Novel oral liquid formulation of Enalapril for children

随着当今制药公司的主要研发活动集中在满足日益老龄化的人口的需求，儿童占全世界受抚养人口的大部分，但仍是一个被广泛忽视的利基市场。适当的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，Proveca的目标是开发一种用于治疗儿童心力衰竭的非专利药物的新剂型，并申请相关授权。目前可获得的许可药物是成人使用的;然而儿童不能或不愿意吞咽药片，证明长期使用存在问题。药物的替代品以液体形式提供，但是缺乏官方监管机构的正式许可引起了一些担忧。由于药物的配方是确保最佳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未经许可的液体制剂的高度变化引起了与患者安全和药物疗效相关的重大问题。随着对药物安全性的压力越来越大，以及对风险评估和缓解策略（REMS）的新要求，越来越需要为患者提供准确和一致的药物供应。Proveca的新配方有可能改善儿童的生理状态，生活质量和寿命。这一概念验证的目的是使Proveca能够进行临床前可行性研究，并开发出一个基本的配方原型，该原型将在临床试验阶段进一步优化。

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.