Tenapanor Hydrochloride

据每日经济新闻报道，2024年国家药监局批准上市创新药48个、创新医疗器械65个，在研新药数量跃居全球第二位，多款国产创新药在全球上市。即将到来的2025年，还有一批重磅药物有望在国内获批。新浪医药结合既往报道，梳理出了2025年有望在国内获批上市的10款创新药。 药物名称：埃万妥单抗 适应症：非小细胞肺癌 研发企业：强生 埃万妥单抗是由强生和Genmab开发的一款first-in-class的c-Met/EGFR双特异性抗体。最早于2021年5月经FDA加速批准用于治疗正在接受或接受过含铂化疗后仍有疾病进展的EGFR Exon 20突变的非小细胞肺癌成年患者。2024年3月FDA批准其联合化疗用于EGFR Exon 20突变的NSCLC患者的一线治疗，这是FDA批准用于一线治疗此类患者的首款疗法，并获得NCCN指南一类推荐。 中国进度方面，强生于2023年10月首次递交埃万妥单抗的上市申请；2024年1月向CDE同时递交兰泽替尼和埃万妥单抗的上市申请，推测申报适应症为二者联合治疗EGFR突变局部晚期或转移性NSCLC患者。III期临床MARIPOSA研究结果显示，对于EGFR突变患者，埃万妥单抗联合兰泽替尼与疗法相较于奥希替尼，可将疾病死亡风险降低30%，并将患者的中位无进展生存期（mPFS）延长7个月。此外，强生于2024年9月向CDE递交埃万妥单抗皮下注射剂的上市申请，目前处于排队待审状态。预计2025Q1埃万妥单抗有望在中国获批。 塔奎妥单抗（talquetamab，Talvey） 预计批准时间：2025Q2 研发企业：强生 塔奎妥单抗是一款CD3/GPRC5D双特异性抗体， 也是首个针对GPRC5D靶点获批的药物。GPRC5D属于G蛋白偶联受体，多表达于多发性骨髓瘤，在正常组织的表达仅限于皮肤（毛囊和小汗腺）和睾丸（输精小管），且其表达水平与靶点BCMA相对独立。 2023年8月，FDA基于II期MonumenTAL-1研究结果加速批准塔奎妥单抗用于治疗患有复发性或难治性多发性骨髓瘤的成年患者，这些患者之前至少接受过4种疗法，包括蛋白酶体抑制剂、免疫调节剂和抗 CD38 单克隆抗体，结果显示，每两周皮下注射给药0.8 mg/kg时，ORR达到73.6%。 2024年欧洲血液学会EHA会议上，首次披露了塔奎妥单抗在中国人群中的有效性数据，结果显示，0.4 mg/kg QW、0.8 mg/kg Q2W队列的ORR分别是69.0% 和66.7%，VGPR率均大于58%，首次缓解的中位时间为1.3个月；安全性方面，与味觉、皮肤和指甲相关的 AE 主要为 1/2 级，且味觉相关的不良事件率为（25.0–41.1%），低于全球（71.0–72.0%）。 2024年2月，塔奎妥单抗在中国首次申请上市并纳入优先审评，用于单药治疗既往接受过至少三种治疗（包括一种蛋白酶体抑制剂、一种免疫调节剂和一种抗CD38抗体）的复发或难治性多发性骨髓瘤（R/R MM）成人患者，预计2025年Q2有望在中国上市。 药物名称：利生奇珠单抗 适应症：克罗恩病等 研发企业：勃林格殷格翰 2019年，利生奇珠单抗首次进入全球市场，目前已获批斑块状银屑病、银屑病关节炎、克罗恩病等多项适应症。2023年7月，利生奇珠单抗首次在华申报上市，推测申报适应症为克罗恩病。2024年6月，利生奇珠单抗申报的新适应症上市申请获受理，推测申报适应症为溃疡性结肠炎。 利生奇珠单抗针对斑块状银屑病、克罗恩病，已开展多项头对头注册性临床试验。2024年7月，NEJM发表了利生奇珠单抗vs乌司奴单抗治疗克罗恩病的临床结果，针对既往接受过生物制品治疗的克罗恩病患者，临床缓解率分别是：58.6% vs 39.5%，内镜缓解率分别是：31.8% vs 16.2%，利生奇珠单抗均取得优效性。 值得注意的是，与在国内已获批的anti-IL-23单抗不同，利生奇珠单抗在国内开展的适应症均为炎症性肠病，包括克罗恩病、溃疡性结肠炎。 药物名称：玛仕度肽 适应症：体重控制、2型糖尿病 研发企业：信达生物、礼来 玛仕度肽是信达生物与礼来开发的一款胰高血糖素样肽-1受体（GLP-1R）/胰高血糖素受体（GCGR）双重激动剂。玛仕度肽除了可通过激动GLP-1R促进胰岛素分泌、降低血糖和减轻体重外，还可通过激动GCGR增加能量消耗而增强减重疗效，同时改善肝脏脂肪代谢。 玛仕度肽在中国超重或肥胖成人受试者中的首个III期临床GLORY-1研究达成主要终点和所有关键次要终点。玛仕度肽6mg组受试者治疗32周后体重相对基线的百分比变化显著优于安慰剂组（-13.38% vs -0.24%）。 2024年7月，信达生物宣布玛仕度肽在中国2型糖尿病受试者中开展的III期临床研究（DREAMS-1）达到主要终点和全部关键次要终点，展现出降糖、减重双达标及心血管肾脏代谢指标的综合获益。 信达生物于2024年2月向NMPA提交玛仕度肽用于成人肥胖或超重患者的长期体重控制的上市申请，且于2024年8月提交第二个适应症2型糖尿病的上市申请，目前两项适应症的上市申请均处在审评阶段，预计可于2025Q2获批。 药物名称：索乐匹尼布 适应症：原发性免疫性血小板减少症 研发企业：和黄医药 索乐匹尼布是由和黄医药自主研发的一款口服选择性Syk抑制剂，在研适应症包括免疫性血小板减少症（ITP）、温抗体型自身免疫溶血性贫血（WAIHA）等。2022年1月，索乐匹尼布获得突破性疗法认定，用于治疗原发性免疫性血小板减少症。目前国内尚无Syk抑制剂获上市，和黄医药于2024年1月提交了索乐匹尼布的上市申请，用于治疗原发免疫性血小板减少症，有望成为中国首款上市的Syk抑制剂。 药物名称：替那帕诺 适应症：慢性肾病血透患者高磷血症 研发企业：Ardelyx 替那帕诺是由Ardelyx原研的唯一一款在全球获批上市的NHE3小分子抑制剂。2023年FDA批准了替那帕诺作为附加治疗，用于降低成年慢性肾病患者的血磷水平。此外，FDA还批准了替那帕诺用于那些对磷结合剂反应不佳或不耐受的成年慢性肾病患者。早在2017年，复星医药以1.25亿美元的价格，从Ardelyx手中获得了替那帕诺在中国内地、香港及澳门特别行政区的独家临床开发和商业化权益。2023年7月，复星医药向国家药监局递交替那帕诺用于治疗慢性肾病血透患者高磷血症的上市申请。 药物名称：罗莫索珠单抗 适应症：骨质疏松症 研发企业：UCB、安进 罗莫索珠单抗是UCB和Amgen共同开发的anti-SOST单抗，通过特异性抑制骨硬化蛋白，提高骨形成并降低骨吸收从而降低骨折风险。 目前仅有罗莫索珠单抗1款作用于SOST的药物获批上市，于2019年在日本、美国和欧洲获批，用于治疗具有高骨折风险的骨质疏松症人群（包括绝经后妇女），但由于可能带来的心血管风险，FDA对罗莫索珠单抗给予黑框警告。2023年，罗莫索珠单抗全球销售额达11.6亿美元。 在中国，UCB和Amgen已完成一项3期临床研究，以评价罗莫索珠单抗在中国绝经后骨质疏松症女性中的疗效、安全性和耐受性。同机制的药品中，罗莫索珠单抗是国内进展最快的，于2024年7月首次向NMPA递交治疗骨质疏松症的上市申请，目前处在排队待审评阶段，预计于2025Q4获批。 药物名称：泽尼达妥单抗 适应症：胆道癌 研发企业：Zymeworks 泽尼达妥单抗是由Zymeworks研发的HER2靶向双特异性抗体，能够同时结合HER2受体的两个非重叠表位，可以引起双重HER2信号阻断。