Novel oral liquid formulation of Enalapril for children

儿童依那普利新型口服液体制剂

• 批准号: 710237
• 金额: $ 10.39万
• 依托单位: PROVECA
• 依托单位国家: 英国
• 项目类别: GRD Proof of Concept
• 财政年份: 2013
• 资助国家: 英国
• 起止时间: 2013 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=710237
• 关键词: Novel; oral; liquid; formulation; Enalapril

With today’s major research and development activities of pharmaceutical companies focusedon meeting the needs of an increasingly aging population, children who represent the bulk ofthe dependent population worldwide still remain a widely neglected niche market.The recognition of the rights of children to have access to safe and effective drugs and theneeds of healthcare providers to receive age-appropriate drug information continues to have amajor effect on all areas of paediatric pharmaceuticals. In line with this challenge, Proveca’sobjective is to develop a new dosage formula of an off-patent drug for the treatment of heartfailure in children and apply for the relevant authorisation. The currently available licensedmedicine is for adults; however children are either unable or unwilling to swallow tablets,proving problematic in the long-term use.Alternatives of the drugs are available in liquid form, however the lack of formalisedlicensing with an official regulatory body causes several concerns. With the formulation ofmedicines being a significant parameter to ensure optimal dosing levels with minimal toxicity,the high variation in the currently unlicensed liquid formulations dispensed to children in theUK raise significant issues related to patient safety and drug efficacy. With a growingpressure on drug safety and new requirements on risk evaluation and mitigation strategies(REMS), there is an increasing need to deliver accuracy and congruence of medication supplyto patients.Proveca’s new formulation has the potential to improve children’s physiologic status, qualityof life and length of life. The aim of this Proof of Concept is to enable Proveca to conductpreclinical feasibility studies and develop a basic prototype of the formulation which will befurther optimised during clinical trial stages.

随着当今制药公司的主要研究和开发活动集中于满足日益老龄化的人口的需求，占全球依赖人口大部分的儿童仍然是一个被广泛忽视的利基市场。承认儿童获得安全有效药物的权利，以及医疗保健提供者需要获得适龄药物信息，继续对儿科药物的所有领域产生重大影响。为了应对这一挑战，普罗维卡的目标是开发一种治疗儿童心力衰竭的非专利药物的新剂量配方，并申请相关授权。目前获得许可的药物是成人使用的；然而，儿童不能或不愿意吞下片剂，这在长期使用中证明是有问题的。这些药物的替代药物可以液体形式获得，然而，缺乏官方监管机构的正式许可引发了几个问题。由于药物配方是确保最优剂量水平和最小毒性的重要参数，目前在英国分发给儿童的无证液体配方的高度多样性引发了与患者安全和药物疗效相关的重大问题。随着对药物安全的压力越来越大，以及对风险评估和缓解策略(REMS)的新要求，越来越需要向患者提供准确和一致的药物供应。普罗维卡的新配方有可能改善儿童的生理状态、生命质量和生命长度。这一概念验证的目的是使普罗维卡能够进行临床前可行性研究，并开发该配方的基本原型，该配方将在临床试验阶段得到进一步优化。