There are currently no FDA-approved treatments for the estimated more than 30,000 diagnosed patients with microcystic LMs in the United States. ... Founded and led by rare disease drug development veterans, Palvella Therapeutics, Inc. (Nasdaq: PVLA) is a clinical-stage biopharmaceutical company ... There are currently no FDA-approved treatments for the estimated more than 30,000 diagnosed patients with microcystic LMs in the United States. ... Founded and led by rare disease drug development veterans, Palvella Therapeutics, Inc. (Nasdaq: PVLA) is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat participants suffering from serious, rare skin diseases and vascular malformations for which there are no FDA-approved therapies. Primary endpoint met with statistically significant improvement (mean change of +2.13; p (Palvella or “the Company”), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat participants suffering from serious, rare skin diseases and vascular malformations for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today announced positive topline results from the Company’s Phase 3 SELVA study of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of microcystic lymphatic malformations (microcystic LMs). If approved, QTORIN™ rapamycin would be the first approved therapy for patients with microcystic LMs. SELVA is a Phase 3, single-arm, baseline-controlled clinical trial evaluating once-daily QTORIN™ rapamycin in individuals aged ≥ 3 years with microcystic LMs. Of the 51 participants enrolled, 50 initiated treatment, including 49 participants aged ≥ 6 years and 1 participant in the exploratory 3- to 5-year-old cohort.

Original title: “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”