于最终批准，结论要等2027年6月25日的目标日期。
（来源：Celcuity）
FDA Accepts Revtorpyk Expansion Application for PIK3CA-Mutant Breast Cancer, Decision Due June 25, 2027
Celcuity Inc. (Nasdaq: CELC), a biotechnology company headquartered in Minneapolis, Minnesota, announced on November 9 that the U.S. Food and Drug Administration (FDA) has accepted for review its supplemental New Drug Application (sNDA) for Revtorpyk (gedatolisib). The application seeks to expand the drug's use to patients with hormone receptor positive (HR+), HER2-negative locally advanced or metastatic breast cancer with a PIK3CA mutation, and the FDA assigned a Prescription Drug User Fee Act goal date of June 25, 2027.
The filing covers patients whose disease progressed on or after at least one line of endocrine therapy in the metastatic setting. It is based on clinical data from the PIK3CA mutant cohort of the Phase 3 VIKTORIA-1 trial, and the drug previously received Breakthrough Therapy and Fast Track designations from the FDA.
"The FDA's acceptance of this sNDA takes us one step closer to bringing Revtorpyk to more patients living with HR+/HER2- locally advanced or metastatic breast cancer," said Brian Sullivan, Celcuity's chief executive officer and co-founder. If approved, he said, Revtorpyk would become available as second-line therapy regardless of a tumor's PIK3CA status.
Revtorpyk was first approved by the FDA on July 14, 2026, in combination with fulvestrant, with or without palbociclib, for patients in the same subtype without a detected PIK3CA mutation. HR+/HER2- is the most common breast cancer subtype, accounting for about 70 percent of all breast cancers, and about 40 percent of those tumors carry PIK3CA mutations, the company said.
Revtorpyk is a kinase inhibitor targeting class I PI3K isoforms and the mTOR complexes mTORC1 and mTORC2. The company's announcement also lists important safety information, including stomatitis, which occurred in up to 72 percent of patients in one combination group. Acceptance for review is not an approval; the FDA's decision is due by the June 25, 2027 goal date.
(Source: Celcuity)
来源：Celcuity
发布：GSM·美国（发布链接）
图片来源：图：美国食品药品监管局总部大楼（GSM联邦固定图库）。 / Photo: U.S. Food and Drug Administration headquarters (GSM federal asset library).
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