Regulatory news - January 2020

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


European Medicines Agency (EMA)

EMA/CHMP Publishes Q&A Document on Comparability Considerations for ATMPs

CHMP scientific advice questions often relate to the suitability of comparability proposals following changes to ATMP manufacturing processes or due to the introduction of additional manufacturing sites. The EMA/CHMP have therefore published further guidance in the form of a Q&A document.

The document aims to provide clarification for the following:

  • quality aspects (manufacturing, chemical, pharmaceutical and biological testing of the medicine);
  • non-clinical aspects (toxicological and pharmacological tests designed to show the activity of the medicine in the laboratory);
  • clinical aspects (appropriateness of studies in patients or healthy volunteers, selection of endpoints, i.e. how best to measure effects in a study, post-authorisation activities including risk management plans);
  • methodological issues (statistical tests to use, data analysis, modelling and simulation).

See here for the full Q&A document.

UK Transition Period Post-Brexit

The United Kingdom (UK) formally left the European Union (EU) on 31 January 2020. A transition period started on 1 February 2020 and is due to end on 31 December 2020.

During the transition period EU pharmaceutical law will continue to be applicable to the UK, and UK-established EMA registered SMEs can continue to access EMA SME incentives during the transition period.

The withdrawal agreement stipulates that following departure from the EU on 31 January 2020, the UK will no longer participate in EU institutions and their decision-making. Therefore, the UK will no longer be able to participate in meetings of EMA’s scientific committees, working parties or the Agency’s Management Board.

See here for the press release from the EMA.



US Food and Drug Administration (FDA)

FDA Publishes Six Final Guidance Documents and a Draft Guidance for Gene Therapies

The FDA continues to provide support for gene therapy developers finalising six guidance documents including guidance for CMC information needed for gene therapy INDs, as well as specific guidance on Gene Therapies for Haemophilia and Retinal Disorders due to the growth in research for these therapeutic areas in recent years.

“The growth of innovative research and product development in the field of gene therapy is exciting to us as physicians, scientists and regulators,” said FDA Commissioner Stephen M. Hahn, M.D. “We understand and appreciate the tremendous impact that gene therapies can have on patients by potentially reversing the debilitating trajectory of diseases. These therapies, once only conceptual, are rapidly becoming a therapeutic reality for an increasing number of patients with a wide range of diseases, including rare genetic disorders and autoimmune diseases.”

The draft guidance ‘Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations’ focuses on how the FDA will evaluate differences between gene therapy products when they are intended to treat the same disease. The FDA will consider the principle molecular structural features of the gene therapy products, which includes transgenes and vectors. The draft guidance is open for comments until 29 April 2020.

See here for the article and related guidance documents.

Public Consultations