News Hub

EU LRA Watchdog - June 2024


Alejandro Barquero, MPharm, MSc
DanausGT Biotechnology Ltd.
Madrid, Spain

Committee of Advance Therapy (CAT)
Minutes of the meetings
CAT quarterly highlights and approved ATMPs
Agendas for upcoming meetings
Updates from Regulatory Agencies
Updates from the EMA
  • New gene therapy treatment for haemophilia B. Durveqtix received support through the PRIority Medicines (PRIME) scheme due to its potential to improve patients’ quality of life.

    EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Durveqtix (fidanacogene elaparvovec) to treat severe and moderately severe haemophilia B in adults who do not have factor IX inhibitors (auto-antibodies produced by the immune system against factor IX replacement medicines) and who have no detectable antibodies to variant adeno-associated virus serotype Rh74 (AAVRh74var) – May 31st 

Clinical Trial Information System (CTIS) updates: 

Clinical Trials Information System (CTIS) sponsor end user training programme – September, October & November 2024 events

  • Sep - Clinical Trials Information System (CTIS): Walk-in clinic - September 2024
Updates from the HMA
  • New European Pharmacopoeia Commission approach to gene therapy – March 2024
At its 178th session held on 19-20 March 2024, the EPC adopted the general monograph Gene therapy medicinal products for human use (1386) and the accompanying general chapter Additional information on gene therapy medicinal products for human use (5.34), which will replace general chapter 5.14.
In addition to a general requirements section containing provisions on the production of all GTMPs and specific requirements for recombinant vectors and genetically modified cells for human use, general monograph 3186 contains three individual sections describing additional requirements for the classes of GTMPs currently approved in Europe:
  • genetically modified human autologous cells modified by integrating retroviral or lentiviral vectors;
  • adeno-associated virus vectors for human use;
  • oncolytic herpes simplex virus for human use.
The accompanying general chapter 5.34 includes recommendations on product classes that are not yet on the European market to assist users and contains:
  • the revised remaining sections of chapter 5.14, covering:
o plasmid vectors for human use;
o adenovirus vectors for human use;
o poxvirus vectors for human use;
o retroviridae-derived vectors for human use.
  • a newly drafted section on genetically modified bacterial cells for human use.
In addition, general chapter 5.2.12. Raw material of biological origin for the production of cell-based and gene therapy medicinal products has also been revised to align it with this new approach.
The new general monograph provides a common framework of requirements for GTMPs, while offering the level of built-in flexibility judged necessary for these products, given the rapidly evolving nature of the field. This new approach marks an important milestone on the path towards a standardised way of controlling GTMPs and, together with the extensive additional guidance provided to users in the new general chapter 5.34, contributes significantly to enhancing public health in Europe and beyond.
Public Consultations
Closed Consultations
Other Updates