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To date, Novo Nordisk’s focus in rare disease has hinged on blood and endocrine disorders, with its efforts yielding a number of approved treatments. While Novo Nordisk has been operating in rare disease for some 45 years, the field has often taken a back seat to the Danish drugmaker’s bread-and-butter business developing treatments for diabetes and other common chronic diseases.Still, Novo is steadfast in its goal to eventually become a leader in the space, the company’s EVP of rare disease, Ludovic Helfgott, said in an interview with Fierce Pharma following the company’s 2025 international press briefing on Thursday. Overall, the near-term success of those ambitions will come down to a handful of new drug launches and a sickle cell disease (SCD) candidate that recently entered phase 3 testing.Plus, Novo will continue to seek external opportunities to refine its rare disease pedigree, Helfgott said.From 'back burner' to full throttle Novo Nordisk's rare disease ambitions first began to shift when Lars Fruergaard Jørgensen donned the mantle of CEO in 2017.“One of the intuitions that Lars had when he joined the CEO chair was to say, we have here something which is an uncut gem—something which is a rough diamond that we might want to polish, because at some point it might be very, very helpful,” Helfgott said of the company’s rare disease unit.When Helfgott joined Novo from AstraZeneca in 2019, he immediately set out on a mission to create “a biotech within Novo Nordisk," he explained.“There was a long endgame in being a leader in rare disease,” Helfgott added.While Novo has subsequently made strides building out capabilities in early research, development and its supply chain to embrace that newfound purpose—including recent designs on a $1.2 billion rare disease production plant in Denmark—there is still much work to be done, Helfgott said.“When I joined the company back in 2019, the R&D efforts [in rare disease] honestly had been a bit put on the back burner, and we had to reengage with research,” he explained. Over the past few years, Novo has relied on its dealmaking prowess to wade deeper into the space, but in order to be a discerning purchaser and partner, the company had to bolster its own R&D abilities too, Helfgott said.