Fajar Dumadi, BSc
International Society for Cell & Gene Therapy (ISCT)
Canada

This report provides links to the latest updates from European-based regulatory agencies, including CAT quarterly highlights and approved ATMPs, warning about unregulated ATMPs in the EU, EMA approval of the first topical gene therapy treatment for dystrophic epidermolysis bullosa (DEB), MHRA conditional approval for obecabtagene autoleucel, and public consultations, including draft GMP guideline for ATMPs by EMA.   

Committee of Advance Therapy (CAT)

Agendas for recent meetings

• Agenda for the meeting on 19-21 February 2025 https://www.ema.europa.eu/en/documents/agenda/agenda-cat-meeting-19-21-february-2025_en.pdf
• Agenda for the meeting on 19-21 March 2025 https://www.ema.europa.eu/en/documents/agenda/agenda-cat-meeting-19-21-march-2025_en.pdf 
• Agenda for the meeting on 14-16 April 2025 https://www.ema.europa.eu/en/documents/agenda/agenda-cat-meeting-14-16-april-2025_en.pdf
• Agenda for the meeting on 14-16 May 2025 https://www.ema.europa.eu/en/documents/agenda/agenda-cat-meeting-14-16-may-2025_en.pdf

CAT quarterly highlights and approved ATMPs

• CAT quarterly highlights and approved ATMPs – February 2025 
  https://www.ema.europa.eu/en/documents/committee-report/cat-quarterly-highlights-approved-atmps-february-2025_en.pdf

Updates from Regulatory Agencies

EMA

• Leveraging the power of data for public and animal health 
  https://www.ema.europa.eu/en/news/leveraging-power-data-public-animal-health
  
  
• New treatment against Duchenne muscular dystrophy 
  https://www.ema.europa.eu/en/news/new-treatment-against-duchenne-muscular-dystrophy
  
  
• First treatment against severe thyroid eye disease 
  https://www.ema.europa.eu/en/news/first-treatment-against-severe-thyroid-eye-disease
  
  
• Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 22-25 April 2025 https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-22-25-april-2025
  
  
• Clinical Trials Information System designated as WHO primary registry 
  https://www.ema.europa.eu/en/news/clinical-trials-information-system-designated-who-primary-registry
  
  
• Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 24-27 March 2025 
  https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-24-27-march-2025
  
  
• EMA Management Board: highlights of March 2025 meeting 
  https://www.ema.europa.eu/en/news/ema-management-board-highlights-march-2025-meeting
  
  
• Unregulated advanced therapy medicinal products pose serious risks to health 
  https://www.ema.europa.eu/en/news/unregulated-advanced-therapy-medicinal-products-pose-serious-risks-health
  
  
• EMA Management Board elects new chair 
  https://www.ema.europa.eu/en/news/ema-management-board-elects-new-chair
  
  
• Combination of cystic fibrosis medicines to treat patients with rare mutations 
  https://www.ema.europa.eu/en/news/combination-cystic-fibrosis-medicines-treat-patients-rare-mutations
   
• First topical gene therapy treatment for dystrophic epidermolysis bullosa 
  https://www.ema.europa.eu/en/news/first-topical-gene-therapy-treatment-dystrophic-epidermolysis-bullosa
  
  
• Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 24-27 February 2025 
  https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-24-27-february-2025  
  
  

MHRA

• MHRA approves guselkumab for Crohn’s disease and ulcerative colitis https://www.gov.uk/government/news/mhra-approves-guselkumab-for-crohns-disease-and-ulcerative-colitis
  
  
• MHRA approves first UK treatment for congenital thrombotic thrombocytopenic purpura (cTTP) https://www.gov.uk/government/news/mhra-approves-first-uk-treatment-for-congenital-thrombotic-thrombocytopenic-purpura-cttp
  
  
• Obecabtagene autoleucel conditionally approved to treat adults with relapsed or refractory B-cell precursor acute lymphoblastic leukaemia https://www.gov.uk/government/news/obecabtagene-autoleucel-conditionally-approved-to-treat-adults-with-relapsed-or-refractory-b-cell-precursor-acute-lymphoblastic-leukaemia
  
  
• Clinical Trials regulations signed into law https://www.gov.uk/government/news/clinical-trials-regulations-signed-into-law
  
  
• Eplontersen approved to treat adults with rare inherited nerve disease or polyneuropathy https://www.gov.uk/government/news/eplontersen-approved-to-treat-adults-with-rare-inherited-nerve-disease-or-polyneuropathy
  
  
• Marstacimab approved to treat patients aged 12 years and above (weighing at least 35 kg) with haemophilia A or B https://www.gov.uk/government/news/marstacimab-approved-to-treat-patients-aged-12-years-and-above-weighing-at-least-35-kg-with-haemophilia-a-or-b
  
  
• Triple combination medicine deutivacaftor/tezacaftor/vanzacaftor approved for cystic fibrosis https://www.gov.uk/government/news/triple-combination-medicine-deutivacaftortezacaftorvanzacaftor-approved-for-cystic-fibrosis
  
