Palvella Therapeutics to Expand Phase 3 SELVA Clinical Trial of QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) for the Treatment of Microcystic Lymphatic Malformations to Include the Younger Pediatric Population, Children 3 to 5 Years Old
Palvella Therapeutics (PVLA) announced the expansion of its Phase 3 SELVA clinical trial for QTORIN™ 3.9% rapamycin anhydrous gel to include children aged 3-5 years old, following FDA approval. The trial, previously to patients 6 years and older, evaluates the treatment for microcystic lymphatic malformations (microcystic LMs).
The company is currently enrolling approximately 40 patients in the 24-week, single-arm, baseline-controlled trial. QTORIN™ rapamycin has received Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation from the FDA. The study is supported by an FDA Orphan Products Grant of up to $2.6 million and aims to report top-line results in Q1 2026.
If successful, QTORIN™ rapamycin could become the first FDA-approved therapy for microcystic LMs in the U.S.
Palvella Therapeutics (PVLA) ha annunciato l'espansione del suo trial clinico di Fase 3 SELVA per il gel anidro di rapamicina QTORIN™ 3.9% per includere bambini di età compresa tra 3 e 5 anni, dopo l'approvazione della FDA. Il trial, precedentemente riservato a pazienti di 6 anni e oltre, valuta il trattamento per le malformazioni linfatiche microcistiche (microcystic LMs).
L'azienda sta attualmente arruolando circa 40 pazienti nello studio controllato di 24 settimane, a braccio singolo e con baseline. La rapamicina QTORIN™ ha ricevuto la Designazione di Terapia Innovativa, Designazione Fast Track e Designazione di Farmaco Orfano dalla FDA. Lo studio è supportato da un finanziamento per Prodotti Orfani della FDA fino a 2.6 milioni di dollari e mira a pubblicare i risultati preliminari nel primo trimestre del 2026.
Se avrà successo, la rapamicina QTORIN™ potrebbe diventare la prima terapia approvata dalla FDA per le LMs microcistiche negli Stati Uniti.
Palvella Therapeutics (PVLA) anunció la expansión de su ensayo clínico de Fase 3 SELVA para el gel anhidro de rapamicina QTORIN™ 3.9% para incluir a niños de 3 a 5 años, tras la aprobación de la FDA. El ensayo, que anteriormente era para pacientes de 6 años en adelante, evalúa el tratamiento para malformaciones linfáticas microquísticas (microcystic LMs).
La empresa está actualmente reclutando aproximadamente a 40 pacientes en el ensayo controlado por línea base de 24 semanas y brazo único. La rapamicina QTORIN™ ha recibido la Designación de Terapia Innovadora, Designación de Vía Rápida y Designación de Medicamento Huérfano por parte de la FDA. El estudio cuenta con el apoyo de una Subvención de Productos Huérfanos de la FDA de hasta 2.6 millones de dólares y tiene como objetivo informar los resultados preliminares en el primer trimestre de 2026.
Si tiene éxito, la rapamicina QTORIN™ podría convertirse en la primera terapia aprobada por la FDA para las LMs microquísticas en los EE. UU.
Palvella Therapeutics (PVLA)는 QTORIN™ 3.9% 라파마이신 무수겔의 3상 SELVA 임상시험을 3-5세 아동을 포함하도록 확대한다고 발표했습니다. 이는 FDA의 승인을 받은 것입니다. 이 시험은 이전에 6세 이상의 환자만을 대상으로 하였으며, 미세 낭포성 림프 기형(microcystic LMs) 치료를 평가합니다.
현재 회사는 24주 동안 진행되는 단일군 기준 조절 시험에 약 40명의 환자를 모집하고 있습니다. QTORIN™ 라파마이신은 FDA로부터 혁신 치료 지정, 신속 승인 지정 및 희귀 약물 지정을 받았습니다. 이 연구는 최대 260만 달러의 FDA 희귀 제품 보조금으로 지원되며, 2026년 1분기 중 결과를 발표할 계획입니다.
성공적으로 진행될 경우 QTORIN™ 라파마이신은 미국에서 미세 낭포성 LMs에 대한 첫 번째 FDA 승인 치료제가 될 수 있습니다.
Palvella Therapeutics (PVLA) a annoncé l'extension de son essai clinique de Phase 3 SELVA pour le gel anhydre de rapamycine QTORIN™ 3.9% afin d'inclure des enfants âgés de 3 à 5 ans, suite à l'approbation de la FDA. L'essai, qui était auparavant réservé aux patients de 6 ans et plus, évalue le traitement des malformations lymphatiques microcystiques (microcystic LMs).
L'entreprise recrute actuellement environ 40 patients pour cet essai contrôlé de 24 semaines, à bras unique. La rapamycine QTORIN™ a reçu la désignation de thérapie innovante, la désignation de voie rapide et la désignation de médicament orphelin de la FDA. L'étude bénéficie d'une subvention de produits orphelins de la FDA allant jusqu'à 2,6 millions de dollars et vise à publier des résultats préliminaires au premier trimestre 2026.