“The problem is that if you don’t have your internal quality research and development, you don’t do good [business development] because you can’t assess quality molecules, and you’re not really a credible partner,” he explained.In an example of how Novo’s rare disease outfit has since grown, Helfgott noted that the company’s early research team for the business has swelled from just eight people to around 90 since the executive’s arrival.Those efforts to bulk up in rare disease R&D, both internally and externally, have since led to some major rare disease deals for Novo in recent years, including the company’s $1.1 billion buyout of sickle cell disease-focused Forma Therapeutics in 2022 and its acquisition of 2seventy bio’s hemophilia A program in 2024.Building a path to the top To date, Novo’s focus in rare disease has hinged on blood and endocrine disorders, with those efforts yielding a number of approved hemophilia A and B treatments like Esperoct, NovoEight and Rebinyn, as well as drugs for growth hormone-related disorders such as Norditropin.More recently, Novo in December won a hard-fought FDA approval for its once-daily hemophilia injection Alhemo. The tissue factor pathway inhibitor (TFPI) antagonist, which is injected under the skin, is specifically approved to prevent or curb the frequency of bleeding episodes in patients 12 and older who have hemophilia A or B with certain factor inhibitors.Factor inhibitors are a complication of the rare bleeding disorder that can prevent factor replacement treatments—a well-established class of drugs for hemophilia—from working properly.Given Alhemo’s unique mode of action, much of Novo’s launch focus in the U.S. currently revolves around investments in medical education, Helfgott said. The company is also playing up the drug’s convenient subcutaneous delivery via prefilled syringe, which is a first in the hemophilia B space, where many medicines are infused, the executive pointed out.Outside the U.S., Alhemo has also won green lights in Australia, Japan and Switzerland and the European Union.Beyond hemophilia, the next frontier for Novo’s blood disorder focus is SCD, where the company recently advanced the asset etavopivat it picked up from Forma Therapeutics into phase 3. Patients with SCD are living with “two evils,” to hear Helfgott tell it. They’re forced to endure painful vaso-occlusive crises (VOCs), in which blood vessels are blocked and deprive tissues of oxygen, as well as the subsequent organ damage that results from those repeated VOCs, he explained.Novo hopes that etavopivat can tackle both issues at once, which even newer SCD meds have failed to achieve, Helfgott said.Back in December, Novo posted phase 2 data on etavopivat showing the highest studied dose (400 mg) nearly halved patients’ annualized VOC rates at the study’s 52-week mark compared with placebo. Etavopivat also appeared to delay the onset of patients’ first VOC, according to data presented at last year’s meeting of the American Society of Hematology (ASH).What's next for Novo in rare diseases As Novo looks to carve out a leadership position in the field, the company’s rare disease unit will retain its focus on blood and growth disorders, Helfgott said.