Palvella Therapeutics Granted Additional U.S. Patent for QTORIN™ Rapamycin for the Treatment of Microcystic Lymphatic Malformations

Rhea-AI Summary

Palvella Therapeutics (PVLA) has been granted its fifth U.S. patent (No. 12,268,673) for QTORIN™ rapamycin, extending patent protection until 2038. The patent broadens protection for QTORIN™ 3.9% rapamycin anhydrous gel and its clinical applications in treating microcystic lymphatic malformations (LMs).

QTORIN™ rapamycin has received multiple FDA designations including Breakthrough Therapy, Orphan Drug, and Fast Track for treating microcystic LMs. If approved, it could become the first FDA-approved therapy for this rare genetic skin disease and receive seven years of orphan drug market exclusivity in the U.S.

Positive

• Fifth U.S. patent granted, extending protection until 2038
• Triple FDA designations secured (Breakthrough, Orphan Drug, Fast Track)
• Potential for 7 years of orphan drug market exclusivity
• Positioned to be first-to-market for microcystic LMs treatment

Negative

• Product still in clinical stage, not yet FDA approved
• No current revenue generation from QTORIN™ rapamycin

Insights

Pharmaceutical Patent Expert

New patent extends QTORIN™ rapamycin protection until 2038, bolstering Palvella's exclusivity position in addressing an untreated rare disease.

The USPTO issuance of patent No. 12,268,673 represents Palvella's fifth U.S. patent for QTORIN™ rapamycin, significantly strengthening their intellectual property position. This patent specifically covers "Anhydrous Compositions of mTOR Inhibitors and Methods of Use," broadening protection for both the formulation and its clinical applications for microcystic lymphatic malformations (LMs).

With patent protection extending into 2038, Palvella secures approximately 13 more years of exclusivity for this technology. In pharmaceutical development, this timeline provides crucial runway for the typically lengthy development, approval, and commercialization processes.

The patent strategy is further reinforced by regulatory designations, with QTORIN™ rapamycin positioned to receive an additional seven years of orphan drug exclusivity upon approval. This multi-layered exclusivity approach creates a substantial competitive moat around their lead asset, particularly valuable in rare disease markets where first-mover advantage often translates to sustainable market position.

The continued expansion of Palvella's patent portfolio specifically targeting rare skin diseases demonstrates a strategic focus on building comprehensive protection around their therapeutic platform. For microcystic LMs specifically, this patent reinforces the company's position in developing what could become the first-ever FDA-approved therapy for this rare genetic skin condition.

Biotechnology Analyst

Triple FDA designations plus strong IP position QTORIN™ rapamycin favorably as first potential treatment for microcystic lymphatic malformations.

Palvella's QTORIN™ rapamycin has secured an impressive regulatory trifecta from the FDA: Breakthrough Therapy, Orphan Drug, and Fast Track designations for treating microcystic lymphatic malformations. This regulatory achievement is particularly significant as these designations provide tangible benefits including intensified FDA guidance, accelerated review timelines, and reduced development hurdles.

The Breakthrough Therapy designation is especially noteworthy as the FDA reserves this for treatments demonstrating substantial improvement over available therapies on clinically significant endpoints. This designation suggests the FDA recognizes the potential impact of QTORIN™ rapamycin for patients with this rare condition.

Microcystic lymphatic malformations represent a classic orphan disease opportunity - a serious genetic condition with no FDA-approved therapies currently available. This therapeutic void creates both a significant unmet medical need and a commercial opportunity where Palvella could establish their product as the standard of care.

The newly granted patent specifically covering anhydrous formulations suggests Palvella has developed specialized delivery technology to optimize rapamycin's effectiveness in dermatological applications. This formulation innovation, protected now until 2038, may provide advantages in treating this challenging skin condition while creating barriers to potential competitors.

The combination of these regulatory advantages with extended patent protection positions Palvella favorably in this orphan disease space, where exclusivity often enables sustainable market position and premium pricing power.

Fifth issued patent in the U.S. for QTORIN™ rapamycin with anticipated patent life extending into 2038

QTORIN™ rapamycin has the potential to be the first approved therapy and standard of care for microcystic lymphatic malformations in the U.S.

WAYNE, Pa., April 22, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) Palvella Therapeutics, Inc. (Palvella or “the Company”), 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. 12,268,673 for claims related to the Company's lead product candidate QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin). The newly granted U.S. patent is part of Palvella’s expanding intellectual property portfolio directed to novel therapies targeting microcystic lymphatic malformations (microcystic LMs) and other rare skin diseases.

The granted patent “Anhydrous Compositions of mTOR Inhibitors and Methods of Use” broadens protection for QTORIN™ rapamycin and the conditions related to its use in clinical indications, including microcystic LMs.

"This fifth U.S. patent reflects the significant innovation behind the development of QTORIN™ rapamycin and Palvella’s QTORIN™ platform,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. “We are pleased to further strengthen our growing intellectual property portfolio as part of our pursuit to develop the first targeted therapy for microcystic lymphatic malformations, a serious, rare, and chronically debilitating genetic disease for which there are no FDA-approved therapies."

QTORIN™ rapamycin has been granted Breakthrough Therapy Designation, Orphan Drug Designation, and Fast Track Designation from the U.S. FDA for the treatment of microcystic LMs. In addition to granted patents, it is anticipated that QTORIN™ rapamycin will be eligible for seven years of orphan drug market exclusivity in the U.S., if approved.

About Microcystic Lymphatic Malformations

Microcystic LMs are a rare, chronically debilitating genetic disease caused by dysregulation of the phosphatidylinositol 3-kinase (PI3K)/mammalian target of rapamycin (mTOR) pathway. The disease is characterized by malformed lymphatic vessels that protrude through the skin and persistently leak lymph fluid (lymphorrhea) and bleed, often leading to recurrent serious infections and cellulitis that can cause hospitalization. The natural history of microcystic LMs is persistent and progressive without spontaneous resolution, with symptoms generally worsening during life, including increases in the number and size of malformed vessels that lead to complications and lifetime morbidity. There are currently no FDA-approved treatments for the estimated more than 30,000 diagnosed patients with microcystic LMs in the United States.

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 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.

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

When will Palvella's QTORIN rapamycin patent expire?

The patent for QTORIN rapamycin extends into 2038, providing long-term intellectual property protection for Palvella Therapeutics.

What FDA designations has PVLA's QTORIN rapamycin received for microcystic LMs?

QTORIN rapamycin has received three FDA designations: Breakthrough Therapy Designation, Orphan Drug Designation, and Fast Track Designation.

How many U.S. patents does Palvella Therapeutics have for QTORIN rapamycin?

Palvella Therapeutics has been granted five U.S. patents for QTORIN rapamycin, with the latest being patent No. 12,268,673.

What market exclusivity period could PVLA's QTORIN receive if FDA approved?

If approved, QTORIN rapamycin would be eligible for seven years of orphan drug market exclusivity in the United States.