Palvella Therapeutics Announces Positive Topline Results from Phase 3 SELVA Clinical Study of QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) in Microcystic Lymphatic Malformations

Core Insights - Palvella Therapeutics announced positive topline results from the Phase 3 SELVA study of QTORIN™ rapamycin for treating microcystic lymphatic malformations, meeting its primary endpoint with a statistically significant improvement [2][3] - The company plans to submit a New Drug Application (NDA) to the FDA in the second half of 2026, with potential approval in the first half of 2027 [3][10] Study Results - The primary endpoint, the Microcystic Lymphatic Malformation Investigator Global Assessment (mLM-IGA), showed a mean improvement of +2.13 points (p<0.001) [5][6] - 95% of participants aged ≥ 6 who completed the efficacy evaluation improved on the mLM-IGA at Week 24, with 86% rated as "Much Improved" or "Very Much Improved" [6][9] - All four secondary efficacy endpoints also achieved statistical significance (all p<0.001) [2][5] Safety and Tolerability - QTORIN™ rapamycin was well-tolerated, with no drug-related serious adverse events reported and systemic rapamycin levels below 2 ng/mL at all timepoints [6][8] - Among the 50 participants, 70% experienced treatment-emergent adverse events, but all were rated mild or moderate [8][9] Future Plans - Palvella aims to present detailed results from the SELVA study at upcoming medical meetings and is advancing QTORIN™ rapamycin for other rare skin diseases [11] - The company has received Breakthrough Therapy, Orphan Drug, and Fast Track designations from the FDA for QTORIN™ rapamycin [10]