“It’s our core, and we will continue to develop this both internally and externally,” he explained.As for how Novo aims to dig deeper into those disciplines, the company has grown “increasingly interested” in what it calls the “hemato-renal area,” which refers to the confluence between rare blood and kidney disorders, Helfgott said.To that end, Novo in late 2023 won an FDA nod for its ribonucleic acid interference (RNAi) drug Rivfloza to treat the rare disease primary hyperoxaluria type 1 (PH1). The disease is characterized by an overproduction of oxalate, which causes kidney stones to form and lead to progressive kidney damage.Rivfloza kicked off its U.S. launch “a few months ago,” with a European rollout soon to follow, Helfgott said. Meanwhile, M&A and other business development deals aren’t off the table as Novo looks to expand in blood, hemato-renal and endocrine disorders, the executive pointed out.External dealmaking could also deepen Novo’s access to new and promising modalities, as was the case with the $600 million collaboration agreement the company inked with NanoVation Therapeutics in September.Under that accord, Novo has secured a license to use NanoVation’s long-circulating lipid nanoparticle technology to develop two base-editing therapies in rare genetic diseases. The deal also covers up to five more targets in rare and cardiometabolic conditions. 

近日，丹麦医药巨头诺和诺德公司迎来了其成立100周年的里程碑。诺和诺德官网更新了一份长度超过100页的2023年报，完整地回顾了这个百年药企走过的第100个年头。在庆祝这一非凡成就的同时，公司的管理层也深刻认识到过去的成功并不能保证未来的持续进步。作为全球领先的慢性病治疗企业，诺和诺德致力于通过创新药物改变严重慢性疾病患者的生活，并以此塑造其长远愿景，即不仅要在核心治疗领域保持领先地位，还要成为推动全球人类健康改善的驱动力。诺和诺德成立于1923年。在走过的第一百年中，诺和诺德的核心贡献体现在行业领先的研发成果上，其疗法惠及了全球超过4000万患有严重慢性疾病的患者。其中，针对II型糖尿病和肥胖症未满足的需求日益增长，GLP-1为基础的疗法需求激增，促使诺和诺德达到了历史性的患者触及数量，北美及国际业务的销售额实现了强劲增长。诺和诺德努力成为一家可持续发展的公司，为社会和我们的未来事业创造价值。我们以对财务、环境和社会负责的方式开展业务，并践行诺和诺德之道。通过取得成功，我们将为慢性病患者、员工、合作伙伴、股东和社会创造长期价值。诺和诺德基金会通过诺和控股公司持有诺和诺德公司 77.1% 的投票权和 28.1% 的股份。诺和诺德基金会在三个战略领域提供资助：健康、可持续性和生命科学生态系统。2023 年，诺和诺德基金会提供超过 90 亿丹麦克朗的资助。诺和诺德在产品管线方面取得了显著进展。SELECT试验显示，司美格鲁肽2.4 mg 可将肥胖患者发生重大心血管事件的风险降低 20%，为此公司积极寻求对 Wegovy® 标签的更新；FLOW肾脏试验提前结束，基于 semaglutide 的新组合疗法 CagriSema 已进入II型糖尿病和肥胖症的三期临床开发阶段；潜在全球首款每周一次的基础胰岛素——icodec胰岛素正在等待监管批准。此外，诺和诺德还通过收购拓展了在心血管疾病领域的布局，如在罕见血液病领域推进 Mim8 和 etavopivat 的三期临床试验。