Department of Pharmacology, Institute of Medical Sciences, Banaras Hindu University, Varanasi, Uttar Pradesh, 221005, India.
Department of Pharmacology and Therapeutics, All India Institute of Medical Sciences, Gorakhpur, Uttar Pradesh, India.
Clin Drug Investig. 2024 Apr;44(4):223-250. doi: 10.1007/s40261-024-01351-5. Epub 2024 Mar 9.
Despite advances in the management of type 2 diabetes mellitus (T2DM), one-third of patients with diabetes do not achieve the desired glycemic goal. Considering this inadequacy, many agents that activate glucokinase have been investigated over the last two decades but were withdrawn before submission for marketing permission. Dorzagliatin is the first glucokinase activator that has been granted approval for T2DM, only in China. As overstimulation of glucokinase is linked with pathophysiological disturbances such as fatty liver and cardiovascular issues and a loss of therapeutic efficacy with time. This review aims to highlight the benefits of glucokinase activators vis-à-vis the risks associated with chronic enzymatic activation. We discuss the multisystem disturbances expected with chronic activation of the enzyme, the lessons learned with glucokinase activators of the past, the major efficacy and safety findings with dorzagliatin and its pharmacological properties, and the status of other glucokinase activators in the pipeline. The approval of dorzagliatin in China was based on the SEED and the DAWN trials, the major pivotal phase III trials that enrolled patients with T2DM with a mean glycosylated hemoglobin of 8.3-8.4%, and a mean age of 53-54.5 years from multiple sites in China. Patients with uncontrolled diabetes, cardiac diseases, organ dysfunction, and a history of severe hypoglycemia were excluded. Both trials had a randomized double-blind placebo-controlled phase of 24 weeks followed by an open-label phase of 28 weeks with dorzagliatin. Drug-naïve patients with T2DM with a disease duration of 11.7 months were enrolled in the SEED trial while the DAWN trial involved patients with T2DM with a mean duration of 71.5 months and receiving background metformin therapy. Compared with placebo, the decline in glycosylated hemoglobin at 24 weeks was more with dorzagliatin with an estimated treatment difference of - 0.57% in the SEED trial and - 0.66% in the DAWN trial. The desired glycosylated hemoglobin (< 7%) was also attained at more than two times higher rates with dorzagliatin. The glycemic improvement was sustained in the SEED trial but decreased over 52 weeks in the DAWN trial. Hyperlipidemia was observed in 12-14% of patients taking dorzagliatin versus 9-11% of patients receiving a placebo. Additional adverse effects noticed over 52 weeks with dorzagliatin included an elevation in liver enzymes, hyperuricemia, hyperlacticacidemia, renal dysfunction, and cardiovascular disturbances. Considering the statistically significant improvement in glycosylated hemoglobin with dorzagliatin in patients with T2DM, the drug may be given a chance in treatment-naïve patients with a shorter disease history. However, with the waning therapeutic efficacy witnessed in patients with long-standing diabetes, which was also one of the potential concerns with previously tested molecules, extended studies involving patients with chronic and uncontrolled diabetes are needed to comment upon the long-term therapeutic performance of dorzagliatin. Likewise, evidence needs to be generated from other countries, patients with organ dysfunction, a history of severe hypoglycemia, cardiac diseases, and elderly patients before extending the use of dorzagliatin. Apart from monitoring lipid profiles, long-term safety studies of dorzagliatin should involve the assessment of serum uric acid, lactate, renal function, liver function, and cardiovascular parameters.
尽管 2 型糖尿病(T2DM)的管理取得了进展,但仍有三分之一的糖尿病患者无法达到理想的血糖目标。考虑到这种不足,过去 20 年来,许多激活葡萄糖激酶的药物已经被研究过,但在提交上市许可申请之前就被撤回了。度拉糖肽是第一个获得批准用于 T2DM 的葡萄糖激酶激活剂,仅在中国。由于葡萄糖激酶的过度刺激与脂肪性肝病和心血管问题等病理生理紊乱以及随着时间的推移治疗效果的丧失有关。本综述旨在强调葡萄糖激酶激活剂相对于与慢性酶激活相关的风险的益处。我们讨论了慢性激活酶时预期的多系统紊乱,过去使用葡萄糖激酶激活剂的经验教训,度拉糖肽的主要疗效和安全性发现及其药理学特性,以及其他处于研发管道中的葡萄糖激酶激活剂的状况。度拉糖肽在中国的获批是基于 SEED 和 DAWN 两项临床试验,这两项主要的 III 期关键性试验在中国多个地点纳入了糖化血红蛋白平均为 8.3-8.4%、平均年龄为 53-54.5 岁的 T2DM 患者。患有未控制的糖尿病、心脏病、器官功能障碍和严重低血糖病史的患者被排除在外。两项试验均有 24 周的随机双盲安慰剂对照阶段,随后是 28 周的度拉糖肽开放标签阶段。SEED 试验纳入了病程为 11.7 个月的 T2DM 药物初治患者,而 DAWN 试验纳入了病程为 71.5 个月且正在接受背景二甲双胍治疗的 T2DM 患者。与安慰剂相比,SEED 试验中 24 周时糖化血红蛋白的下降幅度更大,度拉糖肽的估计治疗差异为-0.57%,而 DAWN 试验为-0.66%。在接受度拉糖肽治疗的患者中,达到理想的糖化血红蛋白(<7%)的比例也高出两倍以上。SEED 试验中的血糖改善持续存在,但 DAWN 试验中在 52 周时下降。在接受度拉糖肽治疗的患者中,12-14%出现血脂异常,而接受安慰剂治疗的患者中为 9-11%。在接受度拉糖肽治疗的患者中,在 52 周时还观察到其他不良反应,包括肝酶升高、高尿酸血症、高乳酸血症、肾功能障碍和心血管紊乱。考虑到度拉糖肽在 T2DM 患者中使糖化血红蛋白显著改善,该药可能有机会用于病程较短的药物初治患者。然而,在病程较长的糖尿病患者中观察到治疗效果减弱,这也是之前测试的分子的潜在关注点之一,需要进行涉及慢性和未控制糖尿病患者的长期研究,才能对度拉糖肽的长期治疗性能进行评价。同样,在将度拉糖肽的使用范围扩大到其他国家、有器官功能障碍、有严重低血糖病史、有心脏病和老年患者之前,需要生成来自其他国家的证据。除了监测血脂谱外,度拉糖肽的长期安全性研究还应包括评估血尿酸、乳酸、肾功能、肝功能和心血管参数。