WAYNE, Pa. — (Nasdaq: PVLA) Palvella Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare skin diseases for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today announced it has received the second year of funding under its FDA Office of Orphan Products Development grant. The grant provides up to $2.6 million in non-dilutive funding over its full 4-year term and supports Palvella’s ongoing Phase 3 SELVA trial of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of microcystic lymphatic malformations (microcystic LMs).
“We are deeply appreciative of the FDA’s continued support of our Phase 3 program, which reflects the urgency and importance of addressing the significant unmet need faced by the estimated more than 30,000 individuals in the U.S. living with microcystic lymphatic malformations,” said Jeff Martini, Ph.D., Chief Scientific Officer of Palvella. “Exceeding our enrollment goal and maintaining momentum toward top-line data in early 2026 demonstrates the strength of our clinical program and our unwavering commitment to bringing QTORIN™ rapamycin to patients who currently have no FDA-approved therapies.”
The SELVA trial is a 24-week, Phase 3, single-arm, baseline-controlled clinical trial of QTORIN™ rapamycin administered once daily for the treatment of microcystic LMs. Designed to enroll 40 subjects, the trial surpassed its enrollment target, enrolling 51 subjects at leading U.S. vascular anomaly centers. Following an 8-week baseline period and 24-week evaluation, eligible participants may continue treatment in an open-label extension study. The SELVA trial remains on track to report top-line results in the first quarter of 2026. Assuming positive Phase 3 results, Palvella plans to submit an NDA to the FDA in the second half of 2026.
As a part of the Orphan Products Grant Program, Palvella is required to submit an annual performance progress report to the FDA. Following the submission and FDA review of this year’s performance progress report, the FDA granted Palvella the second year of funding. Out of 51 applications received by the FDA Orphan Products Grants Program in fiscal year 2024, Palvella’s Phase 3 SELVA trial was one of only seven new clinical trials selected for funding—and the only Phase 3 trial awarded a grant. The FDA Orphan Products Grants Program provides annual grants to support the development of safe and effective medical products to address unmet medical needs for patients with rare diseases or conditions. Grant applications are individually reviewed and scored for scientific and technical merit by an independent ad hoc panel of rare disease and regulatory experts and may involve consultation with the relevant FDA review division to help determine whether the proposed study will provide acceptable data that could contribute to product approval. Since inception, the program has supported clinical research leading to the approval of more than 85 products.
QTORIN™ rapamycin has been granted Breakthrough Therapy, Orphan Drug, and Fast Track designations by the FDA for the treatment of microcystic LMs. If approved, QTORIN™ rapamycin is expected to qualify for seven years of orphan drug market exclusivity in the U.S.
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 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 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.
Contact Information
Investors
Wesley H. Kaupinen
Founder and CEO, Palvella Therapeutics
[email protected]
Media
Marcy Nanus
Managing Partner, Trilon Advisors LLC
[email protected]