XORTX Provides Update on FDA Type B Meeting Request
XORTX Therapeutics has received crucial FDA feedback regarding their new drug application (NDA) for XORLO™, a novel oxypurinol formulation targeting gout treatment. The Type B meeting with FDA reviewed chemistry, manufacturing, pharmacology, toxicology, and clinical evidence for the XRx-026 program.
The company will proceed with five key steps:
- Finalizing FDA meeting minutes
- Filing an Investigative New Drug application
- Characterizing pharmacokinetics of XORLO™ commercial tablet
- Manufacturing and validating commercial drug supplies
- Preparing and filing the NDA
XORTX aims to file the gout NDA in H1 2026, advancing through the FDA 505(b)2 development pathway. The company, which focuses on innovative therapies for gout and progressive kidney disease, will provide updates once FDA meeting minutes are finalized.
XORTX Therapeutics ha ricevuto un feedback fondamentale dalla FDA riguardo alla loro domanda di nuovo farmaco (NDA) per XORLO™, una nuova formulazione di ossipurinolo destinata al trattamento della gotta. L'incontro di tipo B con la FDA ha esaminato chimica, produzione, farmacologia, tossicologia e dati clinici relativi al programma XRx-026.
L'azienda procederà con cinque passi chiave:
- Finalizzazione dei verbali dell'incontro con la FDA
- Presentazione di una domanda di nuovo farmaco sperimentale
- Caratterizzazione della farmacocinetica della compressa commerciale di XORLO™
- Produzione e validazione delle forniture commerciali del farmaco
- Preparazione e presentazione della NDA
XORTX punta a presentare la NDA per la gotta nella prima metà del 2026, avanzando lungo il percorso di sviluppo FDA 505(b)2. L'azienda, specializzata in terapie innovative per la gotta e le malattie renali progressive, fornirà aggiornamenti una volta finalizzati i verbali dell'incontro con la FDA.
XORTX Therapeutics ha recibido comentarios cruciales de la FDA sobre su solicitud de nuevo fármaco (NDA) para XORLO™, una nueva formulación de oxipurinol dirigida al tratamiento de la gota. La reunión de tipo B con la FDA revisó química, fabricación, farmacología, toxicología y evidencia clínica del programa XRx-026.
La compañía procederá con cinco pasos clave:
- Finalizar las actas de la reunión con la FDA
- Presentar una solicitud de nuevo fármaco en investigación
- Caracterizar la farmacocinética del comprimido comercial de XORLO™
- Fabricar y validar los suministros comerciales del fármaco
- Preparar y presentar la NDA
XORTX planea presentar la NDA para la gota en el primer semestre de 2026, avanzando a través de la vía de desarrollo FDA 505(b)2. La compañía, que se centra en terapias innovadoras para la gota y la enfermedad renal progresiva, proporcionará actualizaciones una vez finalizadas las actas de la reunión con la FDA.
XORTX Therapeutics는 통풍 치료를 목표로 하는 새로운 옥시퓨리놀 제형인 XORLO™에 대한 신약 신청서(NDA)에 대해 FDA로부터 중요한 피드백을 받았습니다. FDA와의 Type B 미팅에서는 화학, 제조, 약리학, 독성학 및 임상 증거가 XRx-026 프로그램에 대해 검토되었습니다.
회사는 다섯 가지 주요 단계를 진행할 예정입니다:
- FDA 미팅 회의록 최종화
- 임상시험용 신약 신청서 제출
- XORLO™ 상업용 정제의 약동학 특성화
- 상업용 의약품 공급 제조 및 검증
- NDA 준비 및 제출
XORTX는 2026년 상반기에 통풍 NDA를 제출할 계획이며, FDA 505(b)2 개발 경로를 따라 진행할 예정입니다. 통풍 및 진행성 신장 질환을 위한 혁신 치료제에 집중하는 이 회사는 FDA 미팅 회의록이 최종화되는 대로 업데이트를 제공할 것입니다.
XORTX Therapeutics a reçu des retours importants de la FDA concernant sa demande d'autorisation de mise sur le marché (NDA) pour XORLO™, une nouvelle formulation d'oxypurinol destinée au traitement de la goutte. La réunion de type B avec la FDA a examiné la chimie, la fabrication, la pharmacologie, la toxicologie et les données cliniques du programme XRx-026.
