Regulatory Round-up - March 2023

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


European Directorate for the Quality of Medicines (EDQM)

The future of pyrogenicity testing: new approaches discussed at joint EDQM-EPAA event

The European Directorate for the Quality of Medicines & HealthCare (EDQM) and the European Partnership for Alternative Approaches to Animal Testing (EPAA) have held a three-day joint event in February aimed at phasing out the rabbit pyrogen test (RPT) from the testing of pharmaceuticals worldwide.

The European institutions and EU member states are commited to achieve their 3R goals which are replacement, reduction and refinement of animal testing. Please find further details here.

European Medicines Agency (EMA)

EMA pilots scientific advice for certain high-risk medical devices

EMA has launched a pilot to give scientific advice on the intended clinical development strategy and proposals for clinical investigation for certain high-risk medical devices including all class III devices and class IIb active devices intended to administer and/or remove medicinal products. Manufacturers can submit their letter of interest to be part of the pilot on scientific advice which will be provided by the medical device expert panels. Please find further details here.

Advancing regulatory science in the EU – mid-point report published

The European Medicines Agency (EMA) has published a report summarising the mid-term achievements of its Regulatory Science Strategy (RSS) to 2025. The strategy was developed in 2018 and 2019 in consultation with a wide range of stakeholders, then became published in March 2020. The report provides an overview of the main deliverables achieved between March 2020 and December 2022 across the human and veterinary areas.


Medicines and Healthcare products Regulatory Agency (MHRA)

MHRA trialling pioneering stem cell robot that could transform the availability of life-saving cell therapies

An innovative new robot that grows stem cells, the CellQualiaTM Intelligent Cell Processing System, is being trialled by the Medicines and Healthcare products Regulatory Agency (MHRA) and the MHRA’s UK Stem Cell Bank is one of only two places in the world to test this technology.

Stem cell-based therapeutics are difficult to manufacture and their current availability is limited. Therefore, most treatments for degenerative diseases are focused on limiting the extent of damage rather than fixing the damage that has already occurred. The system being tested at the MHRA has the potential to change this, offering new hope to patients with serious diseases. Please find more details here.

MHRA to receive £10m from HM Treasury to fast-track patient access to cutting-edge medical products

HM Treasury has announced that the Medicines and Healthcare products Regulatory Agency (MHRA) will receive £10 million funding to help bring innovative new medicines and medical technologies to UK patients more quickly. It will support the development of a shortened process to speed up the approval process for cutting-edge treatments developed in the UK as well as support the establishment of an international recognition framework, allowing the MHRA to capitalise on the expertise and decision-making of trusted regulatory partners and provide patients with fast-track access to best-in-class medical products that have been approved in other countries. Further details can be found here.

Consultation outcome: Government response to consultation on legislative proposals for clinical trials

The Medicines and Healthcare products Regulatory Agency (MHRA) has published their response to the consultation on the clinical trials regulation update. A series of new measures will be introduced by the MHRA with support from partners to make it faster and easier to gain approval and to run clinical trials in the UK. These changes represent the biggest overhaul in UK clinical trials regulation in over 20 years and will help to make the UK one of the best countries in the world to conduct clinical research for patients and researchers. Please find MHRA press release here.

In addition to drafting a new legislation, the MHRA is planning to prepare a guidance accompanying the legislation to ensure consistent interpretation.

National Institute for Health and Care Excellence (NICE)

NICE recommends life-changing gene therapy for children with ultra-rare genetic disorder in final draft guidance

The first and currently only gene therapy for children with an ultra-rare genetic disorder has been recommended by NICE in a final draft guidance published on the 23rd of March 2023. Children with the condition, called aromatic L-amino acid decarboxylase (AADC) deficiency, in the UK, may be now eligible for treatment with eladocagene exuparvovec.


Food and Drug Administration (FDA)

OTP Town Hall: Gene Therapy Chemistry, Manufacturing, and Controls – April 2023

On the 25th of April 2023, the FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) is hosting its next virtual town hall. This event will focus on gene therapy chemistry, manufacturing, and controls. Details on how to register can be found here.

Clinical Trials: The Patient Experience

On the 13th of April 2023, the FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) will host a public virual workshop.

The workshop will feature a panel of rare disease patients as well as caregivers and advocates share their experiences with gene therapy clinical trials. Please find further details on this event, including how to register here.

Neovascular Age-Related Macular Degeneration: Developing Drugs for Treatment

This draft guidance which is distributed for comment purposes is to provide recommendations to sponsors regarding eligibility criteria, trial design considerations, and efficacy endpoints to enhance clinical trial data quality and to foster greater efficiency in development programs for drugs for the treatment of neovascular age-related macular degeneration. Comments to be submitted by the 30th of May 2023.

Electronic Systems, Electronic Records, and Electronic Signatures in Clinical Investigations: Questions and Answers

The draft guidance provides recommendations regarding the requirements, including the requirements under 21 CFR part 11, under which FDA considers electronic systems, electronic records, and electronic signatures to be trustworthy, reliable, and generally equivalent to paper records and handwritten signatures executed on paper. This revises the draft guidance for industry issued in June 2017 entitled Use of Electronic Records and Electronic Signatures in Clinical Investigations Under 21 CFR Part 11 — Questions and Answers and, when finalized, will supersede the guidance for industry entitled Computerized Systems Used in Clinical Investigations (May 2007). Comments to be submitted by the 15th of May 2023.

Recommendations for Evaluating Donor Eligibility Using Individual Risk-Based Questions to Reduce the Risk of Human Immunodeficiency Virus Transmission by Blood and Blood Products

The Center for Biologics Evaluation and Research (CBER) has issued a draft guidance to receive comments on revised recommendations for evaluating donor eligibility using individual risk-based questions. This draft guidance, when finalized will provide blood establishments that collect blood or blood components, including Source Plasma, with FDA’s revised donor deferral recommendations for individuals with increased risk for transmitting human immunodeficiency virus (HIV) infection.

Methods and Approaches for Capturing Post-Approval Safety and Efficacy Data on Cell and Gene Therapy Products

The Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) is hosting a virtual public listening meeting on the 27th of April 2023 to solicit input on methods and approaches related to post approval safety and efficacy data for cell and gene therapy products. This event will be held on the 27th of April 2023. Please find more details including how to register here.

FDA Rare Disease Day 2023: “Intersections with Rare Diseases – A patient focused event”

Please find the recording of the FDA Rare Disease Day 2023 public event, held on the 27 February 2023 here.


International Conference on Harmonisation (ICH)

The ICH S12 Guideline reaches Step 4 of the ICH Process

The aim of the ICH S12 guideline is to provide guidance on the conduct of nonclinical biodistribution (BD) studies in the development of gene therapy (GT) products that mediate their effect by the expression (transcription or translation) of transferred genetic materials, and harmonised recommendations to facilitate the development of GT products while avoiding unnecessary use of animals, in accordance with the 3Rs (reduce/refine/replace) principles.

Public consultations

European Directorate for the Quality of Medicines (EDQM)


Consultation Period



Public consultation on Ph. Eur. rabbit pyrogen test replacement texts

End date: 31 March 2023

Public Consultation

National Institute for Health and Care Excellence (NICE)


Consultation Period



Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency

End Date: 06 April


Draft Guideline

Food and Drug Administration (FDA)

Click here to download the PDF