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.
Source: GSM · USA (original Chinese edition)
Photo: 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.
