Aprocitentan

今天分享的是：药渡经纬：2026年药渡全球药物研发进展报告-创新药篇报告共计：61页2026年1月3日-9日全球及国内创新药研发进展总结2026年1月3日至9日，全球与国内创新药领域成果丰硕，在药物批准、临床研发、企业合作及政策优化等方面均有重要突破，为疾病治疗带来新可能。全球层面，新药批准与研发动态亮点突出。统计周期内，全球有4条新药批准状态更新，含NDA批准2个、BLA批准1个及新制剂批准1个。Idorsia的Aprocitentan获加拿大卫生部批准用于成人难治性高血压，葛兰素史克的超长效生物制剂Exdensur在日本获批，用于治疗重度或难治性支气管哮喘等疾病，一年仅需注射两次即可持续抑制2型炎症。全球新药临床研发状态更新达60条，覆盖17个领域，歌礼制药的口服小分子GLP-1 ASC30获FDA批准开展II期研究，信达生物的VEGF/Ang2双特异性抗体IBI324在相关眼病治疗中展现出优异疗效。同时，全球医药交易活跃，多类合作与许可协议达成，涉及肿瘤、代谢疾病等多个治疗领域。国内方面，创新药发展势头强劲。新药批准状态更新15条，涵盖NDA、BLA、新复方、新适应症及新制剂等类型。恒瑞医药的全球首款PD-L1/TGF-βRII双抗瑞拉芙普α注射液获批上市，用于特定胃癌治疗；赛诺菲的瑞达普®获批，破解了家族性乳糜微粒血症综合征在中国“无药可控”的困局。国内47个新药获临床默示许可，16个新药申报上市，74个新药申报临床，涉及化药、生物制品等多个品类。华奥泰的Sotiburafusp alfa启动III期临床，复宏汉霖的HLX-43公布III期临床试验方案设计。政策层面，国家药监局发布多项公告，优化临床急需境外已上市药品审评审批机制，缩短注册检验时限，完善注册核查方式，推动药品注册技术标准与国际接轨。此外，国内外企业合作频繁，宜联生物与罗氏再度联手加速B7H3 ADC全球开发，国内企业间也在靶向药物等领域积极开展合作，为创新药的研发与商业化注入动力。整体来看，该周期内创新药领域在多疾病赛道实现技术突破，政策支持与企业协作共同推动行业持续发展，为患者带来更多治疗选择。以下为报告节选内容返回搜狐，查看更多

Idorsia receives approval from Health Canada for JERAYGO ™ (aprocitentan) as the first and only endothelin receptor antagonist (ERA) for the treatment of resistant hypertension. JERAYGO is a new oral antihypertensive therapy – the first systemic hypertension treatment to target a new pathway in over 30 years. Allschwil, Switzerland – January 5, 2026 Idorsia Ltd (SIX: IDIA) announces that Health Canada has granted marketing authorization for JERAYGO ™ (aprocitentan) for the treatment of resistant hypertension in adult patients in combination with at least three antihypertensive medicinal products. 1 The recommended dose is 12.5 mg orally once daily. The dose can be increased to 25 mg once daily for patients tolerating the 12.5 mg dose and in need of tighter blood pressure (BP) control. 1 Hypertension remains a leading global health challenge and the number one modifiable risk factor for early morbidity and mortality. Despite advances in treatment, many patients still struggle with uncontrolled blood pressure, leaving them at significantly higher risk of heart attack, stroke, kidney failure, and premature death. Approximately 10% of hypertensive patients have resistant hypertension 3 , 4 – defined by uncontrolled blood pressure despite receiving at least three antihypertensive medications from different classes, at optimal doses – underscoring the urgent need for more effective therapies. Srishti Gupta, MD, Chief Executive Officer of Idorsia, commented: “JERAYGO is the first and only hypertension treatment to target the endothelin pathway, a fundamental yet previously unaddressed driver of disease onset, progression, and complications. The results of PRECISION show that targeting the endothelin pathway is crucial to adequately manage uncontrolled blood pressure. JERAYGO has demonstrated rapid, durable, and clinically significant double-digit blood pressure reduction across a broad spectrum of challenging patient populations, including those with obesity, chronic kidney disease, or type 2 diabetes. I am very proud of our team for achieving this milestone.” Idorsia is engaged in discussions to maximize the ability to make JERAYGO available to patients. For more information on the marketing authorization of JERAYGO in Canada and important safety information, please review the Product Monograph . Notes to the editor About the Phase 3 PRECISION study 1,5 The efficacy of aprocitentan was evaluated in one randomized, double-blind (DB), placebo-controlled Phase 3 multicenter study. Patients with uncontrolled blood pressure (systolic blood pressure [SBP] ≥140 mmHg) despite the use of at least three antihypertensive medicinal products and following exclusion of pseudo-resistant hypertension (e.g., white coat effect, inappropriate blood pressure measurement, secondary causes of hypertension) were considered to have resistant hypertension. The patients were switched to standardized background