Palvella Nears Goal Line for First-Ever Rare Skin Disease Therapy

WAYNE, PA – January 09, 2026 – Palvella Therapeutics is on the verge of a potential breakthrough for thousands of patients living with debilitating rare skin diseases, as the company announced its lead drug candidate is nearing the end of late-stage clinical trials. In a corporate update outlining its strategic outlook for 2026, the biopharmaceutical firm detailed significant progress across its pipeline, signaling a pivotal year ahead in its quest to deliver the first-ever FDA-approved treatments for several underserved conditions.

The company's lead candidate, QTORIN™ rapamycin anhydrous gel, is in the final stages of a Phase 3 study for microcystic lymphatic malformations (LMs), a rare and chronic genetic disease with no approved therapies. With topline results expected in March 2026, Palvella is accelerating its preparations for a potential U.S. market launch, a move that could transform the standard of care for this and other serious skin disorders.

The Vanguard Treatment for a Lifelong Burden

At the forefront of Palvella’s efforts is the Phase 3 SELVA study for QTORIN™ rapamycin in treating microcystic LMs. This rare disease, caused by a genetic mutation, leads to malformed lymphatic vessels that leak fluid and bleed, causing persistent pain, disfigurement, and recurrent, severe infections that can require hospitalization. For the more than 30,000 diagnosed patients in the U.S., life is a constant battle against symptoms with no approved therapeutic recourse.

The SELVA study (NCT06239480), a single-arm, baseline-controlled trial, has already surpassed its enrollment target by over 25%, with 51 subjects participating. The study's design involves an eight-week observation period followed by 24 weeks of treatment with the once-daily topical gel. The strong interest and enrollment highlight the significant unmet need within the patient community.

Palvella anticipates releasing topline data from this pivotal trial in March 2026. If the results are positive, the company plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the second half of the year. The agency has already recognized the program's potential by granting it Breakthrough Therapy, Fast Track, and Orphan Drug designations. This suite of designations is reserved for therapies that may demonstrate substantial improvement over available options for serious conditions and helps to expedite the development and review process. Further validating the program's importance, the FDA also awarded it an Orphan Products Grant of up to $2.6 million.

“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,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella Therapeutics. He noted that if approved, QTORIN™ rapamycin "has the potential... to become the first FDA-approved therapy and a first-line, standard-of-care treatment."

A Platform of Possibilities

While microcystic LMs represent the most immediate opportunity, Palvella is strategically leveraging its proprietary QTORIN™ platform to build a broad pipeline targeting multiple rare conditions. The platform is designed to deliver established active pharmaceutical ingredients, like rapamycin, through a specialized topical gel, concentrating the therapeutic effect on the skin while minimizing systemic exposure.

This approach has shown promise in a Phase 2 trial for cutaneous venous malformations (VMs), another rare genetic disorder affecting an estimated 130,000 people in the U.S. In December 2025, the company reported positive results from the TOIVA study, where 73% of participants showed improvement at week 12, and 67% were rated by clinicians as “Much Improved” or “Very Much Improved.” Following this success, Palvella has requested a meeting with the FDA to discuss a potential Breakthrough Therapy Designation and plans to initiate a pivotal Phase 3 study in the second half of 2026.

The pipeline expansion doesn't stop there. Palvella is advancing QTORIN™ rapamycin into a third indication, clinically significant angiokeratomas, a condition affecting over 50,000 U.S. patients that can cause bleeding, pain, and infection. The FDA granted this program Fast Track Designation in December 2025, and a Phase 2 study is expected to begin later this year.

Furthermore, the company has introduced a second product candidate from its platform: QTORIN™ pitavastatin. This therapy is being developed for disseminated superficial actinic porokeratosis (DSAP), a pre-malignant genetic skin disease affecting another 50,000 diagnosed patients. By topically inhibiting the causal mevalonate pathway, it aims to be the first pathogenesis-directed therapy for the condition. A Phase 2 trial is slated to start in the second half of 2026. Palvella has also teased plans to announce a fourth indication for QTORIN™ rapamycin and a third product candidate from the QTORIN™ platform later in the year, underscoring its long-term growth strategy.

Building for a Commercial Breakthrough

To navigate the path from clinical development to market, Palvella has significantly strengthened its leadership team with seasoned industry veterans. This strategic hiring spree signals the company’s transition from a research-focused entity to a commercial-ready organization.

Key appointments include Ashley Kline as Chief Commercial Officer, who previously led the successful U.S. launch of the orphan drug OXERVATE® at Dompé, growing sales to over $500 million annually. Her experience is directly applicable to Palvella's goal of launching a first-in-disease therapy. She is joined by David Osborne, Ph.D., as Chief Innovation Officer, a co-founder of Arcutis Biotherapeutics who has been involved in the development of over three dozen approved topical drugs.

The company has also brought on Vimal Patel, PharmD, to lead Medical Affairs, and Jason Burdette to oversee Technical Operations and manufacturing, both bringing extensive experience from companies like Incyte and Aimmune Therapeutics. This newly assembled team provides Palvella with the cross-functional expertise required to manage late-stage trials, navigate regulatory submissions, scale up manufacturing, and execute a successful commercial launch. The company is actively building out its market access and sales leadership teams in anticipation of a potential approval for QTORIN™ rapamycin.

This focus on commercial readiness is supported by a robust intellectual property strategy. In 2025, Palvella secured two new U.S. patents for QTORIN™ rapamycin, extending its exclusivity through 2038. It also filed a patent for QTORIN™ pitavastatin, aiming for protection through 2046. This multi-layered approach, combining patents with trade secrets and potential regulatory exclusivities like orphan drug status, is designed to protect its innovations and secure its market position for years to come, providing a solid foundation as it prepares to bring its potential first-in-class therapies to patients in need.