2018年，Zymeworks与百济神州达成交易，授予百济神州在亚洲（除日本外）、澳大利亚和新西兰的开发和商业化权益。 2024 年 6 月 7 日，百济神州的 HER2 双抗泽尼达妥单抗上市申请获受理，用于既往接受过全身治疗的 HER2 高表达的不可切除局部晚期或转移性胆道癌患者，该适应症已被纳入优先审评，预计 2025 年 Q2 获批。 此次申请基于关键Ⅱ 期临床 HERIZON-BTC-01 的结果，泽尼达妥单抗在难治性 HER2 阳性的 BTC 患者中显示出快速、持久的缓解，并且安全性可控。 药物名称：伊那利塞 适应症：乳腺癌 研发企业：罗氏 伊那利塞是罗氏开发的一款口服高选择性PI3Kα抑制剂，针对PIK3CA突变、HR阳性乳腺癌。PIK3CA突变在乳腺癌中非常普遍，发生在约40%的HR阳性肿瘤中，这款产品的突出优势在于高选择性，它对PI3Kα的选择性超过其他亚型（PI3K β/δ/γ）300倍，超过PIK家族成员2000倍。目前伊那利塞在中国同赛道排名第一，全球同赛道排名中位于第二，仅次于诺华的阿吡利塞。 今年5月，FDA授予伊那利塞突破性疗法认定，与哌柏西利和氟维司群联合用于一线治疗PIK3CA突变的HR+/HER2-局部晚期或转移性乳腺癌成人患者。目前伊那利塞是中国研发进度最快的选择性PI3Kα抑制剂，于今年6月向CDE提交上市申请，并被纳入优先审评目录，有望2025Q3在中国获批。 替利珠单抗（teplizumab，Tzield） 预计批准时间：2025Q4 研发企业：赛诺菲 替利珠单抗是全球首个且唯一一个可用于自身免疫性1型糖尿病的疾病修饰疗法，为1型糖尿病的预防和治疗提供了新的希望。替利珠单抗是一种不结合Fc受体的靶向CD3的单抗，通过与效应T细胞表面的CD3结合，抑制其对胰岛β细胞的攻击。替利珠单抗的Fc段经氨基酸修饰后可减少其与补体和Fc受体的结合，从而降低相关毒性反应。 礼来于2007年以4100万美元首付款从MacroGenics获得了替利珠单抗的独家权益，但在2010年因III期试验未达主要终点，礼来放弃进一步探索。2018年，Provention Bio从MacroGenics手中收购了替利珠单抗。1年后，替利珠单抗取得里程碑式进展并被FDA授予突破性疗法资格。2020年，Provention Bio启动替利珠单抗的滚动BLA，并于同年10月与赛诺菲达成合作，为替利珠单抗的商业化铺路。 替利珠单抗于2022年11月在美国获批用于成人和8岁及以上儿童1型糖尿病2期患者，以延缓3期1型糖尿病发病，成为全球首个且唯一一个获FDA批准用于延缓1型糖尿病发病的免疫疗法。2023年，赛诺菲以29亿美元收购Provention Bio公司以获得替利珠单抗资产。 Provention Bio于2024年8月向NMPA提交替利珠单抗用于1型糖尿病的上市申请（JXSS2400070），并被纳入优先审评审批程序，或将成为我国首个1型糖尿病延缓发病治疗药物，预计2025Q4获批。 本文编辑：张杰 参考： https://www.msn.cn/zh-cn/news/other/53%E6%AC%BE-2025%E5%B9%B4%E6%9C%89%E6%9C%9B%E8%8E%B7%E6%89%B9%E7%9A%84%E5%9B%BD%E4%BA%A7%E6%96%B0%E8%8D%AF/ar-AA1x1MjQ?ocid=BingNewsSerp https://mp.weixin.qq.com/s?__biz=MzAxNjI5NzI4MA==&mid=2247562950&idx=6&sn=268cc52503afd69f79138f3c3687d01c&chksm=9a0c2e7e867eca45c66a0022b180acd07edad79c551c36b35be77e6c80a7db8384a15d75cc65&scene=27 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Company achieved significant commercial progress in 2024, finishing with total U.S. net product sales revenue of approximately $319 million (unaudited) Company reaffirms peak U.S. net IBSRELA sales revenue of greater than $1 Billion Company announces peak U.S. net XPHOZAH sales revenue of $750 million Company finishes 2024 with approximately $250 million in cash, cash equivalents and investments (unaudited) WALTHAM, Mass., Jan. 13, 2025 (GLOBE NEWSWIRE) -- Ardelyx, Inc. (Nasdaq: ARDX), a biopharmaceutical company founded with a mission to discover, develop and commercialize innovative, first-in-class medicines that meet significant unmet medical needs, today provided an update on the company’s progress in 2024 and initial expectations for 2025. “In 2024, Ardelyx delivered another year of sustained growth and exceptional performance. The growing demand for IBSRELA and XPHOZAH, both important, first-in-class medicines, has demonstrated that there is a clear unmet need among patients for differentiated therapeutic options,” said Mike Raab, president and chief executive officer. “It is an incredibly exciting time for our company with tremendous opportunities in front of us. As you saw in the performance during the fourth quarter, the team is delivering on the promise of IBSRELA. This performance reaffirms our expectation that IBSRELA can achieve greater than $1 billion in annual sales at peak. In 