WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA) has received the initial proceeds from a prestigious FDA Orphan Products Development grant to support its ongoing Phase 3 SELVA trial of QTORIN™ 3.9% rapamycin anhydrous gel for the treatment of microcystic lymphatic malformations (microcystic LMs). The grant, part of a highly competitive federal funding program, provides up to $2.6 million in non-dilutive funding over its duration.
The SELVA trial is a pivotal 24-week, single-arm, baseline-controlled study evaluating the safety and efficacy of QTORIN™ rapamycin. Administered once daily, the treatment aims to address microcystic LMs, a rare genetically driven disease characterized by malformed, leaking lymphatic vessels. Conducted across top vascular anomaly centers in the U.S., the trial is enrolling approximately 40 participants aged three and older, with top-line data expected in the first quarter of 2026.
“We are honored to receive this prestigious FDA grant and the initial non-dilutive funding,” said Wes Kaupinen, Founder and CEO of Palvella. “There are currently no FDA-approved therapies for microcystic lymphatic malformations, a rare and chronically debilitating genetic disease. QTORIN™ rapamycin has the potential to become the first approved therapy—and a transformative new standard of care—for individuals living with this disease.”
The FDA’s Orphan Products Grants Program funds the development of promising treatments for rare diseases, with strict scientific and technical evaluation by an independent panel of experts. Palvella’s Phase 3 SELVA trial was one of only seven clinical trials awarded support in fiscal year 2024 and the only Phase 3 study selected. Since the program’s inception, it has helped advance research that has led to the approval of over 85 therapies.
Microcystic LMs are a severe condition caused by dysregulation of the PI3K/mTOR pathway, resulting in malformed lymphatic vessels that leak fluid, bleed, and introduce significant risks of infection. Affecting more than 30,000 patients in the U.S., the disease is progressive and leads to chronic complications without approved treatment options.
QTORIN™ rapamycin represents a significant step forward for these patients, offering promise for both symptom relief and improved quality of life. Palvella’s leadership in advancing rare disease therapies underscores its commitment to addressing unmet medical needs and pioneering innovative solutions in this space.
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