Palvella Therapeutics Awarded Up to $2.6 million Grant from the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development to Support Phase 3 Single-Arm, Baseline-Controlled Trial in Microcystic Lymphatic Malformations

Core Insights - Palvella Therapeutics has received a grant of up to $2.6 million from the FDA to support its Phase 3 SELVA trial of QTORIN™ 3.9% rapamycin anhydrous gel for treating microcystic lymphatic malformations [1][2] - The grant highlights the significant unmet medical need for effective treatments for microcystic lymphatic malformations, a rare genetic disease affecting over 30,000 patients in the U.S. [2][3] - QTORIN rapamycin is positioned to be the first approved therapy for this condition, with FDA designations including Breakthrough Therapy, Fast Track, and Orphan Drug [5][8] Company Overview - Palvella Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapies for serious, rare genetic skin diseases without FDA-approved treatments [8] - The company utilizes its patented QTORIN™ platform to create topical therapies that minimize systemic absorption and adverse effects [4][5] - Palvella's lead product candidate, QTORIN rapamycin, is under development for microcystic lymphatic malformations and other skin diseases driven by mTOR pathway overactivation [5][8] Clinical Development - The SELVA trial is a 24-week, single-arm, baseline-controlled Phase 3 clinical trial expected to enroll 40 subjects at leading vascular anomaly centers across the U.S. [7] - The primary and key secondary endpoints of the trial are clinician-reported outcomes [7] - QTORIN rapamycin is currently for investigational use only and has not yet received FDA approval [7] Market Context - The FDA Orphan Products Grants Program awarded grants to only seven out of 51 applications in fiscal year 2024, indicating a competitive landscape for funding in rare disease research [2] - The program has historically facilitated the approval of over 85 products, underscoring its importance in advancing treatments for rare diseases [2]