WAYNE, PA — Palvella Therapeutics Inc. (Nasdaq: PVLA) said it achieved the primary and all secondary endpoints in a Phase 3 trial of QTORIN 3.9% rapamycin gel for microcystic lymphatic malformations and plans to submit a New Drug Application to the U.S. Food and Drug Administration in the second half of 2026.
The single-arm, baseline-controlled SELVA study evaluated once-daily QTORIN rapamycin in 51 participants age 3 and older, with efficacy results reported for 49 patients age 6 and older in the intent-to-treat population.
At Week 24, the treatment met the primary endpoint, showing a mean improvement of 2.13 points on the Microcystic Lymphatic Malformation Investigator Global Assessment scale, with statistical significance (p<0.001). Ninety-five percent of participants who completed the evaluation period showed at least a one-point improvement, and 86% were rated “Much Improved” or “Very Much Improved,” the company said.
The trial also met its pre-specified key secondary endpoint and four additional secondary endpoints, all with p-values less than 0.001.
Microcystic lymphatic malformations are congenital, progressive vascular anomalies that can cause leakage, infection and functional impairment. There are currently no FDA-approved therapies for the condition in the United States.
“The SELVA Phase 3 results represent an important milestone for individuals living with microcystic lymphatic malformations,” said Joyce M. Teng, M.D., Ph.D., a principal investigator in the trial and professor at Stanford University School of Medicine. She said the data show that a targeted therapy can improve disease severity in the condition.
Palvella said QTORIN rapamycin was generally well tolerated. Among 50 participants who initiated treatment, 70% experienced treatment-emergent adverse events, most of which were mild or moderate. Four serious adverse events were reported and deemed unrelated to the study drug by investigators. Systemic rapamycin levels remained below 2 ng/mL at all measured timepoints, the company said.
Eighty-eight percent of participants completed the 24-week evaluation period, and 98% of eligible participants elected to continue in an ongoing extension study.
Wes Kaupinen, founder and chief executive officer of Palvella, said the company plans to seek FDA approval based on the results. QTORIN rapamycin has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for microcystic lymphatic malformations.
If approved, QTORIN rapamycin would become the first FDA-approved therapy for the estimated more than 30,000 people in the United States diagnosed with microcystic lymphatic malformations, the company said.
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