L'entreprise va procéder à cinq étapes clés :
- Finaliser le procès-verbal de la réunion avec la FDA
- Déposer une demande de nouveau médicament expérimental
- Caractériser la pharmacocinétique du comprimé commercial de XORLO™
- Fabriquer et valider les stocks commerciaux du médicament
- Préparer et déposer la NDA
XORTX vise à déposer la NDA pour la goutte au premier semestre 2026, en suivant la voie de développement FDA 505(b)2. L'entreprise, spécialisée dans les thérapies innovantes pour la goutte et les maladies rénales progressives, fournira des mises à jour une fois le procès-verbal de la réunion finalisé.
XORTX Therapeutics hat wichtiges Feedback der FDA zu ihrem Antrag auf Zulassung eines neuen Medikaments (NDA) für XORLO™ erhalten, eine neuartige Oxipurinol-Formulierung zur Behandlung von Gicht. Das Type-B-Treffen mit der FDA überprüfte Chemie, Herstellung, Pharmakologie, Toxikologie und klinische Daten des XRx-026-Programms.
Das Unternehmen wird fünf wichtige Schritte durchführen:
- Abschluss des Protokolls der FDA-Sitzung
- Einreichung eines Antrags für ein Prüfpräparat
- Charakterisierung der Pharmakokinetik der kommerziellen XORLO™-Tablette
- Herstellung und Validierung der kommerziellen Arzneimittelversorgung
- Vorbereitung und Einreichung der NDA
XORTX plant, die NDA für Gicht im ersten Halbjahr 2026 einzureichen und folgt dabei dem FDA 505(b)2-Entwicklungsweg. Das Unternehmen, das sich auf innovative Therapien für Gicht und fortschreitende Nierenerkrankungen spezialisiert hat, wird Updates bereitstellen, sobald das Protokoll der FDA-Sitzung abgeschlossen ist.
- FDA provided clear guidance for NDA submission pathway for gout treatment XORLO™
- Company confirmed viable 505(b)2 development pathway for marketing approval
- NDA filing timeline established for H1 2026
- FDA substantially confirmed key steps for marketing approval
- Multiple regulatory steps still required before NDA submission
- Commercial formulation requires additional pharmacokinetic studies
- Manufacturing validation and stability data still pending
- Final FDA meeting minutes not yet available
Insights
FDA confirmation of 505(b)(2) pathway for XORTX's gout treatment significantly reduces regulatory uncertainty and establishes clear development timeline.
The FDA's feedback on XORTX's Type B meeting represents a significant regulatory milestone for their XRx-026 gout program. The agency has substantially confirmed the 505(b)(2) development pathway for XORLO™, their proprietary oxypurinol formulation, which is strategically valuable as it enables partial reliance on previously established safety/efficacy data, potentially reducing development time and costs compared to traditional NDAs.
The FDA has clearly outlined specific requirements including: IND filing, pharmacokinetic characterization with and without food, and manufacturing validation with stability data. These represent standard regulatory steps but now with confirmed specificity for this program. Type B meetings like this one are critical for establishing alignment with regulators on development plans, so this confirmation meaningfully reduces regulatory uncertainty.
The projected H1 2026 NDA submission timeline provides a concrete development horizon, suggesting approximately 12-18 months of remaining preparatory work. The 505(b)(2) pathway is particularly appropriate for reformulations of known active ingredients like oxypurinol, efficiently balancing regulatory thoroughness with development practicality while potentially streamlining the path to market.
FDA's confirmation of streamlined regulatory pathway for XORTX's gout treatment reduces development risk and establishes clear timeline to potential commercialization.
This FDA interaction provides crucial clarity on XORTX's development roadmap and path to potential commercialization for their gout treatment. The confirmation of the 505(b)(2) regulatory pathway is particularly significant as this approach typically requires less extensive original clinical data than standard NDAs, potentially reducing both development costs and timeline risk for investors.
The H1 2026 target for NDA submission establishes a concrete milestone that investors can use to track program progress. The clearly defined next steps—IND filing, pharmacokinetic studies, and manufacturing validation—create a more transparent development trajectory for this asset. While substantial work remains before potential approval, the regulatory clarity significantly reduces one key area of uncertainty in the investment thesis.