在财务表现方面，诺和诺德展现了卓越的商业实力和社会责任。2023年度，公司实现了高达232.261亿丹麦克朗的净销售额，运营利润达到102.574亿丹麦克朗，全球员工人数增至64,319名，并在80个国家设有分支机构，研发投入超过450亿丹麦克朗，同比增长35%。同时，公司不断减少环境足迹，相较于2019年，运营和运输过程中的二氧化碳当量排放减少了34%，并且协助供应商转向100%可再生能源供电。为提升健康公平性，诺和诺德继续强化全球范围内的预防工作。新近与南非阿斯彭制药建立的生产合作关系将大幅提高非洲大陆平价胰岛素的供应量，而长期运行的“改变糖尿病”儿童项目以及“获取胰岛素承诺”等举措则持续支持低收入和中等收入国家的脆弱群体。在美国，更多患者受益于诺和诺德提供的包括非品牌生物制剂、低成本人胰岛素和患者援助计划在内的更广泛可负担方案。诺和诺德同样重视研发领域的投入与创新技术平台的合作，首个RNA干扰衍生疗法Rivfloza™已在美国获批，这标志着公司在新型疗法领域取得突破。加强波士顿地区研发中心的建设，有望打开更多的合作机会，加速药物发现与开发进程。诺和诺德将继续拓宽并深化产品管线，依托内部世界级研发能力和对外部创新及业务发展的聚焦，进军诸如代谢功能障碍相关脂肪性肝炎（MASH）、慢性肾病（CKD）和阿尔茨海默病（AD）等新兴治疗领域，追求疾病修饰和治愈性疗法。材料中也提到了诺和诺德公司所销售产品的专利到期日期的说明，并列出了这些产品在美国、中国、日本及欧洲地区，关于活性成分专利的具体到期日期，包括多款糖尿病、肥胖和罕见病产品。公司在糖尿病治疗领域的全球领导地位，通过GLP-1（胰高血糖素样肽-1）为基础的药物产品强劲增长而得到进一步巩固。在过去的一年里，诺和诺德凭借其GLP-1类药物如Ozempic®（司美格鲁肽）和Rybelsus®（口服司美格鲁肽）在全球范围内实现了显著的增长，尤其是在北美市场和国际业务中，这两大产品的快速接纳和广泛使用极大地推动了公司价值市场份额的提升。在肥胖市场方面，公司针对市场领先肥胖症疗法采取了谨慎与控制性上市策略。Wegovy®基于GLP-1受体激动剂原理开发，该产品建立在公司首款用于肥胖治疗的GLP-1药物Saxenda®的成功基础上，使得诺和诺德在市场上占据了明显的优势地位。Wegovy®的需求急剧增长，但受限于全球供应短缺以及前所未有的媒体关注带来的挑战。为确保疗法能够到达最需要的患者手中，并考虑到产能限制问题，公司在包括美国在内的各个主要市场采取了有计划、有限度的推广方式。在美国市场，Wegovy®的市场需求依然强劲，而在其他国家如英国，这款药品通过国家卫生服务系统内的专科体重管理服务提供给符合严格标准的患者使用，同时也可以通过私人注册医疗机构获取。诺和诺德充分意识到肥胖症在全球范围内的严重性和日益增长的趋势，预计到2030年将有超过12亿成年患者受到肥胖困扰。由于肥胖症与多种疾病的发生息息相关，例如糖尿病、心脏病和肝脏疾病、癌症等，因此，越来越多的医疗专业人员和社会公众认识到有效治疗肥胖症对于改善公共卫生状况和降低长期医疗成本的重要性。为了应对高需求和供应压力，诺和诺德不仅在供应链上加大投资以扩大生产规模，还调整了产品推出和分销模式，确保负责任地向现有患者群体提供适当治疗选择的同时，也为无力承担自费购药费用的患者预留了一定比例的产品供应，尤其是在新上市市场中，彰显了其社会责任感和对健康公平性的承诺。此外，通过监测和评估新市场的反应及患者的反馈，诺和诺德持续优化治疗方案和服务，致力于帮助更多患者实现长期健康改善的目标。在罕见病治疗领域，公司也展开了积极的战略布局，寻求可持续增长。尽管该部门在过去的一年中面临挑战，特别是由于生产限制导致核心产品 Norditropin®（用于生长激素缺乏症的长期疗法）销售额下降15%，但公司仍通过新推出的每周一次治疗方案Sogroya®来开拓市场机会。Sogroya®凭借其减少患者每日注射负担、并展现疗效优势的特点，为生长激素缺乏症患者带来了新的希望。面对供应问题对满足不断增长的产品需求带来的阻碍，诺和诺德将解决供应链瓶颈作为首要任务。为了减轻一次性注射装置供应的压力，公司采取了推出药物的一周制剂而非日制剂的形式，并且正在寻找降低对此类装置依赖的方法。同时，在罕见血液疾病方面，尽管NovoSeven®和NovoEight®销售下滑，但新产品如 Esperoct® 和 Refixia® 的强劲需求带动了血友病A和B治疗领域的整体销售额增长1%。公司已投入超过750亿丹麦克朗的资金用于全球生产网络的扩建，包括丹麦卡尔乌尔布洛普、法国沙特尔以及爱尔兰阿斯隆等地的主要设施扩张与收购棕色地带开发及生产场地。公司努力确保现有工厂24小时不间断地运营，以最大限度提高现有生产基础的产量，从而向全球范围内的更多患者提供所需的药品。最后，材料中小结了2023年业绩亮点：糖尿病治疗业务：诺和诺德继续巩固其在糖尿病治疗领域的领导地位，销售额以丹麦克朗计算增长了24%，按固定汇率计算增长了29%。特别是GLP-1为基础的产品销售增长强劲，推动全球价值市场份额上升至33.8%。肥胖症治疗市场：公司在肥胖症市场的表现显著，销售额按固定汇率（CER）计算增长了154%，达到约416亿丹麦克朗，展现出该领域产品的巨大市场需求和潜力。罕见病管线：尽管面临供应限制导致罕见病产品销售额下降15%（CER），但公司仍致力于解决供应链瓶颈问题，并批准了Somapacitan在美国、欧盟和日本用于儿童生长激素缺乏症的治疗，为罕见病业务恢复增长打下了基础。财务表现：全年实现总销售额增长36%（CER），运营利润增长44%（CER）。通过优化价值链操作效率，增强了公司的盈利能力，确保能够投资于未来的增长资产。可持续发展与社会责任：公司在环保方面取得进步，如碳排放减少；同时扩大了对糖尿病患者的医疗支持，在非洲地区通过合作伙伴生产平价胰岛素，以提高药品可及性。研发进展：完成了一系列关键药物的研发里程碑，包括口服司美格鲁肽不同剂量的三期临床试验成功、CagriSema在II型糖尿病中的三期临床试验启动以及心血管结局研究的积极结果等。