Si cela est un succès, la rapamycine QTORIN™ pourrait devenir la première thérapie approuvée par la FDA pour les LMs microcystiques aux États-Unis.
Palvella Therapeutics (PVLA) hat die Erweiterung seiner Phase 3 SELVA-Studie für QTORIN™ 3.9% Anhydrous Rapamycin Gel bekannt gegeben, um Kinder im Alter von 3 bis 5 Jahren nach Genehmigung durch die FDA einzuschließen. Die Studie richtete sich ursprünglich an Patienten ab 6 Jahren und bewertet die Behandlung von mikrozystischen lymphatischen Malformationen (microcystic LMs).
Das Unternehmen rekrutiert derzeit etwa 40 Patienten für die 24-wöchige, einarmige, kontrollierte Studie. QTORIN™ Rapamycin hat von der FDA die Breakthrough Therapy Designation, Fast Track Designation und Orphan Drug Designation erhalten. Die Studie wird durch einen FDA Orphan Products Grant von bis zu 2,6 Millionen USD unterstützt und hat das Ziel, die vorläufigen Ergebnisse im ersten Quartal 2026 zu veröffentlichen.
Wenn es erfolgreich ist, könnte QTORIN™ Rapamycin die erste von der FDA zugelassene Therapie für mikrozystische LMs in den USA werden.
- FDA approval to expand trial to younger pediatric population (3-5 years)
- Multiple FDA designations secured (Breakthrough, Fast Track, Orphan Drug)
- $2.6 million FDA grant support
- Potential first-to-market advantage for microcystic LMs treatment
- Top-line results not expected until Q1 2026
- trial size of only 40 patients
Insights
The expansion of Palvella's Phase 3 SELVA trial to include children ages 3-5 represents a significant market opportunity enhancement. This strategic move could expand the addressable patient population by approximately 40%, as microcystic LMs typically manifest at birth or early childhood.
The regulatory positioning is particularly compelling. The triple designation from the FDA (Breakthrough Therapy, Fast Track and Orphan Drug) provides several advantages: 1) Expedited review process, potentially reducing time to market by 4-6 months 2) Enhanced FDA interaction during development 3) 7 years of market exclusivity upon approval 4) Potential priority review voucher worth
The
As the first potential FDA-approved therapy for microcystic LMs, Palvella would benefit from:
- First-mover advantage in a virgin market
- Potential for premium pricing due to orphan status
- Stronger position in negotiating coverage with insurers
- Higher barriers to entry for competitors
The single-arm trial design with approximately 40 patients suggests confidence in the treatment's efficacy, while the baseline-controlled approach aligns with regulatory precedent for rare diseases. The expanded age range could accelerate enrollment and potentially improve the statistical power of the study.
Expansion reflects Palvella's commitment to serving all patients affected by microcystic lymphatic malformations (microcystic LMs)
Company remains on track to report top line results from SELVA, a Phase 3 single-arm, baseline-controlled trial evaluating QTORIN™ rapamycin for the treatment of microcystic LMs, in Q1 2026
QTORIN™ rapamycin has the potential to be the first approved therapy and standard of care in the U.S. for microcystic LMs
WAYNE, Pa., Feb. 10, 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 FDA-approved therapies, today announced that it will expand SELVA, the Company's Phase 3 clinical trial of QTORIN™
“Microcystic LMs is a debilitating disease that is chronic, progressive, and usually present at birth or shortly after. Patients with microcystic LMs often have lymphorrhea, bleeding, infection, pain, and disfigurement which may lead to difficulty with physical activities as well as more significant complications like cellulitis and hospitalization,” said Joyce Teng MD, PhD, Professor of Dermatology and Pediatrics at Stanford University and Principal Investigator of the SELVA Study. “Early intervention is essential to minimize disease burden during children’s development which is why I am so excited by the opportunity QTORIN rapamycin presents to the younger pediatric population.”
Palvella is currently enrolling approximately 40 patients in SELVA, a 24-week, Phase 3, single-arm, baseline-controlled trial of QTORIN™ rapamycin for the treatment of microcystic LMs. The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation to QTORIN™ rapamycin for the treatment of microcystic LMs. Additionally, the SELVA study is supported by an Orphan Products Grant of up to
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 drug disease drug development veterans, Palvella Therapeutics (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.
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 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 “Risk Factors” section of Palvella’s registration statement on Form S-1 filed with the Securities and Exchange Commission on December 31, 2024 and other documents filed by Palvella from time to time with the Securities Exchange Commission. 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.
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Contact Information
Investors
Wesley H. Kaupinen
Founder and CEO, Palvella Therapeutics
wes.kaupinen@palvellatx.com
Media
Stephanie Jacobson
Managing Director, Argot Partners
palvella@argotpartners.com
FAQ
When will Palvella (PVLA) report Phase 3 SELVA trial results for QTORIN rapamycin?
What age groups can now participate in PVLA's SELVA trial for QTORIN rapamycin?
How many patients will be enrolled in PVLA's Phase 3 SELVA trial?
What FDA designations has PVLA's QTORIN rapamycin received for microcystic LMs?
How much funding did PVLA receive from the FDA's Orphan Products Grant?