Gout represents a substantial market opportunity with over 9 million sufferers in the US alone. The company's characterization of XORLO™ as a "much needed therapeutic option" suggests they've identified a market need, positioning this program as a potential value driver. This regulatory update enhances visibility into XORTX's clinical-stage pipeline progression and establishes a clearer path to potential market entry for their lead program.
● XORTX will focus on key steps
to advance a NDA filing for Gout indication ●
CALGARY, Alberta, April 30, 2025 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late stage clinical pharmaceutical company focused on developing innovative therapies to treat gout and progressive kidney disease, is pleased to announce it has received responses from the US Food and Drug Administration (the “FDA”) and clarified key steps for a new drug application (“NDA”) for the Company’s novel proprietary formulation of oxypurinol for the treatment of gout. The Type B meeting conducted by the FDA, included review of chemistry, manufacturing, pharmacology, toxicology and clinical evidence to support the Company’s XRx-026 program for the treatment of gout. Responses from the FDA substantially confirmed and clarified the remaining key steps necessary prior to filing a NDA to gain marketing approval for XORLO™ in the US using the FDA 505(b)2 development pathway.
With this FDA guidance, XORTX will now advance the following key steps to support the NDA application and its submission for the XRx-026 program for gout.
i) Finalize meeting minutes with the FDA regarding the agency and XORTX’s responses;
ii) Prepare and file an Investigative New Drug (“IND”) application for the XRx-026 program;
iii) Characterize pharmacokinetics of the commercial tablet formulation of XORLO™ in absence and in the presence of food;
iv) Manufacture, validate commercial supplies of drug accompanied by stability data in parallel with the NDA preparation; and
v) Prepare and file a NDA for marketing approval of XORLO™.
Dr. Allen Davidoff, CEO of XORTX commented, “The clarity gained during this review of supporting data with the FDA will permit the further advancement of the XRx-026 program and will facilitate the timely completion of key steps necessary for filing the NDA. The Company believes that the gout NDA may be filed in the H1 2026. We continue to prioritize our XRx-026 program and advance this much needed therapeutic option for individuals with gout.”
The Company will provide further updates following final communications with the FDA once meeting minutes are finalized and available.
About Hyperuricemia and Gout
In the US it is estimated that approximately 44 million individuals have circulating uric acid above the normal range(1). The prevalence of gout was
About the XRx-026 Program and XORLOTM
The XRx-026 program is developing XORLO™, a proprietary formulation of oxypurinol to treat individuals suffering from gout. At present, oral xanthine oxidase inhibitors (“XOIs”) are the preferred therapeutic option used to inhibit the production of uric acid and decrease chronically high uric acid in the circulation. Allopurinol is the most commonly prescribed XOI, with approximately 3.3 million prescriptions written per year in North America, however 3 to
About Type B Meetings with the FDA
Type B meetings for each potential application (e.g., investigational new drug application (IND), NDA, biologics license application (BLA)) or combination of closely related products developed by the same sponsor or applicant (e.g., same active ingredient but different dosage forms being developed concurrently). Typically, it may be appropriate to conduct more than one of some of the Type B meetings for concurrent development of a product for unrelated claims.
References:
(1) Chen-Xu M_Prevalence of Gout and Hyperuricemia in the US_ArthritisRheumatol_71-6_991-999_2019_nihms-1002533
(2) Singh JA. Quality of life and quality of care for patients with gout. Curr Rheumatol Rep. 2009 4;11(2):154–60. [PubMed: 19296889]
(3) Burke BT, Köttgen A, Law A, Windham BG, Segev D, Baer AN, et al. Physical Function, Hyperuricemia, and Gout in Older Adults. Arthritis Care Res. 2015 12;67(12):1730–8.
