U.S. Food and Drug Administration Awards Year Two Proceeds from Orphan Products Grant Supporting Palvella Therapeutics' Phase 3 SELVA Trial of QTORIN™ Rapamycin for Microcystic Lymphatic Malformations

Core Insights - Palvella Therapeutics has received the second year of funding from the FDA's Office of Orphan Products Development, amounting to up to $2.6 million over a four-year term to support its Phase 3 SELVA trial of QTORIN™ rapamycin for treating microcystic lymphatic malformations [1][3] - The SELVA trial has exceeded its enrollment goal, with 51 subjects enrolled, and is on track to report top-line results in the first quarter of 2026, with a New Drug Application (NDA) submission planned for the second half of 2026 [2][3] Funding and Grant Details - The FDA Orphan Products Grants Program selected Palvella's Phase 3 SELVA trial as one of only seven new clinical trials funded out of 51 applications in fiscal year 2024, highlighting the significance of the trial [3] - The grant supports the development of medical products for rare diseases, with Palvella's trial being the only Phase 3 trial awarded funding [3] Clinical Trial Information - The SELVA trial is a 24-week, single-arm, baseline-controlled study designed to evaluate QTORIN™ rapamycin, which is administered once daily [2] - Eligible participants may continue treatment in an open-label extension study after the initial evaluation period [2] Regulatory Designations - QTORIN™ rapamycin has received Breakthrough Therapy, Orphan Drug, and Fast Track designations from the FDA, which may lead to seven years of orphan drug market exclusivity in the U.S. if approved [4] Company Overview - Palvella Therapeutics is focused on developing therapies for serious, rare skin diseases, with a pipeline that includes QTORIN™ rapamycin and other product candidates [5] - The company is led by veterans in rare disease drug development and aims to address significant unmet medical needs [5]