Palvella Therapeutics has announced the planned pivotal Phase 3 study design for QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) in the treatment of Microcystic Lymphatic Malformations. The announcement was made by the clinical-stage biopharmaceutical company, which aims to address the lack of FDA-approved therapies for this rare genetic disease. The Phase 2 results demonstrated significant improvement in all participants treated with QTORIN rapamycin, positioning it as a potential breakthrough therapy for Microcystic LMs. With an estimated 30,000 individuals affected by this condition in the U.S., Palvella sees a promising commercial opportunity.
In addition to the Phase 3 study design, Palvella also shared topline results from the Phase 2b CODY study, which evaluated QTORIN's efficacy and safety profile.