Palvella Therapeutics reported that its Phase 3 SELVA trial of QTORIN™ 3.9 % rapamycin anhydrous gel met its primary endpoint, showing a mean improvement of +2.13 on the Microcystic Lymphatic Malformation Investigator Global Assessment (mLM‑IGA) with a p‑value of less than 0.001. The result demonstrates a statistically robust benefit in a patient population that has no approved pharmacologic therapy.
All four pre‑specified secondary endpoints were also statistically significant. In the 24‑week efficacy evaluation, 95 % of participants aged six and older achieved at least a one‑point improvement, and 86 % reported being “Much Improved” or “Very Much Improved.” These high response rates underscore the clinical relevance of the treatment effect.
Safety data were favorable: no drug‑related serious adverse events were reported, and systemic rapamycin concentrations remained below 2 ng/mL throughout the study, indicating a low risk of systemic exposure with topical use.
The trial results position QTORIN™ rapamycin as the first FDA‑approved therapy for the estimated 30,000+ patients in the United States with microcystic lymphatic malformations. Palvella plans to file a New Drug Application in the second half of 2026, leveraging its Breakthrough Therapy, Orphan Drug, and Fast Track designations to expedite review.
"We are deeply grateful to the participants, families, caregivers, investigators, and study teams who made the SELVA trial possible," said Wes Kaupinen, Founder and Chief Executive Officer of Palvella Therapeutics. "The positive topline results from SELVA mark a significant milestone for Palvella and for the estimated more than 30,000 diagnosed patients in the U.S. living with microcystic LMs, a serious, rare, and chronically debilitating disease with no FDA‑approved therapies. These data support the potential for QTORIN™ rapamycin to become the first FDA‑approved therapy for microcystic LMs as we advance toward submission of a planned NDA in the second half of 2026." Dr. Joyce M. Teng, a principal investigator on the study, added, "The SELVA Phase 3 results represent an important milestone for individuals living with microcystic lymphatic malformations. For the first time, we have robust, statistically significant Phase 3 data showing that a pharmacologic targeted therapy can meaningfully improve disease severity in this chronically debilitating condition. Microcystic LMs are a congenital, progressive disease."
Palvella’s achievement de‑risks its lead asset and establishes a first‑in‑class position in a niche market with significant unmet need. The strong efficacy and safety profile, combined with regulatory designations, enhance the probability of approval and set the stage for potential expansion into other mTOR‑driven vascular and skin disorders. Investors have responded positively to the announcement, reflecting confidence in the company’s clinical trajectory and the commercial potential of QTORIN™ rapamycin.
The content on EveryTicker is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.