QTORIN rapamycin - (SELVA)

August 31, 2026: Palvella Therapeutics (NASDAQ: PVLA) said it completed the rolling NDA submission to the FDA for QTORIN 3.9% rapamycin anhydrous gel for microcystic lymphatic malformations (microcystic LMs), a rare genetic disease with no FDA-approved therapies. The application was submitted via the 505(b)(2) pathway.

The NDA includes Phase 3 SELVA data, in which QTORIN met the primary endpoint, a pre-specified key secondary endpoint, and all four secondary efficacy endpoints, with all six efficacy endpoints statistically significant (all p<0.001). Among participants aged 6 years and older who completed the efficacy evaluation period, 86% were rated “Much Improved” or “Very Much Improved” at Week 24 on the microcystic LM Investigator Global Assessment. The company said QTORIN was well tolerated, with no drug-related serious adverse events and systemic rapamycin levels below 2 ng/mL at all timepoints. The NDA also includes Phase 2 results that supported Breakthrough Therapy designation, plus published literature and other evidence on off-label rapamycin use.

FDA is expected within 60 days to decide whether to file the application and whether to grant Priority Review; if granted, the review goal would be six months. QTORIN previously received Breakthrough Therapy, Fast Track, and Orphan Drug designations. Palvella is preparing for a potential U.S. launch in 1H 2027 if approved.