Polak Y, Hollak C E M, Swart E L, Kemper E M
Platform Medicine for Society, Amsterdam, the Netherlands; Department of Pharmacy and Clinical Pharmacology, Amsterdam UMC, University of Amsterdam, Amsterdam, the Netherlands.
Platform Medicine for Society, Amsterdam, the Netherlands; Department of Endocrinology and Metabolism, Amsterdam UMC, University of Amsterdam, Amsterdam, the Netherlands.
Eur J Pharm Sci. 2025 Jun 1;209:107083. doi: 10.1016/j.ejps.2025.107083. Epub 2025 Mar 30.
Commercial cholic acid (CA) treatment is currently unavailable to Dutch patients with a bile acid synthesis defect. CA treatment has been hypothesized to correct the biochemical abnormalities associated with these disorders and potentially slowing down disease progression. To address this need, the hospital pharmacy of Amsterdam University Medical Center developed CA capsules for use in a clinical trial in The Netherlands. Challenges arose during the quality control of CA active pharmaceutical ingredient (API) as no specific substance monograph is available in the European Pharmacopoeia or other effective pharmacopoeias. In this article, we share an analytical method validated for testing the purity of CA and present the results of its quality control, offering practical guidance for ensuring adequate quality control of CA. Pharmaceutical quality tests were performed on four batches of CA following the guidance given in the European Pharmacopoeia general monograph on substances for pharmaceutical use. The results confirm the suitability of our analytical method for CA quality control and demonstrate that the CA meets the high-quality standards required for pharmaceutical use.
目前，荷兰患有胆汁酸合成缺陷的患者无法获得商业化的胆酸（CA）治疗。据推测，CA治疗可纠正与这些疾病相关的生化异常，并可能减缓疾病进展。为满足这一需求，阿姆斯特丹大学医学中心医院药房开发了用于荷兰一项临床试验的CA胶囊。在CA活性药物成分（API）的质量控制过程中出现了挑战，因为欧洲药典或其他有效药典中没有特定的物质专论。在本文中，我们分享了一种经过验证的用于测试CA纯度的分析方法，并展示了其质量控制结果，为确保对CA进行充分的质量控制提供了实用指导。按照欧洲药典关于药用物质的通用专论中的指导，对四批CA进行了药品质量测试。结果证实了我们的分析方法适用于CA的质量控制，并表明该CA符合药用所需的高质量标准。
