WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA) said a newly published systematic review strengthens the scientific case for using topical statins to treat porokeratosis, a rare and progressive genetic skin disease with no FDA-approved therapies.
The review, published in the peer-reviewed journal Clinical and Experimental Dermatology, analyzes existing clinical evidence on off-label statin use applied directly to the skin. The findings support the development of Palvella’s investigational therapy, QTORIN pitavastatin, for disseminated superficial actinic porokeratosis, or DSAP, with Phase 2 development anticipated in the second half of 2026.
Porokeratosis is marked by expanding skin lesions and carries a risk of malignant transformation, particularly in patients with DSAP. Researchers have increasingly linked the disease to mutations in the mevalonate metabolic pathway, a biological process targeted by statins.
Maria Gnarra Buethe, MD, PhD, pediatric dermatology director at the University of California, Irvine, and division chief of dermatology at Rady Children’s Hospital of Orange County, said the review highlights why topical delivery may succeed where oral statins have not. She said systemic statins likely failed to show benefit because of limited penetration into the skin, while topical formulations may better address the underlying disease mechanism.
The publication, titled “Topical Statins in the Treatment of Porokeratosis: A Systematic Review,” examined 24 studies involving 95 patients treated with topical versions of statins originally developed for systemic use, including atorvastatin, fluvastatin, lovastatin, rosuvastatin, and simvastatin. Across a broad age range from 2 to 85 years, most patients experienced at least partial clinical improvement, including symptom relief and reduced lesion size.
The authors also emphasized the need for controlled clinical trials using standardized formulations and endpoints to more rigorously assess safety and effectiveness. Dr. Gnarra Buethe, Lihi Atzmony Maoz, MD of Yale School of Medicine, and Jeff Martini, PhD, Palvella’s chief scientific officer, served as authors on the review.
Martini said the findings underscore the unmet need in porokeratosis and reinforce Palvella’s strategy. He noted that DSAP, the most common subtype, affects an estimated more than 50,000 diagnosed patients in the United States and significantly impacts quality of life.
Clinical and Experimental Dermatology is published by Oxford University Press on behalf of the British Association of Dermatologists and features clinically focused research aimed at advancing the understanding and treatment of skin disease worldwide.
For the latest news on everything happening in Chester County and the surrounding area, be sure to follow MyChesCo on Google News and MSN.