Regulatory Round-up - January 2024

See the latest regulatory news from around the world with the Cell and Gene Therapy Catapult regulatory newsletter.


Medicines and Healthcare products Regulatory Agency (MHRA)

MHRA launches new fees calculator

MHRA has launched a new aid to help determine the fee that will be charged upon the submission of an application. Please find the link to the fee calculator here.

Implementation of medical devices future regime

The MHRA has set out new plans to protect patient safety and enable access without delay for UK patients to innovative medical technologies which outlines the timelines for delivering the future regulatory framework for medical devices. Please find further details here.

MHRA Board Meeting: Proposal Of New Operating Model On Regulation Of Clinical Trials

This paper sets out a new operating model for the regulation of clinical trials. It includes the regulatory strategy, capacity/capability considerations, legislative, cost/fee, communications and digital infrastructure elements. The proposals also address how the objectives of the UK Government Life Sciences Vision, the outcomes of the Lord O’Shaughnessy review and act as an enabler to UK life sciences will be met.

British Pharmacopoeia (BP)

New Advanced Therapy Medicinal Products Guidance: Characterisation of the Capsid Particle Population in rAAV Products

This new guidance document addresses the use of methods for the standardisation of vector capsid particles in rAAV-based therapies and will provide current best practices. It aims to promote the standardisation of the currently established methods within the cell and gene therapy community. This document has been created to be read in conjunction with applicable guidance for method development and validation. The document can be downloaded here.


European Commission (EC)

Cloud Computing: Consequences of different service models for Qualification / Validation

The pharmaceutical industry is moving towards cloud computing which has financial and organizational advantages; however, potential dangers and regulatory restrictions should also be taken into account. Nine experts from the pharmaceutical industry and regulatory authorities answer questions from the following GxP-relevant topics:

  • Basics of Cloud Computing Technology
  • Regulations and Expectations of Inspectors
  • Customer-Supplier-Relationship
  • Requirements for Cloud Service Providers (CSP)
  • Requirements for Supplier Evaluation and Supplier Audits
  • Requirements for Qualification / Validation

Please find Q&As on this topic here.

European Directorate for the Quality of Medicines (EDQM)

New strategy for N-nitrosamine impurities in Ph. Eur. monographs

The European Pharmacopoeia Commission (EPC) agreed to delete the Production section covering N-nitrosamine impurities from existing individual monographs on active substances and to avoid including such statements in new monographs in the future, as the general requirement for these impurities given in the revised general monograph 2034 applies to all the substances for pharmaceutical use within the given scope. Please find further information here.

European Medicines Agency (EMA)

ICH Q5A(R2) Guideline on viral safety evaluation of biotechnology products derived from cell lines of human or animal origin

The risk of viral contamination is a concern for all biotechnology products derived from cell lines as it could have serious clinical consequences. This guideline describes the evaluation of the viral safety of biotechnology products including viral clearance and testing, and it outlines what data should be submitted in marketing applications for those products.

Major update of the SME user guide

EMA has released a major revision of its user guide for micro, small, and medium-sized enterprises (SMEs) in the pharmaceutical sector, which reflects major changes in the EU’s legal and regulatory framework for human and veterinary medicines. The new release incorporates the following updates in human medicines:

  • Clinical Trials Regulation (new section 4.4): provides an overview of the clinical trial regulation and Clinical Trials Information System (CTIS)
  • Medical Devices Regulation (new section 4.8): offers insights into the medical devices regulation for human medicines

Other relevant new sections and subsections are:

  • IT Systems (section 1.3): An overview of common platforms, systems, and databases for application submission, data management, and secure communication with EMA/EU network
  • Borderline Products (section 3.6): Guidance on products with regulatory framework uncertainty
  • Environmental Risk Assessment (ERA) (section 4.2.4): Requirements to investigate potential environmental risks of a medicinal product following its use in patients
  • Big Data (section 4.3.6): Guidance on utilising big data for decision making
  • 'EU-M4all' (section 6.16): Guidance for access to high-priority medicines for patients outside of the EU
  • 'OPEN' Initiative (section 6.17): Information on EMA collaboration with non-EU regulators in the scientific evaluation of certain medicines

Please find further information, including the updated relevant documents, here.

PRAC recommendations on signals Adopted at the 27-30 November 2023 PRAC meeting

This document provides an overview of the recommendations adopted by the Pharmacovigilance Risk Assessment Committee (PRAC) on the signals discussed during the meeting of 27-30 November 2023. It provides supplementary information which is directly actionable by the concerned marketing authorisation holders (MAHs).

Paediatric Committee (PDCO): Work Plan 2024

Please find the PDCO’s Work Plan for 2024 including key objectives and activities here.

