Regulatory news - February 2020

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


Medicines and Healthcare products Regulatory Agency (MHRA)

Case Study: Innovation: Clonal neoantigen specific tumour-infiltrating lymphocytes for cancer treatment.

The MHRA have published a case study illustrating how their close collaboration with Achilles Therapeutics has managed to take an investigational clonal neoantigen-based therapy from a concept into the clinic in less than three years obtaining MHRA approval in January 2019 to begin first-in-human trials in both lung cancer and melanoma.

The case study can be viewed here.

UK Introduces Medicines, Medical Devices Bill in Post-Brexit Overhaul

Just two weeks after Brexit, the UK government introduced a bill to update its regulatory framework for human and veterinary medicines, clinical trials and medical devices, while ensuring the UK remains an attractive market for the life sciences industry.

Once the transition period ends, the UK will no longer be able to update its regulatory framework through secondary legislation, thus the new Medicines and Medical Devices Bill 2019-2020 seeks to address this gap through introducing delegated powers. Further information on the new bill can read in the Explanatory Note.

The bill will be read for the second time in the House of Commons on 2 March.


European Medicines Agency (EMA)

Final programming document 2020-2022

The EMA has released its final programming document for 2020 to 2022. It provides a broad plan for the Agency within this period, as well as a more detailed statement of the agency's objectives for 2020.

Within this document the Agency recognizes a need to be agile and keep pace with rapid advances in science and technology, prepare for the future challenges and continue to effectively fulfil its role to safeguard public and animal health. The EMA embarked on a ‘future proofing’ exercise to strategically align activities and roles, according to the expertise and knowledge that will be needed in the future.

A full copy of the document can be found here.

New video launched: How does EMA support SMEs?

EMA addresses the unique needs of micro, small and medium-sized enterprises (or SMEs) through its SME office, which provides regulatory, financial and administrative assistance to small pharmaceutical companies.

EMA has prepared and published a short video to explain how this dedicated office helps SMEs across Europe.

The video can be viewed here.

Brexit: Consequences for Batch Release

The European QP Association's (EQPA) have clarified their understanding on the current situation pertaining to QP batch release during the transition period.

QP certification by UK QPs is valid across the EU, but in practice this might be limited to "confirmation". Since 2016 official advice to the industry has been to prepare for the UK becoming a Third Country and required all EU Marketing Authorisations (MA) with a UK batch release site to be varied in favour of a batch release site in EU. Therefore, although there may be some MAs that have still not been varied, essentially all final batch certification and subsequent release decisions for EU MAs should now be occurring within the EU. During the transition period, QPs in EU will still be able to rely on "confirmation" from UK QPs.

For further information, see here.

Annex 1: European Commission Publishes Revised Document

On 20 February, the Directorate for Health and Food Safety of the European Commission (EC) published a further draft for the revision of Annex 1 of the EU GMP Guide to Good Manufacturing Practice. This document enters a 3-month phase of commenting by concerned organisations and stakeholders.

The original draft (published for public consultation at the end of 2017) received over 6000 comments.

The current document contains a large number of changes compared to the 2017 draft and now comprises just over 50 pages divided into 11 sections:

  1. Scope - Includes additional areas (other than sterile products) where the general principles of the annex can be applied.
  2. Principle - General principles as applied to the manufacture of sterile products.
  3. Pharmaceutical Quality System (PQS) - Highlights the specific requirements of the PQS when applied to sterile products.
  4. Premises - General guidance regarding the specific needs for premises design and guidance on the qualification of premises including the use of Barrier Technology.
  5. Equipment - General guidance on the design and operation of equipment.
  6. Utilities - Guidance with regards to the special requirements of utilities such as water, gas and vacuum.
  7. Personnel - Guidance on the requirements for specific training, knowledge and skills. Also gives guidance to the qualification of personnel.
  8. Production and specific technologies - Discusses the approaches to be taken with regard to aseptic and terminal sterilization processes. Discusses approaches to sterilization of products, equipment and packaging components. It also discusses different technologies such as lyophilization and Form-Fill-Seal where specific requirements apply.
  9. Viable and non-viable environmental and process monitoring - This section differs from guidance given in section 4 in that the guidance here applies to ongoing routine monitoring regarding the design of systems and setting of action limits alert levels and reviewing trend data. The section also gives guidance on the requirements of Aseptic Process Simulation (APS).
  10. Quality control (QC) - Gives guidance on some of the specific Quality Control requirements relating to sterile products.
  11. Glossary - Explanation of specific terminology.

A complete draft of Annex 1: Manufacture of Sterile Products can be found on the EC website.



US Food and Drug Administration (FDA)

FDA Accepts for Priority Review Bristol-Myers Squibb’s BLA for Lisocabtagene Maraleucel (liso-cel) for Adult Patients with Relapsed or Refractory Large B-Cell Lymphoma

Bristol-Myers Squibb have announced the FDA granted their CAR-T treatment, called Liso-cel, priority review, setting up a decision by Aug. 17, 2020. An on-time approval, if secured, would be well ahead of the year-end deadline set out by Bristol-Myers Squibb in an agreement to pay $9 more per Celgene share as part of its takeover offer.

Liso-cel, which treats a type of lymphoma, was originally developed by biotech Juno Therapeutics before its acquisition by Celgene in 2018. An approval would make it the third CAR-T treatment to reach market after Gilead's Yescarta and Novartis' Kymriah.

Facilitating End-to-End Development of Individualized Therapeutics

The Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration (FDA) is announcing a public workshop entitled "Facilitating End-to-End Development of Individualized Therapeutics." On the 3rd March 2020.

The purpose of the public workshop is to foster development of individualized therapeutic products for the treatment of one individual or a very small number of patients, based on engineering a product aimed at the specific molecular mechanism underlying a patient’s (or small group of patients’) illness.

A live webcast of the workshop will be streamed. For more information and to register for the public workshop, see here.

Public Consultations








Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations

28 Jan 2020


29 April 2020

Draft Guidance