WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA) is moving toward what could be a defining year, with a late-stage pipeline aimed at some of the most underserved rare skin and vascular diseases in medicine and a potential first U.S. product launch now in sight.
The company said its Phase 3 SELVA study of QTORIN™ rapamycin for microcystic lymphatic malformations remains on track, with topline results expected in March 2026. If the data are positive, Palvella plans to submit a New Drug Application in the second half of the year, setting up what could become the first FDA-approved therapy for the chronically debilitating condition.
Microcystic lymphatic malformations are caused by dysregulation of the PI3K/mTOR pathway, leading to malformed lymphatic vessels that leak, bleed and are prone to recurrent infections. No FDA-approved treatments currently exist. Palvella has estimated that more than 30,000 diagnosed patients in the United States live with the disease, while research presented in 2025 suggested the broader population with cutaneous involvement could be much higher.
The SELVA trial enrolled 51 patients, well above its original target, and includes both a baseline observation period and 24 weeks of active treatment. QTORIN™ rapamycin already holds Breakthrough Therapy, Fast Track and Orphan Drug designations for this indication, as well as an FDA orphan grant, giving the program a potentially accelerated regulatory path.
Palvella is also building momentum behind QTORIN™ rapamycin for cutaneous venous malformations, the most common form of vascular malformation in the skin. In December 2025, the company reported positive Phase 2 TOIVA results, with 73 percent of patients showing improvement on a key investigator-rated endpoint and two-thirds rated as much or very much improved. The company has asked the FDA for preliminary Breakthrough Therapy advice and plans to launch a Phase 3 trial in the second half of 2026.
Beyond those two programs, Palvella is expanding the reach of its QTORIN™ platform. A Phase 2 study of QTORIN™ rapamycin in clinically significant angiokeratomas is planned to begin in the second half of 2026, after receiving Fast Track designation late last year. The company is also advancing QTORIN™ pitavastatin for disseminated superficial actinic porokeratosis, a premalignant genetic skin disease affecting tens of thousands of patients, with a Phase 2 trial targeted for later in 2026.
Management expects to announce both a new QTORIN™ product candidate and an additional clinical indication for QTORIN™ rapamycin in the second half of the year, signaling that the platform’s expansion is far from finished.
Founder and Chief Executive Officer Wes Kaupinen said Palvella now has four programs that could become first-in-disease therapies for serious, rare conditions with no approved treatments. He also pointed to a strengthened leadership team and growing commercial infrastructure as the company prepares for a possible near-term launch in the United States.
Palvella has also shored up its intellectual property position, adding new patents in 2025 that extend protection for QTORIN™ rapamycin formulations and methods of use through 2038, while filing patents for QTORIN™ pitavastatin that could extend exclusivity into the 2040s.
For investors and rare-disease specialists alike, the next few months will be pivotal. With Phase 3 data approaching and multiple regulatory and clinical milestones lined up for 2026, Palvella is positioning itself not just as a research outfit, but as a potential commercial player in a niche where unmet medical need remains high and competition remains scarce.
For the latest news on everything happening in Chester County and the surrounding area, be sure to follow MyChesCo on Google News and MSN.