(4) Rai SK, Burns LC, De Vera MA, Haji A, Giustini D, Choi HK. The economic burden of gout: A systematic review. Semin Arthritis Rheum. 2015 8;45(1):75–80. [PubMed: 25912932]
(5) Choi HK, Ford ES. Prevalence of the metabolic syndrome in individuals with hyperuricemia. Am J Med. 2007 5;120(5):442–7. [PubMed: 17466656]
(6) Choi HK, Curhan G. Independent impact of gout on mortality and risk for coronary heart disease. Circulation. 2007 8 21;116(8):894–900. [PubMed: 17698728]
(7) Liu S-C, Xia L, Zhang J, Lu X-H, Hu D-K, Zhang H-T, et al. Gout and Risk of Myocardial Infarction: A Systematic Review and Meta-Analysis of Cohort Studies. Pizzi C, editor. PLOS ONE. 2015 7 31;10(7):e0134088. [PubMed: 26230580]
(8) Choi HK, De Vera MA, Krishnan E. Gout and the risk of type 2 diabetes among men with a high cardiovascular risk profile. Rheumatology. 2008 8 13;47(10):1567–70. [PubMed: 18710901]
(9) Roughley MJ, Belcher J, Mallen CD, Roddy E. Gout and risk of chronic kidney disease and nephrolithiasis: meta-analysis of observational studies. Arthritis Res Ther. 2015 12;17(1).
(10) Kuo C-F, See L-C, Luo S-F, Ko Y-S, Lin Y-S, Hwang J-S, et al. Gout: an independent risk factor for all-cause and cardiovascular mortality. Rheumatology. 2010 1;49(1):141–6. [PubMed: 19933595]
(11) Fisher MC, Rai SK, Lu N, Zhang Y, Choi HK. The unclosing premature mortality gap in gout: a general population-based study. Ann Rheum Dis. 2017 7;76(7):1289–94. [PubMed: 28122760]
About XORTX Therapeutics Inc.
XORTX is a pharmaceutical company with three clinically advanced products in development: 1) our lead program XRx-026 program for the treatment of gout; 2) XRx-008 program for ADPKD; and 3) XRx-101 for acute kidney and other acute organ injury associated with respiratory virus infections. In addition, the Company is developing XRx-225, a pre-clinical stage program for Type 2 diabetic nephropathy. XORTX is working to advance products that target aberrant purine metabolism and xanthine oxidase to decrease or inhibit production of uric acid. At XORTX, we are dedicated to developing medications that improve the quality of life and health of individuals with gout and other important diseases. Additional information on XORTX is available at www.xortx.com.
| For more information, please contact: | |
| Allen Davidoff, CEO | Nick Rigopulos, Director of Communications |
| adavidoff@xortx.com or +1 403 455 7727 | nick@alpineequityadv.com or +1 617 901 0785 |
Neither the TSX Venture Exchange nor Nasdaq has approved or disapproved the contents of this news release. No stock exchange, securities commission or other regulatory authority has approved or disapproved the information contained herein.
Forward Looking Statements
This press release contains express or implied forward-looking statements pursuant to applicable securities laws. These forward-looking statements include, but are not limited to, the Company's beliefs, plans, goals, objectives, expectations, assumptions, estimates, intentions, future performance, other statements that are not historical facts and statements identified by words such as "expects", "anticipates", "intends", "plans", "believes", "seeks", "estimates" or words of similar meaning. These forward-looking statements and their implications are based on the current expectations of the management of XORTX only, and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Such risks, uncertainties, and other factors include, but are not limited to, our ability to obtain additional financing; the accuracy of our estimates regarding expenses, future revenues and capital requirements; the success and timing of our preclinical studies and clinical trials; the performance of third-party manufacturers and contract research organizations; our plans to develop and commercialize our product candidates; our plans to advance research in other kidney disease applications; and, our ability to obtain and maintain intellectual property protection for our product candidates. Except as otherwise required by applicable law and stock exchange rules, XORTX undertakes no obligation to publicly release any revisions to these forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. More detailed information about the risks and uncertainties affecting XORTX is contained under the heading “Risk Factors” in XORTX’s Annual Report on Form 20-F filed with the SEC, which is available on the SEC's website, www.sec.gov (including any documents forming a part thereof or incorporated by reference therein), as well as in our reports, public disclosure documents and other filings with the securities commissions and other regulatory bodies in Canada, which are available on www.sedarplus.ca.
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1 Source: Takeda Pharmaceutical Company 2018 Annual Report.
FAQ
What is XORTX's (XRTX) timeline for FDA approval of XORLO for gout treatment?
What are the next steps for XORTX (XRTX) after the FDA Type B meeting?
How will XORTX's XORLO drug formulation treat gout patients?
What regulatory pathway is XORTX (XRTX) using for XORLO approval?
What did the FDA Type B meeting review for XORTX's gout treatment?
