WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA) announced the expansion of SELVA, its Phase 3 clinical trial of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), to include children as young as 3 years old. Previously, participation was limited to those aged 6 or older. The decision, approved by the U.S. Food and Drug Administration (FDA), underscores Palvella’s commitment to addressing microcystic lymphatic malformations (microcystic LMs) in the youngest patients impacted by this debilitating disease.
QTORIN™ rapamycin has the potential to become the first FDA-approved therapy for microcystic LMs, a chronic and progressive genetic condition that affects more than 30,000 individuals in the U.S. This rare disease is caused by abnormalities in the PI3K/mTOR signaling pathway, leading to malformed lymphatic vessels that leak fluid, bleed, and often cause infections, pain, and significant disfigurement. Without treatment, symptoms can escalate over a lifetime.
“Microcystic LMs is a debilitating disease that is chronic, progressive, and usually present at birth or shortly after,” said Dr. Joyce Teng, Professor of Dermatology and Pediatrics at Stanford University and Principal Investigator of the SELVA Study. “Early intervention is essential to minimize disease burden during children’s development, which is why I am so excited by the opportunity QTORIN™ rapamycin presents to the younger pediatric population.”
The SELVA trial, now enrolling 40 patients, employs a 24-week, single-arm, baseline-controlled design to assess the safety and effectiveness of QTORIN™ rapamycin. Backed by the FDA’s Breakthrough Therapy, Fast Track, and Orphan Drug designations, as well as an Orphan Products Grant of up to $2.6 million, the trial remains on track to report top-line results in the first quarter of 2026.
Palvella Therapeutics’ work with QTORIN™ rapamycin represents a significant step forward for families and patients managing microcystic LMs. By broadening clinical access and aiming to deliver the first standard-of-care treatment, the company is positioned to transform outcomes for those affected by this severe condition. The SELVA trial and its potential could mark a pivotal moment in addressing this rare disease and improving lives.
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