Vadadustat

Designed to help dialysis organizations incorporate new treatments into their practices, TDAPA provides two years of reimbursement in addition to the ESRD bundled rate A HCPCS code has also been assigned to Vafseo to facilitate reimbursement at dialysis organizations CAMBRIDGE, Mass., Oct. 10, 2024 /PRNewswire/ -- Akebia Therapeutics®, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, today announced that the Center for Medicare & Medicaid Services (CMS) has determined that Vafseo® (vadadustat) meets the criteria for the Transitional Drug Add-On Payment Adjustment (TDAPA) in the anemia management end-stage renal disease (ESRD) prospective payment system functional category, beginning on January 1, 2025. In March 2024, Vafseo was approved by the U.S. Food and Drug Administration for the treatment of anemia due to chronic kidney disease in adults who have been receiving dialysis for at least three months, and the product is expected to be available in the market in January 2025. The TDAPA program provides two years of reimbursement for Vafseo in addition to the ESRD bundled rate to dialysis organizations. Additionally, Akebia received a Level II Healthcare Common Procedure Coding System (HCPCS) code for Vafseo which will be used for billing for the product by dialysis organizations for Medicare enrollees. Within the next several weeks, CMS is expected to issue a Medicare Claims Processing Change Request that will give further billing guidance to dialysis organizations to obtain the separate TDAPA payment for the next two years under Medicare. "We recognize the important benefit of TDAPA to support dialysis organizations' efforts to bring innovation to patients and incorporate new treatments into their practices," said Nicholas Grund, Chief Commercial Officer of Akebia. "The TDAPA status granted by CMS and issuance of a billing code are the latest milestones marking progress in the Vafseo commercial launch. We continue to actively engage dialysis organizations on the contracting process to facilitate access to Vafseo and look forward to further interacting with nephrologists and healthcare providers at the upcoming American Society of Nephrology conference, as we continue to build interest in Vafseo with a goal to drive usage and demand." About Akebia Therapeutics Akebia Therapeutics, Inc. is a fully integrated biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease. Akebia was founded in 2007 and is headquartered in Cambridge, Massachusetts. For more information, please visit our website at , which does not form a part of this release. About Vafseo® (vadadustat) Tablets Vafseo® (vadadustat) Tablets is a once-daily oral hypoxia-inducible factor prolyl hydroxylase inhibitor that activates the physiologic response to hypoxia to stimulate endogenous production of erythropoietin, increasing hemoglobin and red blood cell production to manage anemia. Vafseo is approved for use in 37 countries. INDICATION VAFSEO is indicated for the treatment of anemia due to chronic kidney disease (CKD) in adults who have been receiving dialysis for at