Palvella Therapeutics Announces Abstract Highlighting the Estimated Diagnosed Prevalence and Annual Incidence of Lymphatic Malformations in the U.S. Accepted for Poster Presentation at the 82nd Annual Meeting of the Society for Investigative Dermatology
Quantitative analysis of medical claims indicates an estimated 44,553 to 92,967 diagnosed U.S. patients with lymphatic malformations (LMs) with cutaneous involvement
Estimates of annual incidence of LMs with cutaneous involvement to be highlighted in the poster presentation
Poster to be presented on Thursday, May 8, 2025, 4:30-6:00 pm PT
WAYNE, Pa., April 23, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) Palvella Therapeutics, Inc. (Palvella or “the Company”), 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 an abstract highlighting the estimated diagnosed prevalence and annual incidence of lymphatic malformation (LM) cases in the U.S. was accepted for poster presentation at the Society for Investigative Dermatology (SID) Annual Meeting, taking place May 7-10, 2025, in San Diego, California.
The abstract includes a medical claims-based analysis over a 7-year period conducted with data from Komodo Health. The analysis was aimed at determining the number of LM cases with cutaneous involvement in the U.S., with the following highlights:
Claims from diagnostic codes that are recommended for use with LM patients by the Vascular Anomalies Special Interest Group of the American Society of Pediatric Hematologist Oncologists were rigorously analyzed by a multi-disciplinary research team, including Dr. Ionela Iacobas, M.D., Medical Director of the Vascular Anomalies Center at Texas Children's Hospital, and David Lapidus, Founder and President of LapidusData and a rare disease epidemiology expert.
An estimated 44,553 high probability LM patients with cutaneous involvement are projected based on the methodology developed by the authors that includes having greater than or equal to two diagnosis claims related to LM.
An additional cohort of an estimated 48,414 potential LM patients with cutaneous involvement are projected based on a methodology developed by the authors that includes having at least 1 diagnosis claim related to LM.
In total, the two cohorts, which include high probability LM patients with cutaneous involvement and potential LM patients with cutaneous involvement, result in up to an estimated 92,967 LM patients with cutaneous involvement.
Estimates of annual incidence of LMs with cutaneous involvement will also be highlighted in the poster presentation.
“This study is the first to quantify the U.S. LM population based on a rigorous, quantitative analysis of medical claims data. The findings reinforce what we are seeing in clinical practice, which is growing demand for multidisciplinary care of patients with complex vascular anomalies, including microcystic lymphatic malformations," said Dr. Ionela Iacobas, M.D., Medical Director of the Vascular Anomalies Center at Texas Children's Hospital. She continued, "Many of the historical estimates in the literature date from a period prior to advances in care; today we are able to offer patients more in terms of clinical trials and potential treatments, which has increased awareness of the disease and also referrals."
The full text of the abstract can be found here and will be published in the August 2025 supplement issue of the Journal of Investigative Dermatology (JID). The details of the poster to be presented at SID are as follows:
Society for Investigative Dermatology Annual Meeting Title: Incidence, prevalence, and care for patients with lymphatic malformations (LMs) in the U.S.: A claims-based analysis Abstract ID: LB1123 Poster Session Category: Clinical Research: Epidemiology and Observational Research Date & Time: Thursday May 8, 2025, 4:30-6:00 pm PT Location: Indigo Ballroom/Foyer, Hilton San Diego Bayfront
About Microcystic Lymphatic Malformations Microcystic LMs are a rare, chronically debilitating genetic disease caused by dysregulation of the phosphatidylinositol 3-kinase (PI3K)/mammalian target of rapamycin (mTOR) pathway. The disease is characterized by malformed lymphatic vessels that protrude through the skin and persistently leak lymph fluid (lymphorrhea) and bleed, often leading to recurrent serious infections and cellulitis that can cause hospitalization. The natural history of microcystic LMs is persistent and progressive without spontaneous resolution, with symptoms generally worsening during life, including increases in the number and size of malformed vessels that lead to complications and lifetime morbidity. There are currently no FDA-approved treatments for the estimated more than 30,000 diagnosed patients with microcystic LMs in the United States.
About Palvella Therapeutics Founded and led by rare disease drug development veterans, Palvella Therapeutics, Inc. (Nasdaq: PVLA) is 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 FDA-approved therapies. Palvella is developing a broad pipeline of product candidates based on its patented QTORIN™ platform, with an initial focus on serious, rare genetic skin diseases, many of which are lifelong in nature. Palvella’s lead product candidate, QTORIN 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), is currently being evaluated in the Phase 3 SELVA clinical trial in microcystic lymphatic malformations and the Phase 2 TOIVA clinical trial in cutaneous venous malformations. For more information, please visit www.palvellatx.com or follow Palvella on LinkedIn or X (formerly known as Twitter).
QTORIN™ rapamycin is for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency for any indication.
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