WAYNE, Pa. — (Nasdaq: PVLA) Palvella Therapeutics, Inc. (Palvella or “the Company”), 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 that the FDA has granted Fast Track Designation to QTORIN™ rapamycin for the treatment of angiokeratomas.
“We are thrilled that the FDA has granted Fast Track Designation for QTORIN™ rapamycin in angiokeratomas,” said Wes Kaupinen, Founder and CEO of Palvella. “Angiokeratomas are chronic, often debilitating lesions with no FDA-approved therapies. This Fast Track designation underscores the potential of QTORIN™ rapamycin to address this significant unmet need and supports our opportunity to advance what could become the first FDA-approved therapy for these patients. We are committed to working closely with the FDA to advance development with urgency.”
Clinically significant angiokeratomas are superficial vascular malformations of lymphatic origin that can cause bleeding, pain, functional impairment, and risk of infection, with no tendency for spontaneous regression. In 2025, the International Society for the Study of Vascular Anomalies reclassified angiokeratomas as an isolated lymphatic malformation, reflecting advancements in understanding of the disease’s underlying biology. Current treatment options are limited to potentially destructive procedural interventions that carry meaningful risks of pain, scarring, and recurrence.
The FDA’s Fast Track program is designed to facilitate the development, and expedite the review, of drugs to treat serious conditions and fill an unmet need. The purpose is to get important new drugs to the patient earlier. Once a drug receives Fast Track designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients. If relevant criteria are met, programs with Fast Track designation can become eligible for accelerated approval and priority review, both of which can reduce the timelines associated with regulatory review and action.
In September 2025, Palvella announced the expansion of its QTORIN™ rapamycin development program into 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, with study initiation expected in the second half of 2026.
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 developed for the treatment of microcystic lymphatic malformations, cutaneous venous malformations, and clinically significant angiokeratomas. Palvella’s second product candidate, QTORIN™ pitavastatin, is currently being developed for the topical treatment of disseminated superficial actinic porokeratosis. For more information, please visit www.palvellatx.com or follow Palvella on LinkedIn or X (formerly known as Twitter).
QTORIN™ rapamycin and QTORIN™ pitavastatin are for investigational use only and neither has been approved or cleared by the FDA or by any other regulatory agency for any indication.
Contact Information
Investors
Wesley H. Kaupinen
Founder and CEO, Palvella Therapeutics
[email protected]
Media
Marcy Nanus
Managing Partner, Trilon Advisors LLC
[email protected]