FDA Grants Palvella Therapeutics Up to $2.6 Million for Microcystic Lymphatic Malformations Trial

Palvella Therapeutics, Inc., a clinical-stage biopharmaceutical firm focusing on groundbreaking treatments for serious, rare genetic skin conditions, has announced a significant milestone. The company reported receiving a grant of up to $2.6 million from the U.S. Food and Drug Administration’s (FDA) Office of Orphan Products Development. This funding is intended to support the Phase 3 SELVA trial of their lead product, QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), designed to treat microcystic lymphatic malformations (microcystic LMs).

Wes Kaupinen, Founder and CEO of Palvella, expressed enthusiasm about the grant, highlighting its importance in advancing their ongoing Phase 3 trial. He emphasized that the support from the FDA underscores the significant unmet medical need for effective treatments for microcystic LMs, a rare and debilitating genetic disease. The grant also acknowledges the scientific rationale behind targeting the PI3K/mTOR pathway, which is implicated in the disease, and the potential of QTORIN rapamycin to become the first FDA-approved therapy for this condition in the U.S.

Palvella's Phase 3 clinical trial was one of only seven new clinical trials to receive a grant from the FDA Orphan Products Grants Program in fiscal year 2024. This program annually awards grants to support the development of promising medical products aimed at treating rare diseases, for which there are often no effective treatments. Each grant application undergoes a rigorous review process by an independent panel of rare disease and regulatory experts, who assess the scientific and technical merit of the proposed study.

Microcystic LMs are characterized by malformed lymphatic vessels that leak lymph fluid and bleed, leading to recurrent serious infections and cellulitis, often requiring hospitalization. The condition is persistent and progressive, worsening over time without spontaneous resolution. Currently, there are no FDA-approved treatments available for the estimated 30,000 patients diagnosed with microcystic LMs in the United States.

Palvella's QTORIN platform represents a promising approach to treating these rare genetic skin diseases. The platform is designed to create topical therapies that penetrate deep skin layers, targeting the pathogenic tissue while minimizing systemic exposure and associated adverse events. QTORIN rapamycin, the lead product from this platform, is a novel 3.9% rapamycin anhydrous gel. It aims to harness the therapeutic benefits of rapamycin, an mTOR inhibitor, specifically for conditions like microcystic LMs and cutaneous venous malformations. The product is protected by multiple patents in the U.S. and Japan, with pending patent applications in Europe and Japan.

In the third quarter of 2024, Palvella initiated the SELVA trial, a 24-week, Phase 3 clinical study of QTORIN rapamycin for treating microcystic LMs. The trial is single-arm and baseline-controlled, focusing on clinician-reported outcomes as its primary and key secondary endpoints. The study aims to enroll 40 subjects across leading vascular anomaly centers in the U.S.

Despite the promising potential of QTORIN rapamycin, it remains investigational and has not yet received approval from the FDA or any other regulatory body.

Palvella Therapeutics, founded by veterans in rare disease drug development, continues to focus on novel therapies for severe, rare genetic skin diseases. The company has a robust pipeline based on its QTORIN platform, with a primary focus on lifelong conditions lacking current FDA-approved treatments. In July 2023, Palvella announced a definitive merger agreement with Pieris Pharmaceuticals, Inc., aiming to combine the companies in an all-stock transaction, marking another significant step in their journey to address unmet medical needs in rare genetic disorders.