WAYNE, PA — Palvella Therapeutics, Inc. has announced the commencement of dosing in its Phase 3 SELVA trial, aimed at evaluating QTORIN™ 3.9% rapamycin anhydrous gel for treating microcystic lymphatic malformations (microcystic LMs). This trial marks a crucial step towards potentially establishing QTORIN™ rapamycin as the first approved treatment for this debilitating condition.
Microcystic LMs are a genetically driven disease affecting over 30,000 individuals in the U.S. Characterized by malformed lymphatic vessels that cause persistent skin protrusions and serious infections, this condition currently lacks any FDA-approved treatments. The disorder results from the hyperactivation of the PI3K/mTOR pathway, leading to significant health challenges from birth through adulthood.
The SELVA trial is a single-arm, baseline-controlled study enrolling approximately 40 participants, aged six and above, across major vascular anomaly centers in the U.S. The main goal is to observe changes in the microcystic LM Investigator Global Assessment from baseline after 24 weeks of treatment. The FDA has granted QTORIN™ rapamycin Breakthrough Therapy, Fast Track, and Orphan Drug designations, underlining its potential impact.
Dr. Joyce M. Teng, a leading investigator in the study, highlights the severe impact of microcystic LMs, emphasizing the trial’s importance in addressing this unmet medical need. The FDA has also supported the study with a grant of up to $2.6 million to aid research efforts.
Palvella’s CEO, Wes Kaupinen, expressed optimism about QTORIN™ rapamycin’s potential to become the first standard care option for patients suffering from microcystic LMs. By targeting the mTOR pathway with minimal systemic exposure, QTORIN™ rapamycin aims to offer a significant therapeutic benefit while reducing adverse effects linked to systemic treatments.
This trial represents a pivotal moment for Palvella and the broader medical community, as it seeks to provide a groundbreaking treatment option for those affected by this challenging condition.
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