Cincinnati Children's Hospital Medical Center, Division of Pulmonary Medicine, ML 2021, 3333 Burnet Ave., Cincinnati, OH 45229, USA.
J Cyst Fibros. 2009 Dec;8(6):405-17. doi: 10.1016/j.jcf.2009.07.006. Epub 2009 Aug 15.
EUR-1008 (Zenpep [pancrelipase]) is a new, enteric-coated, porcine-derived pancreatic enzyme product (PEP) developed for the treatment of cystic fibrosis (CF) patients with malabsorption associated with exocrine pancreatic insufficiency (EPI). Unlike currently marketed PEPs, EUR-1008 contains the label-claimed lipase content. Safety and efficacy were assessed in younger (<7 years) and older (> or =7 years) CF patients with EPI.
Two multicenter studies were conducted: a randomized, double-blind, placebo-controlled, crossover trial in patients > or =7 years of age (N=34) and a supplemental, open-label study in children <7 years of age (N=19). Use of any medications altering gastric pH/motility was prohibited during the studies. Outcome measures in the randomized trial included changes in the coefficient of fat absorption (CFA), coefficient of nitrogen absorption (CNA), and signs/symptoms of malabsorption for EUR-1008 vs. placebo. Outcome measures in the supplemental study included safety and response (defined as no steatorrhea and no overt signs/symptoms of malabsorption) to EUR-1008 vs. previous enzyme treatment.
In the randomized trial, EUR-1008 treatment compared to placebo resulted in a significantly higher mean CFA (88.3% vs. 62.8%, respectively) and CNA (87.2% vs. 65.7%, respectively) (both p<0.001) and reduced the incidence of malabsorption signs and symptoms in 32 evaluable patients. In the supplemental study, 11 of 19 patients met the criteria for responder with EUR-1008 at the end of the study vs. 10 of 19 patients at screening (previous PEP), and improvements in clinical symptoms were reported with EUR-1008 treatment. EUR-1008 was safe and well tolerated, and no serious drug-related AEs were reported in either study.
EUR-1008 was safe, well tolerated, and effective in CF patients of all ages with EPI-associated malabsorption in two clinical trials. Treatment led to clinically and statistically significant improvements in CFA and CNA in the randomized study, and control of malabsorption and clinical symptoms in both studies.
EUR-1008（Zenpep [胰酶]）是一种新型的肠溶性、猪源胰酶产品（PEP），专为伴有外分泌胰腺功能不全（EPI）的吸收不良的囊性纤维化（CF）患者而开发。与目前市售的 PEP 不同，EUR-1008 含有标签声称的脂肪酶含量。在伴有 EPI 的年轻（<7 岁）和年长（> =7 岁）CF 患者中评估了安全性和疗效。
进行了两项多中心研究：一项针对年龄≥7 岁患者的随机、双盲、安慰剂对照、交叉试验（N=34）和一项针对年龄<7 岁儿童的补充、开放标签研究（N=19）。在研究期间，禁止使用任何改变胃 pH/动力的药物。随机试验的结果包括脂肪吸收系数（CFA）、氮吸收系数（CNA）的变化以及 EUR-1008 与安慰剂相比的吸收不良迹象/症状。补充研究的结果包括 EUR-1008 与之前的酶治疗相比的安全性和反应（定义为无脂肪泻和无明显的吸收不良迹象/症状）。
在随机试验中，与安慰剂相比，EUR-1008 治疗导致平均 CFA（分别为 88.3%和 62.8%）和 CNA（分别为 87.2%和 65.7%）显著升高（均 p<0.001），并减少了 32 名可评估患者中吸收不良迹象和症状的发生率。在补充研究中，19 名患者中有 11 名在研究结束时符合 EUR-1008 的应答标准，而在筛选时（之前的 PEP）有 19 名患者中有 10 名符合应答标准，并且报告了 EUR-1008 治疗后临床症状的改善。EUR-1008 安全且耐受良好，在两项研究中均未报告与药物相关的严重不良事件。
EUR-1008 在两项临床试验中在伴有 EPI 相关吸收不良的所有年龄段的 CF 患者中安全、耐受良好且有效。治疗导致随机研究中 CFA 和 CNA 具有临床和统计学意义的改善，并且在两项研究中都控制了吸收不良和临床症状。
