Febuxostat

作者 | 陈芋责编 | 遥望从340元降至4.8元，大批药品挂网价下调。01价格超1.8倍被标记本月底前完成自查降价近日，陕西公共资源交易中心发布《关于对平台异常挂网价格自查自纠的通知》，剑指高价药。据通知，陕西此次以近一年的“加权平均交易价”为锚点，药品当前挂网价与近1年来加权平均交易价比值大于等于1.8倍的，将在省平台被标红管理。医药企业可登陆平台查看标红的相关产品及当前比值（加权平均交易价的计算时间范围为：2024年5月1日至2025年4月30日）。陕西提出，有标红药品及医用耗材产品的企业应在5月31日前进行自查自纠，并主动将挂网价与加权平均交易价的比值降至1.3倍以下，降价后的产品将在6月初取消红色标识，逾期拒绝整改的异常挂网价格产品，将被上报省医保局，按相关规定统一处理。海南也在本月启动了挂网药品价格调整工作，同样对高价药采取红黄标识，但其触发条件与陕西有所不同。不同于陕西的加权平均交易价，海南以近年药品价格治理中新引入的“75%分位数”作为锚点——高于该值1.8倍不高于3倍的，纳入黄标管理；高于3倍的，纳入红标管理。75%分位数是把药品价格按升序排列后找到的一个临界值，即同组中约有75%的药品价格小于等于该值，其余25%的药品价格大于等于该值。海南此批名单共披露436个挂网价高于75%分位数价格1.8倍的药品（名单见文末附1），其中198个因价格高于3倍被标红，238个被标黄。海南提出，企业应在5月12日17:00前登录子系统将价格调至1.8倍以下，符合条件后取消红黄标管理。02从340元降至4.8元上千个药品挂网价调整不止陕西、海南，5月以来，多省相继公布本省挂网价格治理工作进展，上海发布《部分药品红线价格调整公告》，河南发布《关于尼扎替丁分散片等100个药品下调价格的公示》，湖南发布《关于药品挂网价格调整的公告》，江苏发布《关于调整部分药品挂网价格的通知》等；4月，吉林也公布了其《挂网药品价格自查自纠专项治理结果》。从已公布下调结果的省份来看，各省近期的挂网价格调整涉及大批药品。例如，吉林公布的自查自纠专项治理结果共包括2081个药品，涉及注射用头孢曲松钠、磷酸西格列汀片、非布司他片等品种。吉林两千余个药品整体价格降幅约20%，其中，310个药品价格降幅超过50%，降幅最高的一款产品为利伐沙班片，价格从339.72元/盒调整至4.8元/盒，降幅达到98.6%。对于未参与此次调价的企业，吉林表示可于每月1日-10日通过招采子系统展开降价，未按相关要求及时开展价格联动的企业，采购中心将按照国家医保局《关于建立医药价格和招采信用评价制度的指导意见》相关规定处理。河南发布此批公示名单中包括100个药品（名单及企业、价格等见文末附2），涉及紫杉醇注射液、头孢地尼分散片、注射用更昔洛韦等品种，整体平均降幅约14%，其中，降幅最高超过93%，为一款甲磺酸雷沙吉兰片，价格由287.9元按承诺拟供应价降至18元。两年前，国家医保局启动药品挂网价格治理，后逐步完成四同药品价格治理等工作，到2025年，治理进一步深化。全国医疗保障工作会议上，国家医保局明确2025年的八大重点之一是深化医药价格改革治理，不断规范医药价格秩序——推进挂网药品价格治理，推动形成全国药品挂网价格规则共识，全面建立定点药店医保药品量价比较指数等。在医保局组织逐步建立的全国挂网药品价格一览表下，各地挂网价格信息将被汇总，首涨、高涨幅等异常行为也将被标识，各省对各类不合理的挂网高价的纠正还将持续推进。附1：河南关于尼扎替丁分散片等100个药品下调价格的公示附2：海南436个挂网价高于75%分位数价格1.8倍的药品END内容沟通：郑瑶（13810174402）

● 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 3.9% or 9.2 million individuals. Mean serum urate levels were 6.0 mg/dL among men and 4.8 mg/dL among women, with hyperuricemia prevalences of 20.2% and 20.0%, respectively. The prevalence of ULT use among patients with gout was 33% during 2007 to 2014 and remained stable over time (P for trend >0.05)(1). Gout is an inflammatory arthritis that is triggered by the crystallization of monosodium urate inside the joints and is preceded by hyperuricemia. Gout flares lead to substantial morbidity by causing severe pain, reduced quality of life(2), decreased physical function(2,3), increased healthcare costs(4), and lost economic productivity(5). Furthermore, gout is strongly associated with the metabolic syndrome(5), and may contribute to myocardial infarction(6,7), type 2 diabetes mellitus(8), chronic kidney disease(9), and premature mortality(6,10,11). 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 5% of patients cannot tolerate Allopurinol. An alternative XOI, Febuxostat, was launched in the US in 2009 with the hope of treating individuals with gout, however while Febuxostat achieved peak sales greater than US$450 million1 after its launch, a Black Box warning due to its associated risk of sudden cardiovascular death resulted in a decline in its use. XORLO™ can address this unmet medical need and accelerating advancement of the XRx-026 program through an NDA filing is now a priority for XORTX. 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, CEONick Rigopulos, Director of Communicationsadavidoff@xortx.com or +1 403 455 7727nick@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. ___________________ 1 Source: Takeda Pharmaceutical Company 2018 Annual Report.