Palvella Therapeutics Secures New U.S. Patent for QTORIN™ Rapamycin in Treating Microcystic Lymphatic Malformations

Palvella Therapeutics, Inc., a clinical-stage biopharmaceutical company headquartered in Wayne, Pennsylvania, has announced a significant milestone in its efforts to develop treatments for rare genetic skin disorders. On April 22, 2025, the company disclosed that it has been granted its fifth U.S. patent for its lead candidate, QTORIN™ 3.9% rapamycin anhydrous gel, by the United States Patent and Trademark Office. This patent, numbered 12,268,673, forms a crucial part of Palvella's growing intellectual property portfolio aimed at addressing microcystic lymphatic malformations (microcystic LMs) and other rare dermatological conditions.

The newly granted patent, titled “Anhydrous Compositions of mTOR Inhibitors and Methods of Use,” significantly broadens the protective scope for QTORIN™ rapamycin, particularly concerning its application for microcystic LMs. This condition is a rare and debilitating genetic disorder caused by abnormalities in the phosphatidylinositol 3-kinase/mTOR pathway. The condition is characterized by malformed lymphatic vessels that protrude through the skin and cause persistent leakage and bleeding, often resulting in severe infections and complications like cellulitis. With more than 30,000 individuals diagnosed in the United States, there are currently no approved therapies for this chronic condition.

QTORIN™ rapamycin, still under investigation, has received several designations from the U.S. Food and Drug Administration (FDA), including Breakthrough Therapy, Orphan Drug, and Fast Track. These designations highlight the potential of QTORIN™ rapamycin to become the first approved treatment for microcystic LMs in the U.S. If approved, it is anticipated to receive seven years of market exclusivity due to its orphan drug status.

Wes Kaupinen, Palvella's Founder and CEO, hailed the patent issuance as a testament to the company's innovative approach in developing the QTORIN™ platform. According to Kaupinen, the patent marks a significant step in Palvella's mission to introduce the first targeted therapy for microcystic LMs, underscoring the company's commitment to addressing serious, rare, and lifelong genetic skin diseases for which there are no current FDA-approved therapies.

Palvella Therapeutics has positioned itself as a leader in the rare disease space, especially with its focus on severe genetic skin disorders. The company is actively conducting clinical trials to further evaluate the efficacy and safety of QTORIN™ rapamycin. These include the Phase 3 SELVA trial for microcystic LMs and the Phase 2 TOIVA trial targeting cutaneous venous malformations.

The product remains investigational and has yet to receive approval from the FDA or any other regulatory body. Nonetheless, Palvella's ongoing research and development activities reflect its dedication to bringing new treatment options to patients with unmet medical needs. The company's innovative QTORIN™ platform promises to expand its pipeline, potentially transforming the landscape for those suffering from rare skin diseases without current treatment options.

Palvella’s commitment to expanding its intellectual property portfolio not only strengthens its market position but also exemplifies the company's proactive approach in seeking novel therapeutic solutions for rare and serious dermatological conditions. The company's strategic focus on intellectual property protection, clinical trials, and breakthrough drug designations lays a strong foundation for future advancements and potential commercial success in the biopharmaceutical industry.