Novartis Gene Therapies, Inc. – FDA EIR, Jun 16, 2021

An FDA inspection of Novartis Gene Therapies, Inc. in Libertyville, IL, was conducted from June 8 to June 16, 2021. This initial Level 1 Good Manufacturing Practice (GMP) inspection, part of the Team Biologics FY 2021 work plan, evaluated the firm's systems for manufacturing Zolgensma®, an FDA-approved gene therapy regulated as a biological drug product by CBER. The inspection identified five main observations documented on a Form FDA 483. These included failures to record and justify deviations from written specifications and laboratory mechanisms, and not retaining drug product reserve samples beyond their expiration dates. Critical issues also involved a lack of established procedures to prevent microbiological contamination for sterile drug products. Additionally, laboratory records were incomplete regarding test data to ensure compliance with specifications, and appropriate written specifications for component quality were not established. In response to these findings, Novartis Gene Therapies, Inc. management committed to submitting a written response to the FDA.