Core Insights - Palvella Therapeutics has successfully completed the SELVA Phase 3 trial for QTORIN™ rapamycin, which targets microcystic lymphatic malformations, a serious genetic disease with no FDA-approved therapies [2][3] - The trial enrolled 51 subjects, exceeding the initial target of 40 by over 25%, indicating strong demand and interest in this treatment [2][3] - Top-line data from the SELVA trial is expected in the first quarter of 2026, with a New Drug Application submission planned for the second half of 2026 [5] Company Overview - Palvella Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapies for rare genetic skin diseases without FDA-approved treatments [7] - The company is developing a pipeline of product candidates based on its QTORIN™ platform, with QTORIN™ rapamycin as the lead candidate currently in clinical trials [7] - The company has received Breakthrough Therapy, Orphan Drug, and Fast Track designations from the FDA for QTORIN™ rapamycin, which may qualify for seven years of orphan drug market exclusivity if approved [4] Clinical Trial Details - The SELVA trial is a 24-week, single-arm, baseline-controlled study evaluating QTORIN™ rapamycin in individuals aged three years and older with microcystic lymphatic malformations [3] - Following an 8-week baseline period, eligible participants may continue treatment in an open-label extension study, which is also open to new subjects aged three to five years [3] Disease Background - Microcystic lymphatic malformations are characterized by malformed lymphatic vessels that lead to persistent lymph fluid leakage and bleeding, often resulting in serious infections [6] - The disease affects an estimated 30,000 diagnosed patients in the United States, with symptoms worsening over time and no current FDA-approved treatments available [6]