Palvella Therapeutics Granted Sixth U.S. Patent Covering 0.1–20% Anhydrous Compositions of Rapamycin and Other mTOR Inhibitors
- Secured sixth U.S. patent extending protection until at least 2038
- Multiple FDA designations received: Breakthrough Therapy, Orphan Drug, and Fast Track
- Potential for 7 years of orphan drug market exclusivity upon approval
- Awarded FDA Orphan Products Grant worth up to $2.6 million for Phase 3 trial
- None.
Insights
Palvella's new patent significantly strengthens market exclusivity for its lead candidate through 2038, enhancing commercial potential for rare skin disease treatments.
Palvella's newly granted U.S. patent (No. 12,329,748) represents a substantial strengthening of their intellectual property portfolio for QTORIN™ 3.9% rapamycin anhydrous gel. This sixth U.S. patent is particularly valuable as it provides broad composition and method-of-use protection extending through at least 2038 - a critical timeline for pharmaceutical asset valuation.
The patent's scope is impressively comprehensive, covering anhydrous compositions with rapamycin concentrations ranging from 0.1–20% as well as other mTOR inhibitors including temsirolimus and everolimus. This breadth creates a formidable barrier to potential competitors and provides Palvella with significant freedom to optimize their formulations.
Palvella has constructed a multi-layered exclusivity strategy that combines:
- Strong patent protection (now six U.S. patents)
- Proprietary formulation and manufacturing trade secrets
- Regulatory exclusivities including potential 7-year orphan drug exclusivity
The regulatory designations already secured (Breakthrough Therapy, Orphan Drug, Fast Track) indicate FDA recognition of QTORIN™ rapamycin's potential significance for addressing unmet medical needs in rare dermatologic conditions. These designations, combined with the $2.6 million FDA Orphan Products Grant, suggest both clinical promise and regulatory support that complement the strengthened IP position.
New intellectual property builds on Palvella’s multi-layered exclusivity strategy
Patent term expected through at least 2038
WAYNE, Pa., June 18, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) Palvella Therapeutics, Inc. (Palvella), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today announced that the United States Patent and Trademark Office (USPTO) issued patent No. 12,329,748 for claims related to the Company's lead product candidate QTORIN™
“Our sixth issued U.S. patent marks another meaningful milestone in solidifying the exclusivity position of QTORIN™ rapamycin,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. “We’re executing a multi-layered strategy to protect and maximize the long-term value of QTORIN™ rapamycin—anchored by strong intellectual property, proprietary formulation and manufacturing trade secrets, and regulatory exclusivities.”
The newly granted U.S. patent encompasses a wide range of composition and method-of-use claims for QTORIN™ rapamycin and other mTOR inhibitors—including temsirolimus and everolimus—formulated in anhydrous compositions. It also covers treatment of a broad spectrum of dermatologic conditions, with emphasis on rare and difficult-to-treat diseases such as microcystic lymphatic malformations and venous malformations.
QTORIN™ rapamycin has been granted Breakthrough Therapy, Orphan Drug, and Fast Track Designations by the FDA for the treatment of microcystic lymphatic malformations. If approved, QTORIN™ rapamycin is expected to qualify for seven years of orphan drug market exclusivity in the U.S. Palvella has also been named an awardee of an FDA Orphan Products Grant from the FDA’s Office of Orphan Products Development and is eligible to receive up to
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 genetic 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 genetic skin diseases, many of which are lifelong in nature. Palvella’s lead product candidate, QTORIN™
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.
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 ongoing clinical trials, Palvella’s clinical development plans and related anticipated development milestones, Palvella’s cash and financial resources and expected cash runway, and the potential of, and expectations regarding, Palvella’s programs, including QTORIN™ rapamycin, 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 obtain regulatory approval for, and ultimately commercialize, Palvella’s product candidates, including QTORIN™ rapamycin; 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; 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
Managing Partner, Trilon Advisors LLC
mnanus@trilonadvisors.com
FAQ
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