FDA accepts Palvella's NDA for QTORIN rapamycin, grants priority review

Palvella Therapeutics said on October 28, 2026 that the FDA accepted for filing and granted Priority Review to its New Drug Application for QTORIN 3.9% rapamycin anhydrous gel, intended to treat microcystic lymphatic malformations. The company disclosed in an 8-K filing that the FDA assigned a Prescription Drug User Fee Act target action date of February 28, 2027.¹

If approved, QTORIN rapamycin would become the first FDA-approved therapy for children and adults with microcystic LMs, a serious, lifelong condition associated with persistent leaking, bleeding, recurrent infections, and functional impairment.¹ The company said there are no FDA-approved treatments for the estimated 30,000 or more people diagnosed with microcystic LMs in the United States.¹

The filing was built on data from the Phase 3 SELVA trial and earlier Phase 2 work. Palvella said the NDA evidence package draws on the Phase 3 SELVA study, which met statistical significance on all six prespecified efficacy endpoints with p values under 0.001, along with Phase 2 results that had supported the FDA's earlier Breakthrough Therapy designation decision.¹ In SELVA, 95% of participants aged 6 years and older who completed the efficacy evaluation period showed improvement on the mLM-IGA primary endpoint.¹ The company reported the drug was well tolerated, with no drug-related serious adverse events and systemic rapamycin levels staying below 2 ng/mL at all measured timepoints.¹

QTORIN rapamycin already holds Breakthrough Therapy, Fast Track, and Orphan Drug designations from the FDA for this indication, and the program has received grant funding from the FDA Office of Orphan Products Development.¹

Palvella said it is preparing for a possible standalone U.S. commercial launch in the first half of 2027, pending approval, and has built out rare disease commercial leadership, a field-based medical science liaison team, and a patient services organization.¹ The company's other pipeline work includes QTORIN rapamycin for cutaneous venous malformations and angiokeratomas, and QTORIN pitavastatin for disseminated superficial actinic porokeratosis.¹