Palvella Therapeutics (NASDAQ:PVLA) shares climbed about 15% on Tuesday after the company announced positive topline data from its Phase 3 SELVA clinical trial evaluating QTORIN™ 3.9% rapamycin anhydrous gel for the treatment of microcystic lymphatic malformations (mLM).
The late-stage study achieved its primary endpoint, demonstrating a statistically significant improvement on the Microcystic Lymphatic Malformation Investigator Global Assessment, with a mean change of +2.13 points (p<0.0001). The trial also met a key secondary endpoint, showing a statistically significant mean improvement of 3.36 points (p<0.0001) on the blinded mLM Multi-Component Static Scale.
The study enrolled 51 patients aged three years and older at vascular anomaly centers across the United States, with 50 participants initiating treatment. Among the 49 patients aged six and above who completed the 24-week efficacy evaluation period, 95% showed improvement on the mLM-IGA measure, while 86% were classified as “Much Improved” or “Very Much Improved.”
QTORIN™ rapamycin demonstrated a favorable safety profile, with no serious adverse events linked to the drug. Systemic rapamycin exposure remained below 2ng/mL at all measured timepoints. Of the patients completing the evaluation phase, 98% chose to continue treatment in the ongoing extension study.
Following the results, Palvella said it plans to submit a New Drug Application to the U.S. Food and Drug Administration in the second half of 2026, targeting potential approval in the first half of 2027. If authorized, QTORIN™ rapamycin would become the first FDA-approved therapy specifically indicated for microcystic lymphatic malformations, a rare condition estimated to affect around 30,000 people in the United States.
The therapy has previously received Breakthrough Therapy, Orphan Drug, and Fast Track designations from the FDA.