Palvella Therapeutics Gets FDA Grant for Phase 3 QTORIN™ Trial

Palvella Therapeutics, Inc., a biopharmaceutical enterprise focused on developing treatments for rare genetic skin disorders, has successfully secured funding from the FDA Office of Orphan Products Development. This grant is intended to support a pivotal Phase 3 trial, named SELVA, which is centered around the company's promising therapeutic candidate, QTORIN™ 3.9% rapamycin anhydrous gel. This gel is specifically being investigated for its potential efficacy in treating microcystic lymphatic malformations (microcystic LMs), a serious condition with no approved therapies currently available in the United States.

Palvella Therapeutics stands out in the competitive field of rare disease research, having been chosen from among 51 applicants in the fiscal year 2024 by the FDA Orphan Products Grants Program. Only seven new clinical trials were selected for funding, with Palvella's SELVA trial being the only Phase 3 study to receive this support. The trial is designed as a 24-week, single-arm, baseline-controlled study, involving daily administration of the investigational gel to approximately 40 subjects aged three years and above. These subjects will be recruited from prominent vascular anomaly centers across the United States. The company anticipates revealing top-line data from this trial in the first quarter of 2026.

Wes Kaupinen, the Founder and CEO of Palvella Therapeutics, expressed his gratitude for the FDA grant, highlighting the significance of this funding in advancing the development of QTORIN™ rapamycin. He emphasized the transformative potential of this therapy to establish a new standard of care for individuals affected by microcystic LMs, a debilitating genetic disease marked by abnormal lymphatic vessels that lead to persistent leakage of lymph fluid, bleeding, and recurrent infections. These symptoms can escalate, causing severe complications and lifetime morbidity, with no spontaneous resolution observed in its natural progression.

Microcystic lymphatic malformations are associated with the dysregulation of the phosphatidylinositol 3-kinase (PI3K) and mammalian target of rapamycin (mTOR) pathways. The absence of FDA-approved treatments for the estimated over 30,000 diagnosed patients in the United States underscores the urgent need for innovative therapeutic solutions like QTORIN™ rapamycin.

Palvella Therapeutics is committed to addressing unmet medical needs in rare genetic skin diseases through its proprietary QTORIN™ platform. The company's product pipeline is geared towards developing novel therapies that offer hope to patients with lifelong, serious conditions. The lead candidate, QTORIN™ rapamycin, is under evaluation in the Phase 3 SELVA trial for microcystic LMs and a Phase 2 trial for cutaneous venous malformations.

With this non-dilutive funding from the FDA, Palvella aims to advance its clinical research and move closer to the potential commercialization of QTORIN™ rapamycin. This step is crucial not only for the company but also for the broader rare disease community, offering prospects for improved care and quality of life for patients afflicted by these challenging conditions. As the company continues its research endeavors, the anticipation surrounding the SELVA trial results remains high, marking a significant milestone in the ongoing effort to develop effective treatments for microcystic lymphatic malformations.