Technology transfer and Current Good Manufacturing Practice (cGMP) manufacture of danicopan are described. During the acquisition of Achillion Pharmaceuticals (Achillion) by Alexion Pharmaceuticals (Alexion), a staged approach for technology transfer to Cambrex Charles City (CCC) was developed. The difference in Alexion’s and CCC’s approaches to process-validation-enabling studies as well as strategies to bridge the gap and meet both organizations’ expectations within a compressed timeline are discussed. The differences are best illustrated with an example step that was successfully validated concurrently at 45 and 210 kg scales in different work centers at the CCC facility to support multiple indications and programs for an advanced pharmaceutical intermediate. Key learnings are offered from the perspective of a sponsor organization and a contract manufacturer, highlighting a collaborative approach and partnership that allowed a successful execution with ambitious timelines while navigating the challenges of a global pandemic.

中文翻译：

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Danicopan 的技术转让和工艺开发以实现工艺验证


描述了 danicopan 的技术转让和现行良好生产规范 (cGMP) 生产。在 Alexion Pharmaceuticals (Alexion) 收购 Achillion Pharmaceuticals (Achillion) 期间，制定了向 Cambrex Charles City (CCC) 进行技术转让的分阶段方法。讨论了 Alexion 和 CCC 在流程验证支持研究方法上的差异，以及在压缩的时间内弥合差距并满足两个组织期望的策略。通过一个示例步骤可以最好地说明这些差异，该示例步骤在 CCC 设施的不同工作中心同时以 45 公斤和 210 公斤规模成功进行了验证，以支持高级药物中间体的多种适应症和项目。从赞助组织和合同制造商的角度提供了重要的经验教训，强调了协作方法和伙伴关系，使我们能够在雄心勃勃的时间表内成功执行任务，同时应对全球大流行的挑战。