WAYNE, Pa. – Palvella Therapeutics, Inc. (Palvella), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare skin diseases for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today announced the expansion of its QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) development program into Clinically Significant Angiokeratomas.
“Based on scientific discoveries further characterizing the biology of angiokeratomas as well as published case studies on the use of off-label rapamycin, we’re excited to expand the development of QTORIN™ rapamycin into this rare, chronically debilitating disease, which currently has no FDA-approved therapies,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. “Clinically significant angiokeratomas represent our third target clinical indication for QTORIN™ rapamycin and align with Palvella’s strategy of selecting serious, rare skin diseases in which Palvella has the opportunity to introduce the first FDA-approved therapy.”
Clinically significant angiokeratomas are superficial vascular malformations of lymphatic origin which can cause bleeding, pain, functional impairment, and risk of infection, with no tendency for spontaneous regression. Angiokeratomas were recently classified as an isolated lymphatic malformation in 2025 by the International Society for the Study of Vascular Anomalies (ISSVA). Current treatment options include potentially destructive procedural interventions that carry significant risks of pain, scarring, and recurrence. Despite the substantial disease burden, there are currently no FDA-approved treatments available for clinically significant angiokeratomas.
Palvella plans to meet with the FDA in the first half of 2026 to discuss the proposed design of a Phase 2 study of approximately 10-20 patients to evaluate QTORIN™ rapamycin for the treatment of clinically significant angiokeratomas. Study initiation is anticipated in the second half of 2026. Additionally, QTORIN™ rapamycin is currently being evaluated for microcystic lymphatic malformations in the Phase 3 SELVA study and cutaneous venous malformations in the Phase 2 TOIVA study. SELVA top-line results are anticipated in the first quarter of 2026 and TOIVA top-line results are expected in mid-December 2025.
About Palvella Therapeutics
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 patients suffering from serious, rare skin diseases for which there are no FDA-approved therapies. Palvella is developing a broad pipeline of product candidates based on its patented QTORIN™ platform, with an initial focus on serious, rare skin diseases, many of which are lifelong in nature. Palvella’s lead product candidate, QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), is currently being evaluated in the Phase 3 SELVA clinical trial in microcystic lymphatic malformations and the Phase 2 TOIVA clinical trial in cutaneous venous malformations. For more information, please visit www.palvellatx.com or follow Palvella on LinkedIn or X (formerly known as Twitter).
QTORIN™ rapamycin is for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency for any indication.
Investors Contact
Wesley H. Kaupinen
Founder and CEO, Palvella Therapeutics
[email protected]
Media Contact
Marcy Nanus
Managing Partner, Trilon Advisors LLC
[email protected]