Announcement • Jun 22

Palvella Therapeutics Grants Rolling Review of Qtorin Rapamycin Nda for Microcystic Lymphatic Malformations

Palvella Therapeutics, Inc. announced that FDA has granted rolling review for the Company’s New Drug Application (NDA) for QTORIN rapamycin for the treatment of microcystic lymphatic malformations (microcystic LMs). Palvella remains on track to complete the NDA submission in the second half of 2026. QTORIN rapamycin has the potential to become the first FDA-approved therapy and standard of care for the estimated more than 30,000 individuals with microcystic lymphatic malformations in the U.S. 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 data demonstrate that microcystic LMs are persistent and progressive and do not spontaneously regress. Surgery, sclerotherapy and laser can be limited by recurrence and repeated procedures, while systemic PI3K/mTOR inhibitors may be constrained by chronic safety and tolerability considerations, particularly in children, and by challenges in achieving sufficient exposure in affected skin. Advances in molecular genetics have established dysregulated PI3K/mTOR signaling as a central disease driver, supporting precision, mechanism-based treatment. QTORIN rapamycin is designed to deliver rapamycin directly to pathogenic skin tissue to achieve local, on-target inhibition of disease-driving mTOR signaling while minimizing systemic exposure. There are no FDA-approved treatments for the estimated 30,000 or more people diagnosed with microcystic LMs in the United States. Rolling review is an FDA regulatory feature available to programs with Fast Track or Breakthrough Therapy designation and is intended to facilitate expedited FDA review of applications for therapies addressing serious conditions with unmet medical need. Rolling review allows Palvella to submit completed sections of the QTORIN rapamycin NDA as they become available, enabling FDA review to proceed while the Company completes the remainder of the application. QTORIN rapamycin has received Breakthrough Therapy, Orphan Drug and Fast Track designations from FDA for the treatment of microcystic LMs. 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. 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.

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