PDA EU00190 CMC Regulatory Compliance Strategy for ATMPs – CGTPs

Advanced Therapy Medicinal Products - Cellular/Gene Therapy Products

Gothenburg, Sweden
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Chemistry, Manufacturing & Control (CMC) regulatory compliance strategy encompasses all of those activities that are required/expected to be carried out by a regulatory authority to protect the patient from the manufactured drug product. Such activities involve Genetic Development (gene development, cell clonality), Process Development (design of the manufacturing process), Analytical Development (design of the release/stability test methods), Manufacturing (carrying out the upstream and downstream, and drug product processes), Quality Control (testing to meet specifications), Quality Assurance (cGMP compliance) and Regulatory Affairs (communication with the authorities). Compared to other biologic drug products, the coordination and timely completion of all the activities by all these CMC groups has never been more needed than for ATMPs/CGTPs.

While many CMC teams are familiar with the CMC regulatory compliance strategy for other drug products (e.g., chemical drugs, monoclonal antibodies), they may not appreciate the significant differences and extra challenges in the CMC regulatory compliance strategy for ATMPs/CGTPs. These extra CMC challenges are due to the increased size and complexity of these biologics (infectious AAV viral vectors, mRNA non-viral vectors, living genetically modified patient cells), as well as the limitations of the manufacturing processes (e.g., limited number of batches, increased biological variability).

This course will provide insights and practical guidance for the CMC teams to develop a cost-effective, risk-based CMC regulatory-compliant strategy for the manufacture and control of ATMPs/CGTPs from first-in-human (FIH) clinical studies through market approval. The course emphasis will include CMC regulatory guidance (FDA, EMA, and ICH), as well as real-world case examples illustrating CMC regulatory compliance delays in advancing clinical development.

Who Should Attend

This two-day CMC regulatory compliance strategy course for ATMPs/CGTPs is designed for senior management, directors, managers, supervisors, project managers, and personnel in Process/Analytical Development, Manufacturing, Quality, and Regulatory Affairs departments.

Learning Objectives

Upon completion of this course, attendees will be able to:

  • Understand the importance and underlying principles of an effective CMC regulatory strategy for ATMPS – CGTPs to move their products through clinical development into the marketplace
  • Learn how to avoid major CMC delays in clinical development or market approval due to an ineffective CMC regulatory compliance strategy

Standard Registration

Member Price

€ 2.090


€ 2.090

See Qualifying Criteria for Member Types.


WAYS TO REGISTER: 1. Online Registration   2. [email protected]

PDA MEMBERS: Please remember to log in before registering If you come from a Governmental or Health Authority Institution or from an Academic Organization, please contact [email protected] before registering.

If you need to apply for a visa to enter the event country, PDA Europe must be informed of this fact at least 4 weeks before the start of the event.

Day 1

Monday, 23 September 2024

Welcome and Introduction, collecting Participant’s Expectations

CMC Regulatory Compliance is Challenging for ATMPs-CGTPs

  • Discussion of the increasing diversity of ATMPs-CGTPs and the CMC regulatory challenges they present
  • The critical role that the Genetic/Process/Analytical Development groups, Manufacturing, QA/QC and Regulatory Affairs have in assuring that the CMC regulatory compliance protects patients

Coffee Break

Similarities/Differences in CMC Regulatory Compliance

  • FDA/EMA regulatory systems in place for ATMPs-CGTPs
  • Understanding why these products are not regulated like chemical drugs (or in some cases, even like protein-based biopharmaceuticals)

Lunch Break

Risk-Managed CMC Regulatory Compliance Strategy

  • Why a risk-managed approach to CMC regulatory compliance is absolutely necessary for these products
  • Application of Quality by Design (QbD) – QTPP, CQA, CPP – and Quality Risk Management (QRM)

Coffee Break

Applied Risk-Managed CMC Regulatory Compliance Strategy Across the Manufacturing Process

  • Raw materials and components – risk reduction procedures
  • Starting materials (DNA plasmids, viral vectors (LV, mRNA non-viral vector, patient/donor cells)

End of Training Course Day 1

Day 2

Tuesday, 24 September 2024

Applied Risk-Managed CMC Regulatory Compliance Strategy Across the Manufacturing Process

  • Upstream production (transfection, transduction) and downstream purification processes (viral and non-viral vectors)
  • ‘Minimum CMC regulatory compliance continuum’ applied to manufacturing process control

Coffee Break

Applied Risk-Managed CMC Regulatory Compliance Strategy Across the Manufacturing Process

  • Formulation and drug product manufacturing processes
  • Product testing and specification assignments
  • Administering the final drug product – vial to vein concerns

Lunch Break

Challenges in Demonstrating Product Comparability After Manufacturing Changes

  • Regulatory guidance in planning for product comparability studies – adapting ICH Q5E
  • Three risk-based considerations to reduce residual uncertainty of product comparability
  • Industry experience in controlling process changes for ATMPs-CGTPs

Coffee Break

Critical Manufacturing/Quality-Focused Meetings with Regulatory Authorities

  • CMC regulatory compliance deficiencies can seriously delay clinical development advancement
  • Meeting opportunities with regulatory authorities for ATMPs-CGTPs

End of Training Course

Agenda is subject to change.

Featured Trainers

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PDA recommends the reservation at the following hotel

Elite Park Avenue Hotel 
Kungsportsavenyn 36 - 38
411 36 Gothenburg,

PDA Europe has reserved a limited number of rooms until 20 July 2024.

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