FDA EIR - BioMarin Pharmaceutical Inc. - November 06, 2020

BioMarin Pharmaceutical, Inc. underwent an FDA Bioresearch Monitoring inspection from October 20 to November 6, 2020. This comprehensive inspection, conducted partially remotely due to the COVID-19 pandemic, reviewed several clinical protocols under the FDA's Prescription Drug User Fee Act (PDUFA) framework and Compliance Program 7348.810 for sponsors and contract research organizations.While a formal FDA Form 483 (Inspectional Observations) was not issued at the conclusion of the inspection, "Discussion Items" regarding various operational aspects were addressed with BioMarin's management. These discussions highlighted several instances of significant non-compliance previously identified by BioMarin's internal monitoring efforts at clinical sites across multiple studies. Issues included delays in registering a clinical trial on ClinicalTrials.gov, inadequate documentation of protocol amendment training for investigators and staff, and deficiencies in informed consent processes. Furthermore, specific site-level issues involved repeated failures to upload electrocardiograms, enrollment of ineligible subjects, late reporting of adverse events, improper handling of source documents including destruction of originals, and a subject receiving prohibited medication.BioMarin had proactively implemented corrective and preventive actions (CAPAs) for these identified non-compliances, which the Sponsor and Monitor consistently followed up on. The company concluded that these issues, though significant, did not impact patient safety, though some had potential implications for data integrity. The FDA's engagement focused on these already recognized and addressed issues rather than uncovering new, unaddressed violations.