Palvella Therapeutics完成QTORIN rapamycin治疗微囊型淋巴管畸形的NDA滚动提交

QTORIN™ rapamycin has the potential to become the first FDA-approved therapy and establish a new standard of care for an estimated more than 30,000 pediatric and adult patients living with microcystic lymphatic malformations in the U.S.
FDA previously granted Breakthrough Therapy and Fast Track designations; QTORIN™ rapamycin has also received Orphan Drug designation
Accelerating U.S. launch readiness for a potential commercial launch of QTORIN™ rapamycin in the first half of 2027, if approved
WAYNE, Pa., Aug. 31, 2026 (GLOBE NEWSWIRE) -- 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 completed the rolling submission of its 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), a serious, rare, and chronically debilitating genetic disease for which there are currently no FDA-approved therapies.
“Completing the submission of our NDA brings us one step closer to our goal of delivering the first FDA-approved therapy for patients living with microcystic LMs,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella Therapeutics. “We are deeply grateful to the trial participants, investigators, and study teams who made this milestone possible. We also appreciate the FDA’s collaboration throughout the application process, including its recent decision to grant rolling review of our NDA. Patients remain at the heart of everything we do at Palvella as we continue to advance QTORIN™ rapamycin with urgency for the rare disease communities we serve.”
The NDA includes results from the Phase 3 SELVA trial, which met its primary endpoint, pre-specified key secondary endpoint, and all four secondary efficacy endpoints, with all six efficacy endpoints achieving statistical significance (all p<0.001). In SELVA, among participants aged 6 years and older who completed the efficacy evaluation period, 86% were rated as “Much Improved” or “Very Much Improved” at Week 24 on the Microcystic Lymphatic Malformation Investigator Global Assessment (mLM-IGA), the study’s primary endpoint. QTORIN™ rapamycin was also well tolerated in SELVA, with no drug-related serious adverse events reported and systemic rapamycin levels below 2 ng/mL at all timepoints for all participants. The NDA also includes Phase 2 clinical results that supported FDA’s decision to grant QTORIN™ rapamycin Breakthrough Therapy designation, as well as published literature and other evidence regarding the clinical use of off-label rapamycin in microcystic LMs. The NDA was submitted under the 505(b)(2) regulatory pathway, which allows FDA to rely in part on prior findings of safety and effectiveness and other existing data.
Within 60 days, FDA will determine whether the application is complete and acceptable for filing and whether Priority Review will be granted. If granted, Priority Review would provide for a six-month review goal.
Palvella continues to advance U.S. launch readiness and execute key pre-launch activities, supported by a senior commercial leadership team with extensive rare disease and dermatology launch experience, a field-based medical science liaison team deployed across the U.S., and a patient services organization established in preparation for a potential launch in the first half of 2027, if approved.
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
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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.
Forward-Looking Statements
This press release contains forward-looking statements (including within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended (Securities Act)). These statements may discuss goals, intentions, and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the management of Palvella, as well as assumptions made by, and information currently available to, the management of Palvella. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Statements that are not historical facts are forward-looking statements. Forward-looking statements include, but are not limited to, statements regarding the expected timing of the presentation of data from clinical trials, Palvella’s clinical development plans and related anticipated development milestones and anticipated timing of regulatory submissions, Palvella’s expectations with respect to the anticipated FDA review process for the NDA for QTORIN™ rapamycin and potential timing of a commercial launch of QTORIN™ rapamycin, if approved, Palvella’s plans to meet with regulatory authorities, Palvella’s expectations regarding the benefits of breakthrough therapy designation, orphan drug designation and potential benefit of orphan drug exclusivity for QTORIN™ rapamycin for the treatment of microcystic lymphatic malformations, Palvella’s cash, financial resources and expected runway, Palvella’s expectations regarding its programs, including QTORIN™ rapamycin and QTORIN™ pitavastatin, and its research-stage opportunities, including its expected therapeutic potential and market opportunity. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the ability to raise additional capital to finance operations; the ability to advance product candidates through preclinical and clinical development; the ability to make regulatory submissions on anticipated timelines; the ability to obtain regulatory approval for, and ultimately commercialize, Palvella’s product candidates, including QTORIN™ rapamycin and QTORIN™ pitavastatin; the outcome of early clinical trials for Palvella’s product candidates, including the ability of those trials to satisfy relevant governmental or regulatory requirements; the fact that data and results from clinical studies may not necessarily be indicative of future results; Palvella’s limited experience in designing clinical trials and lack of experience in conducting clinical trials; Palvella’s limited experience in commercial manufacturing; the ability to identify and pivot to other programs, product candidates, or indications that may be more profitable or successful than Palvella’s current product candidates; the substantial competition Palvella faces in discovering, developing, or commercializing products; the negative impacts of global events on operations, including ongoing and planned clinical trials and ongoing and planned preclinical studies; the ability to attract, hire, and retain skilled executive officers and employees; the ability of Palvella to protect its intellectual property and proprietary technologies; reliance on third parties, contract manufacturers, and contract research organizations; and the risks and uncertainties described in the filings made by Palvella with the Securities and Exchange Commission (SEC), including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the SEC and available at
www.sec.gov
. The events and circumstances reflected in our forward-looking statements may not be achieved or occur, and actual results could differ materially from those projected in the forward-looking statements. New risk factors and uncertainties may emerge from time to time, and it is not possible for management to predict all risk factors and uncertainties that Palvella may face. Except as required by applicable law, Palvella does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. This press release contains hyperlinks to information that is not deemed to be incorporated by reference into this press release.
Contact Information
Investors
Wesley H. Kaupinen
Founder and CEO
Palvella Therapeutics
wes.kaupinen@palvellatx.com
Media
Marcy Nanus
Vice President of Investor Relations and Corporate Affairs
Palvella Therapeutics
marcy.nanus@palvellatx.com