least three months. Limitations of Use VAFSEO has not been shown to improve quality of life, fatigue, or patient well-being. VAFSEO is not indicated for use: As a substitute for red blood cell transfusions in patients who require immediate correction of anemia. In patients with anemia due to CKD not on dialysis. IMPORTANT SAFETY INFORMATION about VAFSEO (vadadustat) tablets CONTRAINDICATIONS Known hypersensitivity to VAFSEO or any of its components Uncontrolled hypertension WARNINGS AND PRECAUTIONS Increased Risk of Death, Myocardial Infarction (MI), Stroke, Venous Thromboembolism, and Thrombosis of Vascular Access A rise in hemoglobin (Hb) levels greater than 1 g/dL over 2 weeks can increase these risks. Avoid in patients with a history of MI, cerebrovascular event, or acute coronary syndrome within the 3 months prior to starting VAFSEO. Targeting a Hb level of greater than 11 g/dL is expected to further increase the risk of death and arterial and venous thrombotic events. Use the lowest effective dose to reduce the need for red blood cell (RBC) transfusions. Adhere to dosing and Hb monitoring recommendations to avoid excessive erythropoiesis. Hepatotoxicity Hepatocellular injury attributed to VAFSEO was reported in less than 1% of patients, including one severe case with jaundice. Elevated serum ALT, AST, and bilirubin levels were observed in 1.8%, 1.8%, and 0.3% of CKD patients treated with VAFSEO, respectively. Measure ALT, AST, and bilirubin before treatment and monthly for the first 6 months, then as clinically indicated. Discontinue VAFSEO if ALT or AST is persistently elevated or accompanied by elevated bilirubin. Not recommended in patients with cirrhosis or active, acute liver disease. Hypertension Worsening of hypertension was reported in 14% of VAFSEO and 17% of darbepoetin alfa patients. Serious worsening of hypertension was reported in 2.7% of VAFSEO and 3% of darbepoetin alfa patients. Cases of hypertensive crisis, including hypertensive encephalopathy and seizures, have also been reported in patients receiving VAFSEO. Monitor blood pressure. Adjust anti-hypertensive therapy as needed. Seizures Seizures occurred in 1.6% of VAFSEO and 1.6% of darbepoetin alfa patients. Monitor for new-onset seizures, premonitory symptoms, or change in seizure frequency. Gastrointestinal (GI) Erosion Gastric or esophageal erosions occurred in 6.4% of VAFSEO and 5.3% of darbepoetin alfa patients. Serious GI erosions, including GI bleeding and the need for RBC transfusions, were reported in 3.4% of VAFSEO and 3.3% of darbepoetin alfa patients. Consider this risk in patients at increased risk of GI erosion. Advise patients about signs of erosions and GI bleeding and urge them to seek prompt medical care if present. Serious Adverse Reactions in Patients with Anemia Due to CKD and Not on Dialysis The safety of VAFSEO has not been established for the treatment of anemia due to CKD in adults not on dialysis and its use is not recommended in this setting. In large clinical trials in adults with anemia of CKD who were not on dialysis, an increased risk of