2024, XPHOZAH’s performance demonstrated its critical role in the treatment of hyperphosphatemia where there continues to be a high unmet need among patients. It is evident that there is a significant opportunity for XPHOZAH and we expect this product will achieve $750 million in annual sales at peak.” Raab continued, “In 2025, we will be focused on our strategic priorities: Maintaining our growth momentum, building a pipeline and delivering a strong financial performance. We remain steadfast in our commitment to operational excellence and to making decisions in the best interest of patients and our shareholders.” IBSRELA® (tenapanor) records approximately $158 million in net product sales revenue in 2024U.S. net product sales revenue for IBSRELA during the fourth quarter is expected to be approximately $54 million, with full year net product sales revenue totaling approximately $158 million, subject to adjustment in connection with preparation of audited financial statements. IBSRELA performance demonstrated consistent quarter-over-quarter growth throughout the year, accelerating in the fourth quarter following the completion of the company’s field-based sales team expansion. In 2025, the company will focus on continued commercial execution to maintain the strong momentum for IBSRELA. Ardelyx currently expects full-year 2025 U.S. net product sales revenue for IBSRELA to be between $240.0 and $250.0 million. Ardelyx continues to expect IBSRELA to achieve greater than ten percent market share at peak and generate more than $1.0 billion in annual U.S. net product sales revenue before patent term expiration. Strong XPHOZAH® (tenapanor) performance continues, recording approximately $161 million net product sales revenue during 2024XPHOZAH demonstrated an exceptional launch and finished its first full year of commercialization with 2024 U.S. net product sales revenue of approximately $161 million, including approximately $57 million in net product sales during the fourth quarter, subject to adjustment in connection with preparation of audited financial statements. The strong response from the prescribing community to XPHOZAH continued throughout the year, with feedback reflecting positive patient experiences and outcomes. XPHOZAH saw continued, quarter-over-quarter growth across all key indicators. At peak, Ardelyx currently expects XPHOZAH to achieve $750 million in annual U.S. net product sales revenue before patent term expiration. Coverage for oral only therapies, including XPHOZAH, is no longer available under Medicare Part D as of January 1, 2025. Access to XPHOZAH remains through a prescription written by a qualifying healthcare provider sent to Transition Pharmacy, the ArdelyxAssist specialty pharmacy partner. Patients who do not have affordable access will be evaluated for eligibility to receive XPHOZAH through the Ardelyx patient assistance program. Strong Cash PositionAs of December 31, 2024, the company had total cash, cash equivalents and short-term investments of approximately $250 million (unaudited). Ardelyx had approximately 238 million shares outstanding as of