2024年展望稀有疾病领域：预计通过加强现有管线并推进新疗法的开发，实现罕见病业务的持续增长前景。心血管疾病和其他新兴治疗领域：计划进一步拓展心血管疾病（CVD）、代谢功能障碍相关脂肪性肝炎（MASH）和慢性肾病（CKD）等新兴治疗领域，通过启动细胞疗法临床试验、收购高血压治疗药物以及开展针对特定靶点抗体的I期临床试验等方式来建立业务。糖尿病领导力强化：目标是在全球价值市场上继续保持领先地位，争取超过三分之一的市场份额。肥胖症领域：期望到2025年实现超过250亿丹麦克朗的销售收入，继续利用已有的优势产品及即将上市的新药推动销售增长。诺和诺德正在研发中的产品管线，涵盖了糖尿病、肥胖症、罕见病以及心血管疾病共四个治疗领域。糖尿病方向：Oral Semaglutide (NN9924) 描述：一款长效GLP-1类似物，25mg 和 50mg 剂量，设计为每日一次口服给药 适应症：II型糖尿病  阶段：已上市或处于后期开发阶段 Icodec NN1436 描述：一种长效基础胰岛素类似物，旨在每周一次皮下注射 适应症：I型糖尿病和II型糖尿病 阶段：已上市或处于后期开发阶段 CagriSema NN9388 描述：一款 Amylin 类似物 cagrilintide 和 GLP-1 类似物 semaglutide 的复方制剂，用于每周一次的皮下注射治疗。适应症：II型糖尿病和肥胖症 阶段：临床3期开发阶段 OW GLP-1 GIP NN9541 描述：这是一种GLP-1和GIP双重激动剂，设计为每周一次皮下注射 适应症：肥胖症和II型糖尿病 阶段：临床1期开发阶段 DNA Immunotherapy NN9041描述：一种编码前胰岛素和前胰岛素的新型质粒，用于保护β细胞功能。适应症：I型糖尿病 阶段：临床1期开发阶段 肥胖方向：Oral Semaglutide NN9932  描述：一款长效GLP-1类似物，设计为每日一次口服给药。 阶段：临床3期开发阶段 CagriSema NN9838 描述：一款 Amylin 类似物 cagrilintide 和 GLP-1 类似物 semaglutide 的复方制剂，用于每周一次的皮下注射治疗。Amycretin NN9487  描述：这是一款 GLP-1 受体和 amylin 受体的双重激动剂 (co-agonist) , 设计为每周一次皮下注射或一日一次口服阶段：临床1期开发阶段罕见病方向：Concizumab (NN7415)描述：一种针对组织因子途径抑制剂（TFPI）的单克隆抗体适应症：用于血友病A或B患者的预防性治疗，尤其是那些伴有抑制物（inhibitors）的患者阶段：已上市或处于后期开发阶段Nedosiran (NN7022) 描述：一种小干扰RNA药物，靶向乳酸脱氢酶A (LDHA) 适应症：原发性高草酸尿症1型（PH1） 阶段：已上市或处于后期开发阶段 Mim8 (NN7769) 描述：下一代FVIIIa模拟双特异性抗体 适应症：血友病A阶段：临床3期开发阶段Etavopivat NN7535 描述：第二代PKR-激活剂小分子药物 适应症：镰状细胞病（SCD） 阶段：临床2期开发阶段Etavopivat NN7536描述：同为第二代PKR激活剂小分子药物，与NN7535具有相似作用机制 适应症：地中海贫血 阶段：临床2期开发阶段NDec NN7533 描述：是一种针对镰状细胞病和/或地中海贫血的疗法，该药物是一种口服组合疗法，由去甲基化剂 decitabine 和 tetrahydrouridine 组成。这种联合用药旨在改善患者的疾病状况，并可能通过改变骨髓中血红细胞生成的过程来减轻症状和并发症。适应症：镰状细胞病和地中海贫血阶段：临床2期开发阶段心血管代谢方向：Ziltivekimab (NN6018) 描述：一款针对IL-6活性的单克隆抗体 适应症：慢性肾脏病（CKD）、动脉粥样硬化性心血管疾病（ASCVD）以及射血分数保留型心力衰竭 (HFpEF)阶段：临床3期开发阶段Ocedurenone NN6023描述：非甾体类选择性盐皮质激素受体拮抗剂（nsMRA），作为口服疗法用于心血管疾病（CVD）的治疗。适应症：心血管疾病阶段：临床3期开发阶段ATTR-CM NN6019描述：一种用于静脉注射的抗淀粉样蛋白免疫疗法。适应症：心血管疾病阶段：临床2期开发阶段CM4HF NN9003描述：一种用于恢复慢性心力衰竭患者心脏功能的试验性细胞疗法。适应症：心血管疾病阶段：临床2期开发阶段Anti-ANGPTL3 mAb NN6491描述：一种 ANGPTL3 中和扫抗体，用于每月一次的皮下注射治疗。适应症：心血管疾病阶段：临床2期开发阶段Semaglutide NN6535 ｜NN9931适应症：阿尔茨海默氏症和代谢功能障碍相关性脂肪性肝炎CagriSema NN9588 适应症：代谢功能障碍相关性脂肪性肝炎FGF21 NN9500描述：一种长效 FGF21 类似物，用于每周一次的皮下注射治疗。适应症：代谢功能障碍相关性脂肪性肝炎阶段：临床2期开发阶段LXRa NN6582描述：一种靶向 LXRa 的 siRNA，用于每月一次的皮下注射治疗。适应症：代谢功能障碍相关性脂肪性肝炎阶段：临床1期开发阶段MARC1 NN6581描述：一种靶向 MARC1 的 siRNA，用于每月一次的皮下注射治疗。适应症：代谢功能障碍相关性脂肪性肝炎阶段：临床1期开发阶段VAP-1i NN6561描述：一种用于每日一次口服治疗的 VAP-1 抑制剂。适应症：代谢功能障碍相关性脂肪性肝炎阶段：临床1期开发阶段SC4PD NN9001描述：一种用于改变疾病治疗的低温保存细胞疗法。适应症：帕金森病阶段：临床1期开发阶段STAT3 NN4002描述：GalXC 衍生脂质共轭物一次性皮下注射疗法。适应症：癌症阶段：临床1期开发阶段以上文章来源于GPCR drug discovery，作者段思研       本文仅作信息交流之目的，文中观点不代表相关疾病治疗立场，也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。