mortality, stroke, MI, serious acute kidney injury, serious hepatic injury, and serious GI erosions was observed in patients treated with VAFSEO compared to darbepoetin alfa. Malignancy VAFSEO has not been studied and is not recommended in patients with active malignancies. Malignancies were observed in 2.2% of VAFSEO and 3.0% of darbepoetin alfa patients. No evidence of increased carcinogenicity was observed in animal studies. ADVERSE REACTIONS The most common adverse reactions (occurring at ≥ 10%) were hypertension and diarrhea. DRUG INTERACTIONS Iron supplements and iron-containing phosphate binders: Administer VAFSEO at least 1 hour before products containing iron. Non-iron-containing phosphate binders: Administer VAFSEO at least 1 hour before or 2 hours after non-iron-containing phosphate binders. BCRP substrates: Monitor for signs of substrate adverse reactions and consider dose reduction. Statins: Monitor for statin-related adverse reactions. Limit the daily dose of simvastatin to 20 mg and rosuvastatin to 5 mg. USE IN SPECIFIC POPULATIONS Pregnancy: May cause fetal harm. Lactation: Breastfeeding not recommended until two days after the final dose. Hepatic Impairment : Not recommended in patients with cirrhosis or active, acute liver disease. Please note that this information is not comprehensive. Please click here for the Full Prescribing Information, including BOXED WARNING and Medication Guide. Forward-Looking Statements Statements in this press release regarding Akebia Therapeutics, Inc.'s ("Akebia's") strategy, plans, prospects, expectations, beliefs, intentions and goals are forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, as amended, and include, but are not limited to, statements regarding: Akebia's expectations as to the timing of the availability of Vafseo and the receipt and timing of the issuance by CMS of a Medicare Claims Processing Change Request that will give further billing guidance to dialysis organizations to obtain the separate TDAPA payment for the next two years under Medicare; Akebia's belief about the important benefit of TDAPA to support dialysis organizations' efforts to bring innovation to patients and incorporate new treatments into their practices; Akebia's progress in the Vafseo commercial launch, including that Akebia continues to actively engage dialysis organizations on the contracting process to facilitate access to Vafseo and further interact with nephrologists and healthcare providers at the American Society of Nephrology conference; and Akebia's plans to continue to build interest in Vafseo, including its goal to drive usage and demand. The terms "intend," "believe," "plan," "goal," "potential," "anticipate, "estimate," "expect," "future," "will," "continue," derivatives of these words, and similar references are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results, performance or experience may differ materially from those expressed or implied by any forward-looking statement as a result of various risks, uncertainties and other factors, including, but not limited to, risks associated with: whether