FDA EIR - BioMarin Pharmaceutical, Inc. - November 09, 2018

An FDA Good Laboratory Practice (GLP) inspection of BioMarin Pharmaceutical, Inc. took place in San Rafael, CA, from November 5 to November 9, 2018. This preannounced, for-cause inspection was requested by the European Medicines Agency (EMA) and conducted under Compliance Program 7348.808, focusing on three specific nonclinical studies. Although no formal Form FDA 483 (Inspectional Observations) was issued, several significant issues were discussed at the close-out meeting.

Main findings included that freeze-thaw stability was not fully validated for certain samples, and numerous samples were retested beyond established freeze-thaw cycles without proper reporting or coverage in stability data. Inspectors also identified incomplete information within bioanalytical reports, and instances where Standard Operating Procedures (SOPs) were not fully complied with. Additional problems involved inappropriate calibration standards and quantitative ranges in some studies, as well as errors in source documents and unreported failed runs. Data inconsistencies stemming from different software versions were also noted.

BioMarin management acknowledged these findings, stating they would provide a formal written response to the discussion items within 15 business days. They also emphasized their assessment that some bioanalytical method issues were clinically irrelevant based on human clinical trial results. This inspection was the firm's first GLP inspection.