Hybrid public meeting on August 5th will explore approaches to selecting and prioritizing promising FDA-approved drugs for repurposing, with the potential to accelerate patient access to effective therapies for significant unmet medical needs
Dr. Martini to discuss evidence frameworks and regulatory approaches for advancing repurposed therapies for patients with serious, rare diseases
Meeting builds on FDA’s May 2026 drug repurposing initiative focused on advancing new uses for approved medicines, particularly in chronic or rare diseases with significant unmet medical needs
WAYNE, Pa., July 30, 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, announced today that Jeffrey Martini, Ph.D., Chief Scientific Officer of Palvella, has been selected to provide public comment at
Drug Repurposing: Considerations for Selection Criteria and Prioritization
,
an FDA public meeting to be held on August 5, 2026, in Washington, D.C.
“As pioneering research in rare diseases uncovers genetic drivers and causal biology, it can reveal opportunities to repurpose medicines as targeted therapies for previously untreatable diseases, building on accumulated scientific knowledge and clinical experience,” said Jeffrey Martini, Ph.D., Chief Scientific Officer of Palvella Therapeutics. “The combination of an established safety profile and emerging evidence from real-world use suggesting potential clinical benefit can provide a strong foundation for accelerating the development of repurposed therapies, particularly in chronic or rare diseases. I am honored to contribute to this important discussion on how scientific and regulatory frameworks can continue to foster innovation and accelerate access to meaningful therapies for patients with significant unmet medical needs.”
The hybrid public meeting, convened by the Reagan-Udall Foundation in collaboration with FDA, builds on the Agency’s broader initiative to identify new therapeutic uses for approved medicines and expand treatment options for patients with significant unmet medical needs. Bringing together representatives from federal agencies, clinicians, researchers, and patient organizations, the meeting will examine approaches for identifying and prioritizing promising repurposing candidates, as well as the evidence standards and regulatory considerations relevant to advancing them efficiently. During the public comment session, Dr. Martini is expected to address one of the meeting's central discussion questions: “What evidence gaps or policy/regulatory barriers most commonly hinder the advancement of promising repurposing opportunities?”
“Jeff’s selection to provide public comment at this important meeting reflects Palvella’s leadership in advancing first-in-disease therapies for serious, rare diseases through a unique approach that leverages well-characterized existing molecules and real-world clinical evidence,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella Therapeutics. “Recent years have seen growing momentum behind the development of established molecules for new therapeutic indications, particularly in rare diseases, as advances in the understanding of disease biology create opportunities to apply these molecules as on-target therapies that can directly address the causal drivers of disease. Novel, indication-specific formulations of existing molecules can further enhance the overall benefit-risk profile for patients. We appreciate the opportunity to contribute our perspective as FDA continues to advance scientific and regulatory approaches that can help bring meaningful new therapies to patients.”
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.
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 plans with respect to the timing of, and anticipated FDA review process for, the NDA for QTORIN™ rapamycin, Palvella’s plans to pursue Breakthrough Therapy Designation, Palvella’s plans to meet with regulatory authorities, Palvella’s expectations regarding the benefits of 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 Chief Executive Officer
Palvella Therapeutics
wes.kaupinen@palvellatx.com
Media
Marcy Nanus
Vice President of Investor Relations and Corporate Affairs
Palvella Therapeutics
marcy.nanus@palvellatx.com
View the Zacks #1 Rank List
Palvella Therapeutics to Provide Public Comment at FDA Meeting on Drug Repurposing Criteria and Prioritization
A hybrid public meeting on August 5 will discuss FDA-approved drug repurposing to enhance patient access to new therapies.
Quiver AI Summary
Palvella Therapeutics, Inc. announced its participation in a hybrid public meeting on August 5, 2026, organized by the FDA to discuss strategies for selecting and prioritizing promising repurposed FDA-approved drugs for treatment of serious, rare diseases with unmet medical needs. Dr. Jeffrey Martini, Chief Scientific Officer of Palvella, will provide insights on evidence frameworks and regulatory approaches for advancing these therapies, highlighting the importance of combining established safety profiles with real-world evidence of clinical benefits. The meeting aims to foster collaboration among federal agencies, clinicians, researchers, and patient organizations to enhance opportunities for repurposing approved medications. Palvella, which focuses on developing novel treatments for rare skin diseases, emphasizes the potential of leveraging existing drugs for new therapeutic indications as a part of the FDA's ongoing drug repurposing initiative.
Potential Positives
Potential Negatives
FAQ
What is the purpose of the FDA public meeting on August 5th?
The meeting will explore ways to select and prioritize promising FDA-approved drugs for repurposing to accelerate patient access to effective therapies.
Who will represent Palvella Therapeutics at the FDA meeting?
Dr. Jeffrey Martini, the Chief Scientific Officer of Palvella Therapeutics, has been selected to provide public comment at the meeting.
How does repurposing FDA-approved drugs benefit patients?
Repurposing can provide targeted therapies for serious, rare diseases by leveraging established safety profiles and real-world clinical evidence.
What is Palvella's focus as a biopharmaceutical company?
