FDA 483 - BioMarin Pharmaceuticals, Inc. - September 24, 2013

An FDA Form 483 was issued to Jeiovarin maraceuticale, tac. following an inspection conducted from September 16 to September 24, 2013. The primary observation highlighted a failure to ensure proper monitoring of a clinical study. Specifically, the company's contract research organization (CRO) was found to have inadequately reviewed and verified case report forms and source documents for a study subject. This oversight led to a critical error where a discrepancy in the subject's reported weight resulted in an overdose of the study drug during weeks 5 through 8 of the clinical trial. These observations signify potential non-compliance with regulatory expectations for clinical trial oversight, which mandate rigorous monitoring to safeguard patient safety and data integrity. While the document itself details the observation, an FDA 483 typically requires the inspected firm to thoroughly investigate the identified issues, propose comprehensive corrective actions to address the deficiencies, and implement preventive measures to avoid recurrence. Such actions are crucial to uphold patient welfare and ensure the reliability of clinical trial data, aligning with the regulatory framework governing pharmaceutical research.