Vafseo will be commercially available when expected; the potential demand and market potential and acceptance of, as well as coverage and reimbursement related to, Auryxia® and Vafseo, including estimates regarding the potential market opportunity; the competitive landscape for Auryxia and Vafseo, including potential generic entrants; the ability of Akebia to attract and retain qualified personnel; Akebia's ability to implement cost avoidance measures and reduce operating expenses; decisions made by health authorities, such as the FDA, with respect to regulatory filings; the potential therapeutic benefits, safety profile, and effectiveness of Vafseo; the results of preclinical and clinical research; the direct or indirect impact of the COVID-19 pandemic on the markets and communities in which Akebia and its partners, collaborators, vendors and customers operate; manufacturing, supply chain and quality matters and any recalls, write-downs, impairments or other related consequences or potential consequences; and early termination of any of Akebia's collaborations. Other risks and uncertainties include those identified under the heading "Risk Factors" in Akebia's Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, and other filings that Akebia may make with the U.S. Securities and Exchange Commission in the future. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release, and, except as required by law, Akebia does not undertake, and specifically disclaims, any obligation to update any forward-looking statements contained in this press release. Akebia Therapeutics® and Vafseo® are registered trademarks of Akebia Therapeutics, Inc. and its affiliates. Akebia Therapeutics Contact Mercedes Carrasco [email protected] SOURCE Akebia Therapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

作者｜momo 截止目前，FDA已经批准35款新药上市，其中多款药物具有里程碑意义。    01   2024年FDA已批准药物 1.1.Zelsuvmi (berdazimer) 适应症：局部传染性软疣 批准日期：2024年1月5日 企业：Novan Inc. 1.2.Exblifep (cefepime and enmetazobactam) 适应症：尿路感染 靶点：LACTB、PBP 批准日期：2024年2月22日 企业：Orchid Chemicals & Pharmaceuticals Ltd. 1.3.Letybo (letibotulinumtoxin a-WLBG) 适应症：中度至重度眉间纹的外观改善 靶点：BoNT/A 批准日期：2024年2月29日 企业：Hugel Inc. 1.4.Tevimbra (tislelizumab-JSGR) 适应症：不可切除、局部晚期或转移性食管鳞状细胞癌 靶点：PD-1 批准日期：2024年3月13日 企业：百济神州 1.5.Rezdiffra (resmetirom) 适应症：中晚期非肝硬化性非酒精性脂肪性肝炎 靶点：THR-β 批准日期：2024年3月14日 企业：罗氏 1.6.Tryvio 适应症：高血压 靶点：ET-A、ET-B 批准日期：2024年3月19日 企业：Idorsia. 1.7.Duvyzat 适应症：杜氏肌营养不良症 靶点：HDAC 批准日期：2024年3月21日 企业：Italfarmaco Spa. 1.8.Winrevair (sotatercept-CSRK) 适应症：肺动脉高压 靶点：INHBA 批准日期：2024年3月26日 企业：Acceleron Pharma Inc. 1.9.Vafseo 适应症：成人慢性肾脏疾病引起的贫血 靶点：HIF 批准日期：2024年3月27日 企业：宝洁 1.10.Voydeya 适应症：阵发性夜间血红蛋白尿治疗血管外溶血 靶点：CFD 批准日期：2024年3月29日 企业：Achillion Pharmaceuticals Inc. 1.11.Zevtera (ceftobiprole medocaril sodium) 适应症：血液感染、细菌性皮肤和相关组织感染、社区获得性细菌性肺炎 靶点：PBP 批准日期：2024年4月3日 企业：Roche 1.12.Lumisight 适应症：乳腺癌荧光成像 批准日期：2024年4月17日 企业：Lumicell Inc., 杜克大学 1.13.Anktiva (nogapendekin alfa inbakicept-pmln) 适应症：膀胱癌 靶点：IL-15R 批准日期：2024年4月22日 企业：Altor Bioscience Corp. 1.14.Ojemda (tovorafenib) 适应症：复发或难治性儿童低级别胶质瘤 靶点:RAF 批准日期：2024年4月23日 企业：Viracta Therapeutics Inc. 