Author:
Aisha Khan, PhD, MBA
University of Miami, Miller Scholl of Medicine
USA
The FDA has recently released a draft guidance document titled “Potency Assurance for Cellular and Gene Therapy Products,” providing recommendations for developing a science- and risk-based strategy to ensure the potency of cellular and gene therapy (CGT) products. This guidance is crucial for developers and manufacturers in the CGT field, as it outlines the necessary steps to ensure that these innovative therapies meet their intended therapeutic effects consistently.
Key Highlights:
1. Introduction and Background: The draft guidance emphasizes the importance of a comprehensive potency assurance strategy that includes manufacturing process design, process control, material control, in-process testing, and potency lot release assays. The goal is to ensure that each lot of a product released has the specific ability to achieve the intended therapeutic effect.
2. Regulatory Framework: The guidance provides detailed information on the regulatory requirements for both licensed and investigational CGT products. It stresses the necessity for potency testing for lot release and outlines the requirements for validating potency assays and ensuring continued potency through manufacturing changes.
3. Developing a Potency Assurance Strategy: A significant portion of the guidance is dedicated to the development of a robust potency assurance strategy. This involves:
• Quality Risk Management: Identifying and mitigating risks to product potency.
• Product and Process Understanding: Gaining a deep understanding of the product’s mechanism of action and the manufacturing process.
• Control Strategy: Implementing controls for materials, process parameters, in-process testing, and lot release testing.
4. Potency Assays and Acceptance Criteria: The guidance outlines the characteristics of effective potency assays, including precision, accuracy, specificity, and robustness. It also discusses the importance of setting appropriate acceptance criteria to ensure the potency of CGT products
Resources and Next Steps:
To align with this guidance, CGT developers need to:
• Utilize available resources like prior knowledge and published information to inform their potency assurance strategies.
• Engage with the FDA early in development for feedback and advice on potency assay development.
• Implement and validate potency assays as part of their control strategy to ensure the consistent potency of their products.
The draft guidance document is open for public comment, and stakeholders in the CGT field are encouraged to review and provide feedback to the FDA.
References: FDA. (2023). Potency Assurance for Cellular and Gene Therapy Products: Draft Guidance for Industry.