Meeting highlights from the Pharmacovigilance Risk Assessment Committee (PRAC) 8-11 January 2024APM- project manager

Please find Meeting highlights from the Pharmacovigilance Risk Assessment Committee (PRAC) 8-11 January 2024 including some updates on the safety review of the six approved chimeric antigen receptor (CAR) T-cell medicinal products.


Food and Drug Administration (FDA)

Submission and Review of Sterility Information in Premarket Notification (510(k)) Submissions for Devices Labeled as Sterile

Guidance for Industry and Food and Drug Administration Staff

This revised draft guidance document updates and clarifies the information regarding sterilization processes that the FDA recommend sponsors to include in 510(k)s for devices labeled as sterile. It also provides details about the pyrogenicity information that the FDA recommend sponsors to include in a 510(k) submission.

Potency Assurance for Cellular and Gene Therapy Products

The FDA has released a draft guidance on potency assurance for Cell and Gene Therapy Products. This document provides recommendations for developing a science- and risk-based strategy to help assure the potency of a human cellular therapy or gene therapy product through manufacturing process design, manufacturing process control, material control, in-process testing, and potency lot release assays. The goal of a potency assurance strategy is to ensure that every lot of a product released will have the specific ability or capacity to achieve the intended therapeutic effect. Please submit comments by the 27th of March 2024.

Data Standards for Drug and Biological Product Submissions Containing Real-World Data

The 21st Century Cures Act, signed into law on 13 December 2016, is intended to accelerate medical product development and bring innovations faster and more efficiently to patients. The FDA has issued a final guidance which provides recommendations to sponsors for complying with section 745A(a) of the FD&C Act (21 U.S.C. 379k-1(a)) when submitting RWD as study data in applicable drug submissions.

Rare Diseases: Considerations for the Development of Drugs and Biological Products

The FDA has issued a final guidance for industry entitled “Rare Diseases: Considerations for the Development of Drugs and Biological Products.” This guidance is intended to assist sponsors of drugs and biological products for treatment of rare diseases in conducting efficient and successful drug development programs through a discussion of selected issues commonly occuring in rare disease drug development.

BLA Approval: Casgevy

The FDA has granted BLA approval for Casgevy (Exagamglogene autotemcel), a cell-based gene therapy, for the treatment of patients aged 12 years and older with transfusion-dependent ß-thalassemia.

Please find Summary Basis for Regulatory Action here.

Summary Basis for Regulatory Action:LYFGENIA

Please find Summary Basis for Regulatory Action for lovotibeglogene autotemcel (LYFGENIA) which is indicated for treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events (VOEs). LYFGENIA consists of an autologous CD34+ cell enriched population containing hematopoietic stem and progenitor cells (HSCs) transduced with a non-replicating lentiviral vector (LVV), referred to as BB305, containing the human βA-T87Q-globin transgene sequence.

FDA eyes collaborative review pilot for gene therapies

The FDA is working on a pilot program that will explore the possibility of concurrent collaborative review of new gene therapy applications with other global regulators. The aim of the pilot is for partners to participate in internal regulatory meetings with sponsors of new gene therapy applications in order to increase the efficiency of the regulatory reviews, reducing the time and the cost for agencies and sponsors to bring these therapies to the market. Please find further information here.

Commission proposes to extend IVDR transition, accelerate EUDAMED adoption

The In Vitro Diagnostic Regulation (EU IVDR 2017/746) replaced the In Vitro Diagnostic Medical device Directive (EU IVDD 98/79/EC) and entered into force in 2017 with 26 May 2022 as date of application (DOA). Prior to this, in late 2021 the transition periods for diagnostics to comply with the regulation were extended, due to pressure from industry and fears of a market collapse for diagnostics. At the time, the EU extended the transition periods to 26 May 2025 for high-risk IVDs, 26 May 2026 for moderate-risk IVDs, and 26 May 2027 for lower-risk IVDs. Concerned that in vitro diagnostic manufacturers don’t have enough time to comply with the In Vitro Diagnostic Medical Devices Regulation (IVDR), the EC has again proposed extending the regulation’s transition period. Under the new proposal, high-risk class D diagnostics would get until December 2027 to undergo a conformity assessment under IVDR; class C, or high individual and/or moderate public health risk tests, will have until December 2028, and low risk class A sterile and class B tests would have the deadline extended to December 2029.

The EC is also proposing to make some European Database on Medical Devices (EUDAMED) modules mandatory by late 2025. EUDAMED will not become mandatory until all 6 of its modules are in place; however, under its proposed revisions, EUDAMED compliance will be rolled out gradually. Please find further information here.

Public consultations

Food and Drug Administration (FDA)

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