WAYNE, Pa. — January 9, 2026 — Leads & Copy — Palvella Therapeutics, Inc. (Nasdaq: PVLA), a biopharmaceutical company focused on developing therapies for rare skin diseases and vascular malformations, has provided a corporate update and strategic outlook for 2026.
The Phase 3 SELVA study evaluating QTORIN™ rapamycin 3.9% anhydrous gel (QTORIN™ rapamycin) for microcystic lymphatic malformations (microcystic LMs) is on track, with topline results expected in March 2026. Pending positive results, an NDA submission is planned for the second half of 2026.
Palvella is accelerating U.S. launch readiness for QTORIN™ rapamycin for microcystic LMs, which could become the first FDA-approved therapy for this disease affecting over 30,000 diagnosed patients in the U.S.
Following positive Phase 2 results for QTORIN™ rapamycin in treating cutaneous venous malformations announced in December 2025, the company requested a Preliminary Breakthrough Therapy Designation Advice meeting with the U.S. Food and Drug Administration, anticipated in the first quarter of 2026.
Phase 2 clinical studies evaluating QTORIN™ pitavastatin in disseminated superficial actinic porokeratosis and QTORIN™ rapamycin in clinically significant angiokeratomas are expected to begin in the second half of 2026.
Palvella anticipates announcing a new QTORIN™ product candidate and a fourth clinical indication for QTORIN™ rapamycin in the second half of 2026.
“As we continue to advance our leadership in addressing serious, rare skin diseases and vascular malformations, we look forward to reporting Phase 3 SELVA topline results later this quarter for microcystic lymphatic malformations where QTORIN™ rapamycin has the potential, if approved, to become the first FDA-approved therapy and a first-line, standard-of-care treatment,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella Therapeutics.
Kaupinen added that Palvella has four programs with the potential to be first-in-disease therapies for serious, rare conditions with no FDA-approved treatments in commercially attractive markets, including microcystic lymphatic malformations, cutaneous venous malformations, clinically significant angiokeratomas, and disseminated superficial actinic porokeratosis.
Palvella has strengthened its leadership team and is pleased with progress across R&D efforts and commercial preparations for a potential near-term U.S. launch.
Microcystic LMs are a rare genetic disease characterized by malformed lymphatic vessels that protrude through the skin and leak lymph fluid and bleed, leading to recurrent infections and cellulitis that can cause hospitalization. No FDA-approved therapies currently exist for microcystic LMs.
In May 2025, data presented at the 82nd Annual Meeting of the Society for Investigative Dermatology indicated an estimated 44,553 to 92,967 diagnosed U.S. patients with lymphatic malformations with cutaneous involvement.
In June 2025, Palvella surpassed its target enrollment in the Phase 3 SELVA trial, enrolling 51 subjects in the study evaluating once-daily QTORIN™ rapamycin in individuals aged three years and older with microcystic LMs.
Cutaneous VMs are a rare genetic disease leading to dysfunctional veins within the skin, causing morbidity and functional impairment, and are associated with severe bleeding, thrombosis, and pain. No FDA-approved therapies currently exist for the disease.
In October 2025, a study published in Orphanet Journal of Rare Diseases indicated an estimated annual prevalence of over 130,000 diagnosed U.S. patients with cutaneous only venous malformations.
In December 2025, Palvella announced positive topline efficacy results from the Phase 2 TOIVA study, achieving statistical significance on multiple efficacy endpoints.
Palvella plans to commence a Phase 3 pivotal study in the second half of 2026.
In September 2025, Palvella expanded the development of QTORIN™ rapamycin into clinically significant angiokeratomas, with no FDA-approved therapies currently existing for the estimated more than 50,000 diagnosed U.S. patients.
In December 2025, the FDA granted Fast Track Designation to QTORIN™ rapamycin for angiokeratomas.
Palvella plans to meet with the FDA in the first quarter of 2026 to discuss the design of a Phase 2 study, with study initiation expected in the second half of 2026.
In November 2025, Palvella announced QTORIN™ pitavastatin for the treatment of disseminated superficial actinic porokeratosis, with no FDA-approved therapies currently existing for the estimated more than 50,000 diagnosed patients in the U.S.
Palvella plans to meet with the FDA in the first half of 2026 to discuss the proposed design of a Phase 2 clinical trial evaluating QTORIN™ pitavastatin, with study initiation expected in the second half of 2026.
Palvella plans to announce the fourth target clinical indication for QTORIN™ rapamycin and the third product candidate from the QTORIN™ platform in the second half of 2026.
In 2025, Palvella expanded its intellectual property portfolio with two U.S. patents for QTORIN™ rapamycin and filed a patent for its QTORIN™ pitavastatin program.
Key leadership hires have strengthened Palvella’s capabilities, including Ashley Kline as Chief Commercial Officer, David Osborne, Ph.D., as Chief Innovation Officer, Vimal Patel, PharmD, as Senior Vice President of Medical Affairs, Jason Burdette as Senior Vice President of Technical Operations, Peter Finlayson as Vice President of Marketing, and Shama Munim as Senior Director of Regulatory Affairs.
Palvella is strengthening medical affairs and commercial leadership in anticipation of a potential U.S. launch of QTORIN™ rapamycin for microcystic lymphatic malformations, if approved.
QTORIN™ rapamycin and QTORIN™ pitavastatin are for investigational use only and have not been approved by the FDA or any other regulatory agency.
Contact Information
Investors
Wesley H. Kaupinen
Founder and CEO, Palvella Therapeutics
wes.kaupinen@palvellatx.com
Media
Marcy Nanus
Managing Partner, Trilon Advisors LLC
mnanus@trilonadvisors.com
Source: Palvella Therapeutics
