Regulatory news - February 2019

Keep up to date with regulatory news from around the world with the Cell and Gene Therapy Catapult regulatory newsletter.


European Commission (EC)

Letter from EC on Recognition of UK Based Testing

On 21st February 2019 the EC published a letter updating their notice to marketing authorisation holders (MAHs). The updated guidance allows quality control testing to remain in the UK and thus temporary exemption from the requirement to have the batch release site in the EU27 (“the exemption”), providing MAHs have made future plans to move batch release into the EU27.

For the exemption to apply, MAHs must notify the EMA or National Competent Authority (NCA) by 29th March 2019 of their intention and set out their precise timeline for transferring the quality control testing site to the EU27. The MAH should have a plan allowing for the transfer process to be completed by the end of 2019 at the latest.

Follow this link to read the published letter.

European Medicines Agency (EMA)

EMA Opens Consultation on Guideline for Advanced Therapies in Clinical Trials

The EMA has opened a public consultation on the structure and data requirements for clinical trial application (CTA) for exploratory and confirmatory trials with advanced therapy investigational medicinal products (ATIMPs). This guideline includes sections on quality, non-clinical and clinical documentation requirements as well as aspects of development, manufacturing and quality control. Exploratory trials, including first‑in‑human trials, are the main focus of the guidance.

The EMA advocate the use of a risk-based approach for identifying the content of an investigational medicinal product dossier based on existing knowledge on the type of product and its intended use. However, it is also highlighted that adequate product characterisation is vital in ensuring data acquired from clinical studies is of value for future marketing authroisation application.

Comments on the consultation are due by 1st August 2019.

To read the full article see here and the consultation document can be accessed here.

Two Additional Countries to Benefit from EU-US Mutual Recognition Agreement for Inspections

On 7th February 2019, the FDA included Poland and Slovenia into the mutual recognition agreement between the EU and US. The agreement mutually recognises inspections of manufacturing sites for human medicines conducted in the different territories and with the addition of these two countries, the FDA will now rely on 22 Member States whos inspection results can replace their own inspections. It is planned for the agreement to be operational in all EU Member States by 15th July 2019.

Read the full article here.

United Kingdom

Medicines and Healthcare products Regulatory Agency (MHRA)

UK Offers No-Deal Brexit Guidance for Biologic Manufacturers

The MHRA has released guidance which covers the approach to be taken for biologics manufacturers in the event of a no-deal Brexit. If the UK leave the EU without an agreement in place on 29th March 2019, then from this date the National Institute for Biological Standards and Control (NIBSC) will become a stand-alone National Control Laboratory, meaning that the UK will require national certification by NIBSC before batches of biological medicines can be placed on the UK market. The UK will carry out its own batch release of biological medicines at NIBSC taking a risk based approach to laboratory testing.

For the full article see here and the NIBSC guidance is available here.


Food and Drug Administration (FDA)

CBER Guidance on Gene Therapies: What to Expect in 2019

The US FDA Centre for Biologics Evaluation and Research (CBER) has released plans for finalised guidance to be expected in 2019 for gene therapies. These guidances include:

  • Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry
  • Chemistry Manufacturing and Control Information for Human Gene Therapy Investigational New Drug Applications
  • Human Gene Therapy for Hemophilia
  • Human Gene Therapy for Retinal Disorders
  • Human Gene Therapy for Rare Diseases
  • Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus during Product Manufacure and Patient Follow-Up

For the full list of guidances CBER plans to publish in 2019, see here.

Regenerative Medicines: FDA Finalises Guidance on Expedited Programmes

The FDA’s CBER have finalised the guidance on regenerative medicine advanced therapy (RMAT) designation, which was drafted in 2017. According to the guidance, RMAT designation provides all the benefits of fast track and breakthrough therapy designation programmes including early interactions with the FDA, without the requirement to provide evidence of the therapy offering substantial improvement over other available therapies (required for a product to be eligible for breakthrough designation).

Therapies that could qualify for an RMAT designation include cell therapies, therapeutic tissue engineering products, human cell and tissue products and combination products using such therapies. For the finalised guidance document see here.

In addition, the FDA also finalised a second guidance dealing with a wide range of devices that may be used in conjunction with an RMAT. This guidance provides FDA’s current thinking regarding evaluation of devices used in the recovery, isolation, or delivery of regenerative medicines advanced therapies. See here for the full guidance document.

For more information and to read the full article, see here.

Public consultations