1.15.Xolremdi (mavorixafor) 适应症：治疗12岁及以上患有WHIM综合征的患者 靶点：CXCR4 批准日期：2024年4月26日 企业：赛诺菲 1.16.Imdelltra (tarlatamab-dlle) 适应症：广泛期小细胞肺癌 靶点：CD3E、DLL3 批准日期：2024年5月16日 企业：Amgen Inc. 1.17.Rytelo (imetelstat) 适应症：低危骨髓增生异常综合征伴输血依赖性贫血 靶点：Telomerase 批准日期：2024年6月6日 企业：Geron Corp. 1.18.Iqirvo (elafibranor) 适应症：原发性胆汁性胆管炎 靶点：PPARɑ/γ 批准日期：2024年6月10日 企业：Genfit SA 1.19.Sofdra (sofpironium) 适应症：原发性腋窝多汗症 靶点：AChR 批准日期：2024年6月18日 企业：Bodor Laboratories Inc. 1.20.Piasky (crovalimab-akkz) 适应症：阵发性睡眠性血红蛋白尿症 靶点：C5 批准日期：2024年6月20日 企业：Sino-Foreign Pharmaceutical Co., Ltd. 1.21.Ohtuvayre（Ensifentrine） 适应症：治疗慢性阻塞性肺病 靶点：PDE3/4 批准日期：2024年6月26日 企业：Verona Pharma. 1.22.Kisunla（Donanemab-azbt） 适应症：阿尔兹海默病 靶点：pGlu3Aβ 批准日期：2024年7月2日 企业：lilly 1.23.Leqselvi（Deuruxolitinib） 适应症：严重斑秃 靶点：JAK 批准日期：2024年7月25日 企业：Sun Pharmaceuticals 1.24.Voranigo（vorasidenib） 适应症：IDH1或IDH2易感突变的2级星形细胞瘤或少突胶质细胞瘤。 靶点：IDH1、IDH2 批准日期：2024年8月6日 企业：Servier Pharmaceuticals 1.25.Yorvipath（palopegteriparatide） 适应症：甲状旁腺功能亢进 批准日期：2024年8月9日 企业：ascendis Pharma. 1.26.Nemluvio（nemolizumab-ilto） 适应症：结节性痒疹 靶点:IL-31 批准日期：2024年8月12日 企业：Galderma Pharma. 1.27. Livdelzi（seladelpar） 适应症：原发性胆汁性胆管炎 靶点：PPAR-δ 批准日期：2024年8月14日 企业：Gilead Sciences 1.28.Niktimvo（axatilimab-csfr） 适应症：慢性移植物抗宿主病 靶点CSF-1R 批准日期：2024年8月14日 企业：Incyte/Syndax制药 1.29.Lazcluze（lazertinib） 适应症：非小细胞肺癌 靶点：EGFRm 批准日期：2024年8月19日 企业：强生 1.30.Ebglyss（lebrikizumab-lbkz） 适应症：中度至重度特应性皮炎 靶点：IL-13 批准日期：2024年9月13日 企业：lilly 1.31.Miplyffa（arimoclomol） 适应症：C型尼曼-匹克病 靶点：HSP 批准日期：2024年9月20日 企业：Zevra Therapeutics 1.32.Aqneursa （levacetylleucine） 适应症：儿童和成人C型尼曼匹克病 批准日期：2024年9月24日 企业：IntraBio. 1.33.Cobenfy（xanomeline and trospium chloride） 适应症：成人精神分裂症 靶点：M1-和M4-选择性毒蕈碱受体 批准日期：2024年9月26日 企业：百时美施贵宝 1.34.Flyrcado（flurpiridaz F 18） 适应症：评估心脏血流阻塞（心肌缺血）和心脏病发作（心肌梗塞） 批准日期：2024年9月27日 企业：GE HealthCare 1.35.Tevimbra（Tislelizumab） 适应症：不可切除或转移性食管鳞状细胞癌 靶点：PD-1 批准日期：2024年10月4日 企业：百济神州   02   2024年FDA批准的突出药物 Voranigo（vorasidenib）这种双重IDH1/IDH2抑制剂处理导致癌细胞代谢变化的突变。它被批准用于2级星形细胞瘤或少突胶质细胞瘤，这是一种生长缓慢的脑肿瘤，具有显著的风险。这是一个突破，因为它直接针对驱动肿瘤生长的突变，提供了一种更有效的治疗选择，可以延缓疾病进展，减少对辐射等侵入性治疗的依赖。 Niktimvo（Axatilimab）是一种CSF-1R单克隆抗体，通过限制巨噬细胞活性来治疗cGVHD，巨噬细胞在免疫应答中起着至关重要的作用。这种疗法对患有cGVHD的患者至关重要，这是干细胞移植后的一种严重并发症，通常对现有治疗无效。它提供了一种更有针对性的策略来改善患者的生活质量。 Cobenfy作为Xanomeline和Trospium Chloride这种联合治疗方法，通过调节毒蕈碱受体，为治疗精神分裂症带来了新的途径。通过平衡大脑中的神经递质活动，它解决了这种疾病的阳性和阴性症状，这使得它对那些用其他药物都没有成功的患者特别有价值。它的双重机制提供了更全面的症状管理，改善了那些受这种具有挑战性的精神健康状况影响的结果。 Rezdiffra（resmetirom）是一款甲状腺激素受体（THR）-β口服选择性激动剂，THR-β在肝脏中高度表达，能够调节脂代谢，降低LDL-C、甘油三酯和致动脉粥样硬化性脂蛋白。此外，THR-β还能通过促进脂肪酸的分解和刺激线粒体的生物发生来减少脂肪毒性并改善肝功能，从而减少肝脏脂肪。Rezdiffra用于治疗患有中重度肝纤维化（F2至F3期）的非肝硬化非酒精性脂肪性肝炎（NASH）患者，成为FDA批准的首款NASH疗法，具有里程碑意义。 Anktiva是一款IL-15超级激动剂。IL-15通过影响自然杀伤（NK）细胞和免疫T细胞的发育、维持和功能，在免疫系统中发挥着至关重要的作用。Anktiva由与IL-15受体α/IgG1 Fc融合蛋白结合的IL-15突变体（IL-15N72D）组成。它与βγ T细胞受体结合，直接特异性刺激CD8阳性T细胞和NK细胞，同时避免刺激调节性T细胞（Treg）。与天然、非复合IL-15相比，Anktiva在患者体内具有更优的药代动力学特性，且能在淋巴组织中存在更长时间，表现出增强的抗肿瘤活性。Anktiva在2019年获FDA授予突破性疗法认定，用于与BCG联合治疗此前对BCG应答不佳的非肌层浸润性原位膀胱癌患者。 参考资料 [1]Novel Drug Approvals for 2024 | FDA [2]百济神州官网 BiG近期会议 10月26日，第三届生物计算大会保留栏目-第二届吐槽大会即将再度来袭！本届吐槽大会将围绕投融资、行业心态、年轻人，及AI等热门话题展开策划，通过移幕换景，大咖带新秀，让更多80-00后的行业亲历者来到聚光灯下，邀与会嘉宾一同探讨AI+Biomed的融合之道！ 共建Biomedical创新生态圈！ 如何加入BiG会员？