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Palvella Therapeutics Announces Scientific Publication in Lymphatic Research and Biology Highlighting Recent Advances in the Pathogenesis of Venous Malformations and the Real-World Use of Rapamycin as an Emerging Targeted Therapy
Recent advances in understanding venous malformation disease pathogenesis highlight the PI3K/AKT/mTOR pathway as a key driver of disease proliferation, spurring real-world off-label use of systemic rapamycin (sirolimus) Publication includes a systematic review of 26 studies evaluating the use of rapamycin for the treatment of venous malformations Study authors highlight unmet clinical need for venous malformations and lack of FDA-approved pharmacologic options while advocating for an FDA-approve
About this update from Palvella Therapeutics, Inc.
[{"type":"image","alt":"Palvella Therapeutics Inc.","displaySize":"","headline":null,"caption":"Palvella Therapeutics Inc.","className":"","disableSlideshowImg":false,"size":{"original":{"width":300,"height":300,"url":"https://media.zenfs.com/en/globenewswire.com/4b273ae029a5e8cb06a3771e7b41e04d"},"resized":{"url":"https://s.yimg.com/ny/api/res/1.2/IZ4VZ0Qdm50kE5TTbiIBBg--/YXBwaWQ9aGlnaGxhbmRlcjt3PTQyMDtoPTQyMDtjZj13ZWJw/https://media.zenfs.com/en/globenewswire.com/4b273ae029a5e8cb06a3771e7b41e04d","width":300,"height":300}},"lazy":false},{"type":"text","content":"Recent advances in understanding venous malformation disease pathogenesis highlight the PI3K/AKT/mTOR pathway as a key driver of disease proliferation, spurring real-world off-label use of systemic rapamycin (sirolimus)","length":219,"tagName":"p"},{"type":"text","content":"Publication includes a systematic review of 26 studies evaluating the use of rapamycin for the treatment of venous malformations","length":128,"tagName":"p"},{"type":"text","content":"Study authors highlight unmet clinical need for venous malformations and lack of FDA-approved pharmacologic options while advocating for an FDA-approved targeted topical formulation of rapamycin for patients with cutaneous venous malformations","length":243,"tagName":"p"},{"type":"text","content":"WAYNE, Pa., Sept. 17, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) Palvella Therapeutics, Inc. (Palvella), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today 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 (VMs). The manuscript supports the scientific rationale and clinical potential of Palvella’s QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of cutaneous VMs, currently under evaluation in the Phase 2 TOIVA trial with top-line data anticipated in mid-December 2025.","length":842,"tagName":"p"},{"type":"text","content":""This publication highlights the significant unmet need in the management of cutaneous venous malformations and the large, growing real-world experien...