  
• Lazertinib approved for use in combination with amivantamab for the treatment of adults with non-small cell lung cancer https://www.gov.uk/government/news/lazertinib-approved-for-use-in-combination-with-amivantamab-for-the-treatment-of-adults-with-non-small-cell-lung-cancer
  
  
• Nemolizumab approved to treat prurigo nodularis and atopic dermatitis (eczema) for patients in the UK https://www.gov.uk/government/news/nemolizumab-approved-to-treat-prurigo-nodularis-and-atopic-dermatitis-eczema-for-patients-in-the-uk
  
  
• Trastuzumab deruxtecan approved to treat adults with HER2-positive cancer that has spread or cannot be removed by surgery https://www.gov.uk/government/news/trastuzumab-deruxtecan-approved-to-treat-adults-with-her2-positive-cancer-that-has-spread-or-cannot-be-removed-by-surgery
  
  
• Lawrence Tallon begins role as new MHRA CEO https://www.gov.uk/government/news/lawrence-tallon-begins-role-as-new-mhra-ceo

Public Consultations

• MHRA – Consultation - Draft guideline on the use of external control arms based on real-world data to support regulatory decisions
  Consultation ends on 30 June 2025
  https://www.gov.uk/government/consultations/mhra-draft-guideline-on-the-use-of-external-control-arms-based-on-real-world-data-to-support-regulatory-decisions
  
  
• EMA - Consultation - Concept paper on the revision of Part IV guidelines on good manufacturing practice specific to advanced therapy medicinal products
  Consultation ends on 8 July 2025
  https://www.ema.europa.eu/en/documents/scientific-guideline/concept-paper-revision-part-iv-guidelines-good-manufacturing-practice-specific-advanced-therapy-medicinal-products_en.pdf
  
  
• EMA – Consultation - Concept paper on the revision of the guideline on the evaluation of anticancer medicinal products and appendices - Revision
  Consultation ends on 31 July 2025
  https://www.ema.europa.eu/en/documents/scientific-guideline/concept-paper-revision-guideline-evaluation-anticancer-medicinal-products-appendices-revision-7_en.pdf

Events

Past events

• ACT EU workshop on ICH E6 R3 (principles and Annex 1) – 19-20 February 2025
  https://www.ema.europa.eu/en/events/act-eu-workshop-ich-e6-r3-principles-annex-1
  
  
• EMA workshop on the challenges in drug development, regulation and clinical practice for immunoglobulins – 05 March 2025
  https://www.ema.europa.eu/en/events/ema-workshop-challenges-drug-development-regulation-clinical-practice-immunoglobulins
  
  
• Finding clinical trials with the ACT EU Trial Map – 07 March 2025
  https://www.ema.europa.eu/en/events/finding-clinical-trials-act-eu-trial-map
  
  
• Cancer Medicines Forum – 31 March 2025
  https://www.ema.europa.eu/en/events/cancer-medicines-forum-march-2025
  
  
• European Medicines Agency (EMA) Patients' and Consumers' (PCWP) and Healthcare Professionals' (HCPWP) Working Parties joint meeting – 1-2 April 2025 
  https://www.ema.europa.eu/en/events/european-medicines-agency-ema-patients-consumers-pcwp-healthcare-professionals-hcpwp-working-parties-joint-meeting-3
  
  
• Enpr-EMA Coordinating Group meeting – 29 April 2025
  https://www.ema.europa.eu/en/events/enpr-ema-coordinating-group-meeting-april-2025
  
  
• EMA Open Door Day – 09 May 2025
  https://www.ema.europa.eu/en/events/ema-open-door-day
  
  
• Press Release: ICH Assembly Meeting, Madrid, Spain – 21 May 2025
  https://www.ich.org/pressrelease/press-release-ich-assembly-meeting-madrid-spain-may-2025   

Upcoming events

• EMA workshop on primary efficacy endpoints for antivirals and monoclonal antibodies intended for the treatment of COVID-19 and influenza – 05 June 2025 https://www.ema.europa.eu/en/events/ema-workshop-primary-efficacy-endpoints-antivirals-monoclonal-antibodies-intended-treatment-covid-19-influenza
  
  
• Workshop on the use of Bayesian statistics in clinical development – 17 June 2025
  https://www.ema.europa.eu/en/events/workshop-use-bayesian-statistics-clinical-development
  
  
• Fourteenth meeting of the industry stakeholder platform on the operation of the centralised procedure for human medicines – 23 June 2025
  https://www.ema.europa.eu/en/events/fourteenth-meeting-industry-stakeholder-platform-operation-centralised-procedure-human-medicines
  
  
• EMA’s 30th anniversary scientific conference - Medicines, regulation and the future – 25 June 2025
  https://www.ema.europa.eu/en/events/emas-30th-anniversary-scientific-conference-medicines-regulation-future
  
  
• Training session for patients, consumers and healthcare professionals involved in medicine regulatory activities – 02 July 2025
  https://www.ema.europa.eu/en/events/training-session-patients-consumers-healthcare-professionals-involved-medicine-regulatory-activities-july-2025