Palvella Therapeutics, Inc. has announced the start of dosing in its Phase 3 SELVA trial, which is designed to evaluate the efficacy and safety of QTORIN™ 3.9% rapamycin anhydrous gel for the treatment of microcystic lymphatic malformations (microcystic LMs). This pivotal trial could pave the way for QTORIN™ rapamycin to become the first FDA-approved therapy for this debilitating condition.
Addressing an Unmet Need in Microcystic LMs
Microcystic LMs are a genetically driven disease affecting over 30,000 individuals in the United States. The condition is characterized by malformed lymphatic vessels, leading to persistent skin protrusions and serious infections. Currently, there are no FDA-approved treatments available, leaving a significant unmet medical need. The pathogenesis of microcystic LMs involves hyperactivation of the PI3K/mTOR pathway, resulting in substantial health challenges from birth throughout adulthood.
Trial Design and Objectives
The SELVA trial is a single-arm, baseline-controlled study that will enroll approximately 40 participants aged six years and older. These participants will be recruited from major vascular anomaly centers across the U.S. The primary endpoint of the study is to observe changes in the microcystic LM Investigator Global Assessment from baseline following 24 weeks of treatment with QTORIN™ rapamycin. The FDA has granted QTORIN™ rapamycin Breakthrough Therapy, Fast Track, and Orphan Drug designations, underscoring its potential to address this rare and challenging condition. The FDA is also supporting the study with a grant of up to $2.6 million.
Expert Perspectives
Dr. Joyce M. Teng, a leading investigator in the SELVA trial, emphasized the significant impact of microcystic LMs on affected individuals, stating the trial’s importance in addressing this unmet medical need. Wes Kaupinen, CEO of Palvella, expressed optimism about QTORIN™ rapamycin’s potential to become the first standard of care for patients with microcystic LMs. By targeting the mTOR pathway with minimal systemic exposure, QTORIN™ rapamycin aims to provide a substantial therapeutic benefit while minimizing adverse effects associated with systemic treatments.
Potential Impact
This Phase 3 trial represents a crucial milestone for Palvella Therapeutics and the broader medical community. If successful, it promises to deliver a groundbreaking treatment option for individuals suffering from microcystic LMs, offering hope for improved management and quality of life.
