WAYNE, Pa. — Palvella Therapeutics, Inc. (Palvella or the “Company”) (Nasdaq: PVLA), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies for serious, rare skin diseases and vascular malformations for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today announced that the Company has submitted the first module of its rolling New Drug Application (NDA) to the FDA seeking approval of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of microcystic lymphatic malformations (microcystic LMs). Palvella remains on track to submit the remaining modules and complete the NDA submission in the second half of 2026.
“Initiating the rolling NDA submission represents an important milestone in advancing QTORIN™ rapamycin toward potential approval for patients living with microcystic LMs, a serious, chronically debilitating genetic disease for which there are no FDA-approved therapies,” said Jeff Martini, Ph.D., Chief Scientific Officer of Palvella Therapeutics. “Recent advances in molecular genetics have established aberrant activation of the PI3K/mTOR pathway as the central molecular driver of microcystic LMs, creating the opportunity to develop targeted therapies that address the underlying biology of the disease. QTORIN™ rapamycin was designed to deliver rapamycin directly to pathogenic skin tissue to achieve local, on-target inhibition of mTOR signaling while minimizing systemic exposure. The Phase 3 SELVA study demonstrated highly statistically significant improvements across the primary endpoint, key secondary endpoint, and all prespecified secondary endpoints, and QTORIN™ rapamycin was well tolerated, supporting the potential of this on-target, in-tissue approach.”
As Palvella advances toward completion of the NDA submission, the Company is accelerating U.S. launch readiness for a potential standalone commercial launch of QTORIN™ rapamycin in the first half of 2027, if approved. Palvella has recruited core leadership across its commercial, medical affairs, and patient services organizations, including leaders with track records of successfully launching first-in-disease therapies for serious and rare skin diseases. The Company continues to build these organizations through the addition of field commercial leaders, medical science liaisons, and patient access liaisons. In March 2026, Palvella launched the BEYOND mLM campaign and BeyondMLM.com in collaboration with CaNVAS, LGDA, LE&RN, PeDRA, and VAccess.org to increase disease awareness, support earlier recognition and diagnosis, and advance disease education among physicians caring for patients with microcystic LMs, including specialists at vascular anomaly centers.
“For patients and families living with microcystic LMs, submission of the first NDA module brings us closer to our goal of delivering the first FDA-approved therapy for this serious and lifelong disease,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella Therapeutics. “We are completing the remaining NDA modules and U.S. launch-readiness activities with urgency, discipline, and a deep sense of responsibility to the patients, families, and physicians we seek to serve.”
QTORIN™ rapamycin has received Breakthrough Therapy, Orphan Drug, and Fast Track designations from the FDA for the treatment of microcystic LMs, as well as an FDA Orphan Products Development grant.
About Microcystic Lymphatic Malformations
Microcystic LMs are a rare, chronically debilitating genetic disease driven by dysregulation of the PI3K/mTOR pathway. Malformed lymphatic vessels can protrude through the skin, persistently leak and bleed, and cause recurrent infections, cellulitis and hospitalization. Published natural history studies demonstrate that microcystic LMs are persistent and progressive and do not spontaneously regress. Advances in molecular genetics have established dysregulated PI3K/mTOR signaling as a central disease driver, supporting precision, mechanism-based treatment. There are no FDA-approved treatments for the estimated 30,000 or more people diagnosed with microcystic LMs in the United States.
About Palvella Therapeutics
Founded and led by rare disease biotech veterans, Palvella Therapeutics, Inc. (Nasdaq: PVLA) is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients living with serious, rare skin diseases and vascular malformations 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 and vascular malformations, many of which are lifelong in nature. Palvella’s lead product candidate, QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), is currently being developed for the treatment of microcystic lymphatic malformations, cutaneous venous malformations, and clinically significant angiokeratomas. Palvella’s second product candidate, QTORIN™ pitavastatin, is currently being developed for the treatment of disseminated superficial actinic porokeratosis. For more information, please visit www.palvellatx.com or follow Palvella on LinkedIn or X (formerly known as Twitter).
QTORIN™ rapamycin and QTORIN™ pitavastatin are for investigational use only and neither has been approved 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
Vice President of Investor Relations and Corporate Affairs
Palvella Therapeutics
[email protected]