FDA EIR - BioMarin Pharmaceutical, Inc. - April 02, 2021

An FDA Pre-Approval Inspection (PAI) of BioMarin Pharmaceutical, Inc.'s Novato, CA facility was conducted from March 25 to April 2, 2021. This inspection, requested by CDER for an undisclosed New Drug Application (NDA) and following up on a previous 2017 inspection related to BLA 761079 Palynziq, aimed to assess readiness for manufacturing, conformance to application details, and data integrity. The previous 2017 inspection resulted in a 10-item Form FDA-483, citing deficiencies such as inadequate real-time documentation of laboratory equipment use, failure to determine root causes for deviations, unsuitable pH meter calibration procedures for in-process materials, and incomplete process and cleaning validation for commercial manufacturing readiness. During the current inspection, two new observations were issued to management. These included the absence of a written procedure for requesting shipping samples for laboratory testing and a lack of a written procedure for managing test results from contract laboratories. BioMarin management acknowledged the observations and committed to providing a written response within 15 business days. Notably, despite these observations, the inspection recommended approval for the NDA, indicating that the issues, including follow-ups from the previous inspection, were deemed resolvable or not critical enough to impede approval.