Technology Transfer and Process Development of Danicopan to Enable Process Validation

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.