Palvella Therapeutics announces publication of literature review of rapamycin

Palvella Therapeutics (PVLA) announced the publication of a manuscript in Lymphatic Research and Biology which summarizes a systematic literature review of rapamycin for the treatment of venous malformations. The manuscript supports the scientific rationale and clinical potential of Palvella’s QTORIN 3.9% rapamycin anhydrous gel for the treatment of cutaneous VMs, currently under evaluation in the Phase 2 TOIVA trial with top-line data anticipated in mid-December 2025. Key findings from the publication include: Recent advances in the pathogenesis of VMs have implicated abnormal activation of the phosphatidylinositol 3-kinase/protein kinase B/mammalian target of rapamycin signaling pathway. Interventional treatments, including surgery, sclerotherapy, and laser therapy, are highlighted as potentially inadequate for VMs patients with extensive, cutaneous, or invasive disease. The majority of patients showed evidence of treatment efficacy with off-label rapamycin. A targeted, topical formulation of rapamycin could provide clinical benefit for cutaneous VMs by achieving therapeutic levels in the dermis while minimizing the risk of off-target effects of oral rapamycin.