antihypertensive therapy consisting of an angiotensin receptor blocker (valsartan 160 mg), a calcium channel blocker (amlodipine 5 or 10 mg), and a diuretic (hydrochlorothiazide 25 mg) throughout the study. Patients with concomitant use of beta-blockers continued this treatment throughout the study, in addition to the standardized background antihypertensive therapy and study treatment. A total of 730 patients received either aprocitentan 12.5 mg, aprocitentan 25 mg, or placebo once daily during the initial 4-week DB treatment (part 1). Thereafter, patients received aprocitentan 25 mg once daily during the 32-week single-blind treatment (part 2). At the end of the 32 weeks, patients were re-randomized to receive either aprocitentan 25 mg or placebo, once daily, during the 12-week double-blind withdrawal (DB-WD) treatment (part 3). The primary efficacy endpoint was the change in sitting SBP (SiSBP) from baseline to Week 4 during DB treatment (part 1), measured at trough by unattended automated office blood pressure (uAOBP). The key secondary endpoint was the change in SiSBP measured at trough by uAOBP from DB-WD baseline (Week 36) to Week 40 (part 3). Patients had a mean age of 61.7 years (range 24 to 84 years; 34.1% were ≥ 65 and < 75 years; 9.9% were ≥ 75 years) and 59.5% were male. Patients were White (82.9%), African American (11.2%) or Asian (5.2%). The mean body weight was 97.6 kg (range 46 to 196 kg) and mean BMI was 33.7 kg/m2 (range 18 to 64 kg/m2). Patients had a medical history of type 2 diabetes mellitus (54.1%), ischemic heart disease (30.8%), central nervous system vascular disorders (23.0%), chronic kidney disease stages 3 and 4 (22.2%; 19.3% of patients had eGFR 30–59 mL/min/1.73 m2 and 2.9% had eGFR 15–29 mL/min/1.73 m2), congestive heart failure (19.6%), and sleep apnea syndrome (14.1%). 63.0% of patients had four or more antihypertensive medicinal products. Key PRECISION findings 1,5 Doses of aprocitentan 12.5 and 25 mg showed a statistically significant reduction vs placebo on SiSBP at Week 4. The treatment effect was consistent for sitting diastolic blood pressure (SiDBP). The persistence of the BP-lowering effect of aprocitentan was shown in DB-WD treatment (part 3). In patients re-randomized to placebo, the mean SiSBP increased, whereas in patients re-randomized to aprocitentan 25 mg the mean effect on SiSBP was stable, resulting in a statistically significant difference. The treatment effect was consistent for SiDBP. The effect was also consistent across SBP and DBP measured by ambulatory BP monitoring (ABPM) and assessed as daytime, night-time, and 24h periods at Week 4 and Week 40. A substantial proportion (i.e., at least 90%) of the BP-lowering effect was observed within the first two weeks of treatment with aprocitentan. The effect of aprocitentan was consistent across subgroups of age (including patients ≥ 75 years), sex, race (including patients with Black or African American origin), BMI, baseline urine albumin-to-creatinine ratio (UACR), baseline eGFR and medical history of diabetes. The most frequently reported adverse reactions with aprocitentan were edema/fluid retention (mostly peripheral edema) (9.1%, 12.5 mg; 18.4%, 25 mg) and hemoglobin decreased (3.7%, 12.5 mg; 1.2%, 25 mg). JERAYGO is contraindicated for use in women who are pregnant, breast-feeding, in women of childbearing potential who are not using reliable contraception, patients with severe hepatic impairment, and in patients with hypersensitivity to the active substance or to any of the excipients. About aprocitentan Aprocitentan is Idorsia’s once-daily, orally active, dual endothelin receptor antagonist, which inhibits the binding of ET-1 to ET A and ET B receptors. Aprocitentan is approved as TRYVIO™ in the US for the treatment of systemic hypertension in combination with other antihypertensives and