Palvella Therapeutics focuses on developing therapies for serious, rare skin diseases and vascular malformations lacking FDA-approved treatments.
What are QTORIN™ rapamycin and QTORIN™ pitavastatin?
They are investigational product candidates from Palvella, targeting specific rare diseases, currently under development and not yet FDA-approved.
Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.
$PVLA Insider Trading Activity
$PVLA insiders have traded $PVLA stock on the open market 20 times in the past 6 months. Of those trades, 3 have been purchases and 17 have been sales.
Here’s a breakdown of recent trading of $PVLA stock by insiders over the last 6 months:
To track insider transactions, check out Quiver Quantitative's insider trading dashboard. You can access data on insider stock transactions through the Quiver Quantitative API insider transaction endpoint.
$PVLA Hedge Fund Activity
We have seen 123 institutional investors add shares of $PVLA stock to their portfolio, and 43 decrease their positions in their most recent quarter.
Here are some of the largest recent moves:
To track hedge funds' stock portfolios, check out Quiver Quantitative's institutional holdings dashboard. You can access data on hedge funds moves and 13F filings through the Quiver Quantitative API 13F endpoint.
$PVLA Analyst Ratings
Wall Street analysts have issued reports on $PVLA in the last several months. We have seen 1 firms issue buy ratings on the stock, and 0 firms issue sell ratings.
Here are some recent analyst ratings:
To track analyst ratings and price targets for $PVLA, check out Quiver Quantitative's $PVLA forecast page.
$PVLA Price Targets
Multiple analysts have issued price targets for $PVLA recently. We have seen 5 analysts offer price targets for $PVLA in the last 6 months, with a median target of $240.0.
Here are some recent targets:
Full Release
Hybrid public meeting on August 5th will explore approaches to selecting and prioritizing promising FDA-approved drugs for repurposing, with the potential to accelerate patient access to effective therapies for significant unmet medical needs
Dr. Martini to discuss evidence frameworks and regulatory approaches for advancing repurposed therapies for patients with serious, rare diseases
Meeting builds on FDA’s May 2026 drug repurposing initiative focused on advancing new uses for approved medicines, particularly in chronic or rare diseases with significant unmet medical needs
WAYNE, Pa., July 30, 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, announced today that Jeffrey Martini, Ph.D., Chief Scientific Officer of Palvella, has been selected to provide public comment at Drug Repurposing: Considerations for Selection Criteria and Prioritization , an FDA public meeting to be held on August 5, 2026, in Washington, D.C.
“As pioneering research in rare diseases uncovers genetic drivers and causal biology, it can reveal opportunities to repurpose medicines as targeted therapies for previously untreatable diseases, building on accumulated scientific knowledge and clinical experience,” said Jeffrey Martini, Ph.D., Chief Scientific Officer of Palvella Therapeutics. “The combination of an established safety profile and emerging evidence from real-world use suggesting potential clinical benefit can provide a strong foundation for accelerating the development of repurposed therapies, particularly in chronic or rare diseases. I am honored to contribute to this important discussion on how scientific and regulatory frameworks can continue to foster innovation and accelerate access to meaningful therapies for patients with significant unmet medical needs.”
The hybrid public meeting, convened by the Reagan-Udall Foundation in collaboration with FDA, builds on the Agency’s broader initiative to identify new therapeutic uses for approved medicines and expand treatment options for patients with significant unmet medical needs. Bringing together representatives from federal agencies, clinicians, researchers, and patient organizations, the meeting will examine approaches for identifying and prioritizing promising repurposing candidates, as well as the evidence standards and regulatory considerations relevant to advancing them efficiently. During the public comment session, Dr. Martini is expected to address one of the meeting's central discussion questions: “What evidence gaps or policy/regulatory barriers most commonly hinder the advancement of promising repurposing opportunities?”
“Jeff’s selection to provide public comment at this important meeting reflects Palvella’s leadership in advancing first-in-disease therapies for serious, rare diseases through a unique approach that leverages well-characterized existing molecules and real-world clinical evidence,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella Therapeutics. “Recent years have seen growing momentum behind the development of established molecules for new therapeutic indications, particularly in rare diseases, as advances in the understanding of disease biology create opportunities to apply these molecules as on-target therapies that can directly address the causal drivers of disease. Novel, indication-specific formulations of existing molecules can further enhance the overall benefit-risk profile for patients. We appreciate the opportunity to contribute our perspective as FDA continues to advance scientific and regulatory approaches that can help bring meaningful new therapies to patients.”
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.
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 plans with respect to the timing of, and anticipated FDA review process for, the NDA for QTORIN™ rapamycin, Palvella’s plans to pursue Breakthrough Therapy Designation, Palvella’s plans to meet with regulatory authorities, Palvella’s expectations regarding the benefits of 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 Chief Executive Officer
Palvella Therapeutics
wes.kaupinen@palvellatx.com
Media
Marcy Nanus
Vice President of Investor Relations and Corporate Affairs
Palvella Therapeutics
marcy.nanus@palvellatx.com
Read full article here »