WAYNE, Pa. — February 2, 2026 — Leads & Copy —
Palvella Therapeutics, Inc. (Nasdaq: PVLA), a biopharmaceutical company focused on developing therapies for rare skin diseases and vascular malformations, has announced the publication of a systematic review in Clinical and Experimental Dermatology regarding off-label statin use for cutaneous application in porokeratosis. The review supports the development of Palvella’s QTORIN™ pitavastatin for disseminated superficial actinic porokeratosis (DSAP), with Phase 2 development anticipated in the second half of 2026.
The review, titled “Topical Statins in the Treatment of Porokeratosis: A Systematic Review,” analyzed 24 studies involving 95 patients treated with topical statins, including atorvastatin, fluvastatin, lovastatin, rosuvastatin, and simvastatin. The journal is published by Oxford University Press on behalf of the British Association of Dermatologists.
Dr. Maria Gnarra Buethe, Pediatric Dermatology Director at the University of California, Irvine, noted the risk of malignant transformation in DSAP patients and highlighted advances in understanding the mevalonate pathway as central to the disease. While systemic statins have shown limited benefit, the review supports topical statins as a targeted therapeutic approach.
The review identified loss-of-function mutations in the mevalonate metabolic pathway as a major driver of porokeratosis, leading to off-label statin use to inhibit HMG-CoA reductase. Most patients in the review experienced at least partial clinical benefit, including symptom relief and lesion size reduction, across a broad age range (2 to 85 years).
Dr. Jeff Martini, Chief Scientific Officer of Palvella, emphasized the unmet need in porokeratosis, with no FDA-approved therapies. He noted the potential role for QTORIN™ pitavastatin and validated Palvella’s approach to addressing DSAP, which affects an estimated 50,000 diagnosed patients in the U.S.
Palvella Therapeutics is focused on developing therapies for serious, rare skin diseases and vascular malformations. Their lead product candidate, QTORIN™ 3.9% rapamycin anhydrous gel, is being developed for microcystic lymphatic malformations, cutaneous venous malformations, and angiokeratomas.
QTORIN™ rapamycin and QTORIN™ pitavastatin are for investigational use only and have not been approved by the FDA.
Authors on the publication include Dr. Maria Gnarra Buethe, Lihi Atzmony Maoz, MD, and Jeff Martini, PhD.
Source: Palvella Therapeutics
