Palvella Therapeutics’ qtorin meets primary endpoint in lymphatic malformations

Palvella Therapeutics (PVLA) announced positive topline results from the company’s Phase 3 SELVA study of qtorin 3.9% rapamycin anhydrous gel for the treatment of microcystic lymphatic malformations. The Phase 3 trial met its primary endpoint with statistically significant improvement on the Microcystic Lymphatic Malformation Investigator Global Assessment. The study also met its pre-specified key secondary endpoint and all four additional secondary endpoints with statistical significance. Based on these results, Palvella plans to submit an NDA to FDA for qtorin rapamycin for patients with microcystic LMs in the second half of 2026, with potential U.S. approval for qtorin rapamycin in the first half of 2027. If approved, qtorin rapamycin would be the first approved therapy for patients with microcystic LMs. On the mLM-IGA at Week 24 in the ITT population, qtorin rapamycin demonstrated a mean improvement of +2.13 points, meeting the study’s primary endpoint. Qtorin rapamycin was well-tolerated. Amongst the 50 participants who initiated treatment, 35 participants experienced treatment-emergent adverse events. Qtorin rapamycin has received Breakthrough Therapy, Orphan Drug, and Fast Track designations from the FDA for the treatment of microcystic LMs, as well as an FDA Orphan Products Development grant. Palvella is also advancing qtorin rapamycin in other serious, rare skin diseases and vascular malformations.