WAYNE, PA — Palvella Therapeutics, Inc. has announced the commencement of dosing in its Phase 3 SELVA trial, aimed at evaluating QTORINTM 3.9% rapamycin anhydrous gel for treating microcystic lymphatic malformations (microcystic LMs).
This trial marks a crucial step towards potentially establishing QTORINTM 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 QTORINTM 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 .