Palvella Therapeutics Receives FDA Grant for Phase 3 Trial of QTORIN Rapamycin in Microcystic Lymphatic Malformations
Key Insights
Palvella Therapeutics (search) has been awarded a $2.6 million grant from the FDA to support the Phase 3 SELVA trial of QTORIN 3.9% rapamycin anhydrous gel.
The SELVA trial is evaluating QTORIN rapamycin (search) for the treatment of microcystic lymphatic malformations (search) (LMs), a rare and debilitating genetic disease.
QTORIN rapamycin (search) has received FDA Breakthrough Therapy, Fast Track, and Orphan Drug Designations for microcystic LMs, highlighting its potential impact.
Palvella Therapeutics (search), Inc., a clinical-stage biopharmaceutical company, has received a grant of up to $2.6 million from the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development. This funding will support the ongoing Phase 3 SELVA trial evaluating QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of microcystic lymphatic malformations (search) (LMs).
FDA Support for Rare Disease Treatment
The FDA's Orphan Products Grants Program is designed to foster the development of medicines addressing unmet needs in rare diseases. Palvella's Phase 3 trial was one of only seven new studies selected for funding from a pool of 51 applications in fiscal year 2024. The program emphasizes rigorous review for scientific and technical merit, often involving consultation with FDA review divisions to ensure the studies yield data that could contribute to product approval. To date, the Orphan Products Grants Program has facilitated the approval of over 85 products.
QTORIN Rapamycin for Microcystic LMs
Palvella's lead product candidate, QTORIN rapamycin (search), is a patented 3.9% rapamycin anhydrous gel. It is designed to deliver the therapeutic benefits of rapamycin, an mTOR (search) inhibitor, while minimizing systemic exposure and potential adverse reactions. The ongoing SELVA trial is a 24-week, single-arm, baseline-controlled clinical trial with primary and key secondary endpoints based on clinician-reported outcomes. The trial, which began in the third quarter of 2024, aims to enroll 40 subjects at vascular anomaly centers across the U.S.
Unmet Need in Microcystic Lymphatic Malformations
Microcystic LMs is a rare, chronically debilitating genetic disease caused by dysregulation of the PI3K/mTOR pathway (search). It is characterized by malformed lymphatic vessels that protrude through the skin, leading to lymphorrhea, bleeding, recurrent infections, and cellulitis. These complications can result in hospitalization and significant lifetime morbidity. There are currently no FDA-approved treatments for the estimated 30,000 diagnosed patients with microcystic LMs in the United States.
Company Perspective
Wes Kaupinen, Founder and CEO of Palvella, stated, "We are pleased to receive this grant from the FDA which will support the advancement of SELVA... We believe this grant underscores the high unmet medical need in this serious, rare and chronically debilitating genetic disease, the scientific rationale for targeted inhibition of the causative PI3K/mTOR pathway (search), and QTORIN rapamycin (search)’s potential to be the first approved therapy and standard of care in the U.S. for microcystic LMs."
QTORIN Platform
Palvella's QTORIN platform is designed to generate novel topical therapies that penetrate deep layers of the skin to treat serious, rare genetic skin diseases locally. QTORIN rapamycin (search) has received FDA Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation for microcystic LMs, as well as Fast Track Designation for venous malformations. The drug is protected by multiple patents in the U.S. and Japan.