December 31, 2024. Company Presentation at 42nd Annual J.P. Morgan Healthcare ConferenceThe company will present and provide a business update at the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15, 2025 at 7:30pm ET / 4:30pm PT. A webcast of the presentation can be accessed by visiting the Investor page of the company's website, www.ardelyx.com, and will be available on the website for at least 30 days following the call. IMPORTANT SAFETY INFORMATION (IBSRELA) WARNING: RISK OF SERIOUS DEHYDRATION IN PEDIATRIC PATIENTSIBSRELA is contraindicated in patients less than 6 years of age; in nonclinical studies in young juvenile rats administration of tenapanor caused deaths presumed to be due to dehydration. Avoid use of IBSRELA in patients 6 years to less than 12 years of age. The safety and effectiveness of IBSRELA have not been established in patients less than 18 years of age. CONTRAINDICATIONS IBSRELA is contraindicated in patients less than 6 years of age due to the risk of serious dehydration.IBSRELA is contraindicated in patients with known or suspected mechanical gastrointestinal obstruction. WARNINGS AND PRECAUTIONSRisk of Serious Dehydration in Pediatric Patients IBSRELA is contraindicated in patients below 6 years of age. The safety and effectiveness of IBSRELA in patients less than 18 years of age have not been established. In young juvenile rats (less than 1 week old; approximate human age equivalent of less than 2 years of age), decreased body weight and deaths occurred, presumed to be due to dehydration, following oral administration of tenapanor. There are no data available in older juvenile rats (human age equivalent 2 years to less than 12 years).Avoid the use of IBSRELA in patients 6 years to less than 12 years of age. Although there are no data in older juvenile rats, given the deaths in younger rats and the lack of clinical safety and efficacy data in pediatric patients, avoid the use of IBSRELA in patients 6 years to less than 12 years of age. Diarrhea Diarrhea was the most common adverse reaction in two randomized, double-blind, placebo-controlled trials of IBS-C. Severe diarrhea was reported in 2.5% of IBSRELA-treated patients. If severe diarrhea occurs, suspend dosing and rehydrate patient. MOST COMMON ADVERSE REACTIONS The most common adverse reactions in IBSRELA-treated patients (incidence ≥2% and greater than placebo) were: diarrhea (16% vs 4% placebo), abdominal distension (3% vs <1%), flatulence (3% vs 1%) and dizziness (2% vs <1%). INDICATION IBSRELA (tenapanor) is indicated for the treatment of Irritable Bowel Syndrome with Constipation (IBS-C) in adults. Please see full Prescribing Information, including Boxed Warning, for additional risk information. IMPORTANT SAFETY INFORMATION (XPHOZAH) CONTRAINDICATIONSXPHOZAH is contraindicated in: Pediatric patients under 6 years of agePatients with known or suspected mechanical gastrointestinal obstruction WARNINGS AND PRECAUTIONSDiarrhea Patients may experience severe diarrhea. Treatment with XPHOZAH should be discontinued in patients who develop severe diarrhea. MOST COMMON ADVERSE REACTIONS Diarrhea, which