WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA) has successfully completed enrollment in the Phase 3 SELVA clinical trial of QTORIN™ 3.9% rapamycin anhydrous gel, a potential breakthrough treatment for microcystic lymphatic malformations (microcystic LMs). The study enrolled 51 participants, exceeding the original target by over 25%, signaling high interest among scientific and medical communities in this novel therapy.
“The strong demand and over-enrollment in SELVA underscore the high interest within the scientific and clinical communities in establishing a new paradigm of localized, pathogenesis-directed therapy,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella Therapeutics. “With SELVA now fully enrolled, we remain on track to deliver top-line Phase 3 data in the first quarter of 2026 to support a planned NDA submission for QTORIN™ rapamycin as the first targeted therapy for this chronically debilitating disease.”
The 24-week, single-arm trial is designed to evaluate the safety and efficacy of QTORIN™ rapamycin when applied daily to affected areas. Participants aged three and older were enrolled from prominent U.S. vascular anomaly centers. Following the core trial period, eligible participants will have the option to join an open-label extension study, which also remains open to younger patients aged three to five years who meet the inclusion criteria.
QTORIN™ rapamycin holds Breakthrough Therapy, Orphan Drug, and Fast Track designations from the U.S. Food and Drug Administration. If approved, it stands to become the first targeted therapy for microcystic LMs in the U.S., potentially benefiting more than 30,000 patients diagnosed with this debilitating condition. The drug is expected to qualify for seven years of orphan drug market exclusivity.
Microcystic lymphatic malformations are caused by dysregulation in the PI3K/mTOR pathway, resulting in malformed lymphatic vessels that leak fluid and bleed, often leading to severe infections and hospitalizations. The natural course of the disease is progressive, with no existing FDA-approved treatments available.
Palvella’s SELVA study has also received an FDA Orphan Products Grant, awarding the company up to $2.6 million to support the trial. With top-line results expected in early 2026 and a New Drug Application submission targeted for the second half of the year, QTORIN™ rapamycin could redefine the standard of care for patients with microcystic LMs.
By advancing QTORIN™ rapamycin, Palvella Therapeutics is taking meaningful steps toward addressing the significant unmet medical needs of individuals coping with this rare and chronic disease.
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