Year: 2011-12

Company: BioMarin Pharmaceuticals

Liaison(s): Kurt Yanagimachi

BioMarin Pharmaceutical Inc. is a leading innovator of biopharmaceuticals for the treatment of rare diseases. Since 1997, BioMarin has launched four products: Naglazyme®, Aldurazyme®, Kuvan®, and Firdapse®. These products treat mucopolysaccharidosis VI (MPS VI), mucopolysaccharidosis I (MPS I), phenylketonuria (PKU) and Lambert Eaten Myasthenic Syndrome, respectively. With commercial operations in over forty countries and a robust clinical pipeline, BioMarin is a key player in the orphan genetic disease market, providing fast access to treatments for patients with historically unmet medical needs. As BioMarin’s pipeline grows, the company intends to expand their process development capabilities. In support of this goal, BioMarin intends to construct a pilot plant facility that can accommodate larger-scale process development activities and produce sufficient quantities of material for early product characterization and pre-IND studies. The pilot plant would also be used for large-scale process testing which currently takes place in BioMarin’s clinical and commercial manufacturing facilities. Shifting process development capabilities to a dedicated pilot facility would provide additional flexibility to evaluate and troubleshoot processes, and possibly create a longer-term cost savings for the company. In three phases, the BioMarin TMP evaluated and designed a pilot plant facility to meet current and future process development needs. First, through discussions with functional leaders in BioMarin’s Process Sciences department, the team analyzed internal scale-up challenges in the department and identified the possible capabilities that would address these needs. Several necessities were recognized, such as the need for dedicated areas to perform process equipment testing, troubleshooting, and scale-up testing. Second, the BioMarin TMP team created a full layout of the potential pilot plant footprint based on a detailed analysis of all the required and desirable equipment for the facility. A range of scenarios and layouts were created to best analyze the opportunities for process development expansion. Finally, the team formed a business case for the implementation of a pilot plant. A Return on Investment (ROI) analysis was completed to present a preliminary financial justification for a pilot plant. Through these steps, the BioMarin TMP team was able to analyze how the benefits of these expanded capabilities might affect BioMarin’s process development strategy going forward.