occurred in 43-53% of patients, was the only adverse reaction reported in at least 5% of XPHOZAH-treated patients with CKD on dialysis across trials. The majority of diarrhea events in the XPHOZAH-treated patients were reported to be mild-to-moderate in severity and resolved over time, or with dose reduction. Diarrhea was typically reported soon after initiation but could occur at any time during treatment with XPHOZAH. Severe diarrhea was reported in 5% of XPHOZAH-treated patients in these trials. INDICATION XPHOZAH (tenapanor), 30 mg BID, is indicated to reduce serum phosphorus in adults with chronic kidney disease (CKD) on dialysis as add-on therapy in patients who have an inadequate response to phosphate binders or who are intolerant of any dose of phosphate binder therapy. For additional safety information, please see full Prescribing Information. About Ardelyx, Inc.Ardelyx was founded with a mission to discover, develop and commercialize innovative, first-in-class medicines that meet significant unmet medical needs. Ardelyx has two commercial products approved in the United States, IBSRELA® (tenapanor) and XPHOZAH® (tenapanor). Ardelyx has agreements for the development and commercialization of tenapanor outside of the U.S. Kyowa Kirin commercializes PHOZEVEL® (tenapanor) for hyperphosphatemia in Japan. A New Drug Application for tenapanor for hyperphosphatemia has been submitted in China with Fosun Pharma. Knight Therapeutics commercializes IBSRELA in Canada. For more information, please visit https://ardelyx.com/ and connect with us on X (formerly known as Twitter), LinkedIn and Facebook. Forward Looking StatementsTo the extent that statements contained in this press release are not descriptions of historical facts regarding Ardelyx, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor of the Private Securities Reform Act of 1995, including Ardelyx’s currents expectations regarding: opportunities for continued adoption of IBSRELA and XPHOZAH, the annual U.S. net product sales revenue for IBSRELA at peak; the annual U.S. net product sales revenue for XPHOZAH at peak; net product sales revenue for IBSRELA for full year 2024 and the fourth quarter ended December 31, 2024; net product sales revenue for IBSRELA for full year 2025; the potential market share for IBSRELA at peak; net product sales revenue for XPHOZAH for the full year 2024 and the fourth quarter ended December 31, 2024; and its cash position at December 31, 2024. Such forward-looking statements involve substantial risks and uncertainties that could cause Ardelyx's future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, uncertainties associated with the development of, regulatory process for, and commercialization of drugs in the U.S. and internationally. Ardelyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ardelyx's business in general, please refer to Ardelyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on October 31, 2024, and its future current and periodic reports to be filed with the Securities and Exchange Commission. Investor and Media Contacts: Caitlin Lowieclowie@ardelyx.com