has been commercially available since October 2024. Aprocitentan is approved as JERAYGO™ for the treatment of resistant hypertension in combination with at least three antihypertensives in the European Union, the UK, and Switzerland, and now in Canada. Idorsia Pharmaceuticals Ltd has transferred its rights for aprocitentan (including JERAYGO™) to Idorsia Investments SARL to allow the repayment of notes issued in connection with the repurchase offer completed in August 2025. More details on the transfer can be found in the press release issued on May 21, 2025 and on the exchange offer in the press release issued on August 27, 2025. References JERAYGO ™ Product Monograph NCD Risk Factor Collaboration (NCD-RisC). Worldwide trends in hypertension prevalence and progress in treatment and control from 1990 to 2019: a pooled analysis of 1201 population-representative studies with 104 million participants. Lancet 2021; 398:957-80. Noubiap JJ, et al. Global prevalence of resistant hypertension: a meta-analysis of data from 3·2 million patients. Heart 2019; 105: 98–105. Williams B, et al. 2018 ESC/ESH guidelines for the management of arterial hypertension. Eur Heart J 2018; 39: 3021–104. Schlaich MP, et al. A randomized controlled trial of the dual endothelin antagonist aprocitentan for resistant hypertension. The Lancet, 2022; Dec 3;400(10367):1927-1937. About Idorsia The purpose of Idorsia is to challenge accepted medical paradigms, answering the questions that matter most. To achieve this, we will discover, develop, and commercialize transformative medicines – either with in-house capabilities or together with partners – and evolve Idorsia into a leading biopharmaceutical company, with a strong scientific core. Headquartered near Basel, Switzerland – a European biotech hub – Idorsia has a highly experienced team of dedicated professionals, covering all disciplines from bench to bedside; QUVIVIQ™ (daridorexant), a different kind of insomnia treatment with the potential to revolutionize this mounting public health concern; strong partners to maximize the value of our portfolio; a promising in-house development pipeline; and a specialized drug discovery engine focused on small-molecule drugs that can change the treatment paradigm for many patients. Idorsia is listed on the SIX Swiss Exchange (ticker symbol: IDIA). For further information, please contact: Investor & Media Relations Idorsia Pharmaceuticals Ltd, Hegenheimermattweg 91, CH-4123 Allschwil +41 58 844 10 10 investor.relations@idorsia.com – media.relations@idorsia.com – The above information contains certain "forward-looking statements", relating to the company's business, which can be identified by the use of forward-looking terminology such as “intend”, "estimates", "believes", "expects", "may", "are expected to", "will", "will continue", "should", "would be", "seeks", "pending" or "anticipates" or similar expressions, or by discussions of strategy, plans or intentions. Such statements include descriptions of the company's investment and research and development programs, business development activities and anticipated expenditures in connection therewith, descriptions of new products expected to be introduced by the company and anticipated customer demand for such products and products in the company's existing portfolio. Such statements reflect the current views of the company with respect to future events and are subject to certain risks, uncertainties and assumptions. Many factors could cause the actual results, performance or achievements of the company to be materially different from any future results, performances or achievements that may be expressed or implied by such forward-looking statements. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described herein as anticipated, believed, estimated or expected. Attachment Press Release PDF