Here we review strategies for gaining Food and Drug Administration (FDA) approval of allogeneic, pluripotent cell therapies. The crux of the discussion is that when developing a cell therapeutic, it is critical to look as much as a decade ahead to when FDA approval will be sought to commercialize the product through a biologics license application (BLA). While this discussion focuses on FDA approval of cell therapies, it is important to acknowledge the vast number of cell therapy clinical efforts occurring globally, particularly in Europe and Asia, and the specific regulatory requirements for those studies as well. Since many outstanding reviews on cell manufacturing are available, we specifically focus on the “how and why” of integrating high-quality raw materials and ancillary materials into the manufacturing process of pluripotent cell therapies throughout the product development process.
This consideration is especially crucial for pluripotent cell therapies because unlike many other forms of cell therapeutics that are tailored for individual patients and rely on small batch production, pluripotent cell therapies have as one of the first steps the establishment of a master cell bank that must last the lifetime of the product. Thus, raw material quality is paramount from the beginning of the development process. Obtaining high-quality raw materials from suppliers experienced in supporting cell therapy development—from manufacturing to delivery— can increase the probability of success and head off costly surprises that could cause an untimely demise for a promising pluripotent cell therapy candidate.