Europe Legal and Regulatory Affairs Watchdog Update February 2023

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Alejandro Barquero, MPharm, MSc
ISCT Legal and Regulatory Affairs Committee: Europe
DanausGT Biotech Ltd.
Spain

Hind Al Belushi, PhD
ISCT Legal and Regulatory Affairs Committee: Europe
Royal Hospital
Sultanate of Oman

Committee of Advance Therapy (CAT)

Minutes of the meetings

Minutes of the meeting on 3-4 November 2022
https://www.ema.europa.eu/en/documents/minutes/minutes-cat-meeting-3-4-november-2022_en.pdf

Minutes of the meeting on 5-7 October 2022
https://www.ema.europa.eu/en/documents/minutes/minutes-cat-meeting-5-7-october-2022_en.pdf

Minutes of the meeting on 7-9 September 2022
https://www.ema.europa.eu/en/documents/minutes/minutes-cat-meeting-7-9-september-2022_en.pdf

Agendas

Agenda for the meeting on 18-19 January 2023

https://www.ema.europa.eu/en/documents/agenda/agenda-cat-meeting-18-19-january-2023_en.pdf

Meetings report

CAT quarterly highlights and approved ATMPs (November 2022 - January 2023)

https://www.ema.europa.eu/en/documents/report/cat-quarterly-highlights-approved-atmps-january-2023_en.pdf

CAT quarterly highlights and approved ATMPs (August 2022 - October 2022)

https://www.ema.europa.eu/en/documents/report/cat-quarterly-highlights-approved-atmps-october-2022_en.pdf

Updates and Regulatory News

EMA Committee for Advanced Therapies elects Ilona Reischl as its new Chair for a three-year mandate

https://www.ema.europa.eu/en/news/ema-committee-advanced-therapies-elects-ilona-reischl-its-new-chair

EMA is offering Clinical Trials Information System (CTIS) training sessions

https://www.ema.europa.eu/en/human-regulatory/research-development/clinical-trials/clinical-trials-information-system-training-support#training-and-information-events-section

EMA´s CHMP recommended granting a conditional marketing authorisation for the advanced therapy medicinal product (ATMP) Hemgenix, the first gene therapy for the treatment of severe and moderately severe Haemophilia B

https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-12-15-december-2022

Zolgensma: fatal cases of acute liver failure

https://www.ema.europa.eu/en/news/meeting-highlights-pharmacovigilance-risk-assessment-committee-prac-9-12-january-2023

Mandatory use of CTIS from 31 January 2023 for all new clinical trial applications

https://www.ema.europa.eu/en/news/mandatory-use-ctis-31-january-2023-all-new-clinical-trial-applications

Guideline on the responsibilities of the sponsor with regard to handling and shipping of investigational medicinal products for human use in accordance with Good Clinical Practice and Good Manufacturing Practice – comes into effect from 01 January 2023.

https://www.gmp-compliance.org/files/guidemgr/guideline_handling-shipping_investigational-mp_en%20(9).pdf

Priority Medicines (PRIME): List of products granted eligibility

https://www.ema.europa.eu/en/documents/chmp-annex/recommendations-eligibility-prime-scheme-adopted-chmp-meeting-12-15-december-2022_en.pdf

Ebvallo, first therapy to treat transplant patients with post-transplant lymphoproliferative disease.

The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion., recommending the granting of a marketing authorisation under exceptional circumstances for the advanced medicinal product Ebvallo, intended for the treatment of Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD).

The applicant for this medicinal product is Atara Biotherapeutics Ireland Limited.

https://www.ema.europa.eu/en/medicines/human/summaries-opinion/ebvallo

FDA Approves First Gene Therapy for the Treatment of High-Risk, Non-Muscle-Invasive Bladder Cancer - Adstiladrin

https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-high-risk-non-muscle-invasive-bladder-cancer

FDA Approves First Gene Therapy to Treat Adults with Hemophilia B - Hemgenix

https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treat-adults-hemophilia-b

EMA Quality Innovation Group - A new regulatory group, part of the European Medicines Agency, will aid innovative approaches for the development and quality control of medicines, including new technologies for EU patients.

https://www.ema.europa.eu/en/committees/working-parties-other-groups/chmp/quality-innovation-group

Guidance Documents

The ICH E19 Guideline on a selective approach to safety data collection in specific late-stage pre-approval or post-approval clinical trials. Step 4 is reached, E19 step 4 presentation:

https://database.ich.org/sites/default/files/ICH_E19_Step_4_Presentation_2022_1012.pdf

ICH guideline E8 (R1) on general considerations for clinical studies

https://www.ema.europa.eu/en/documents/scientific-guideline/ich-e-8-general-considerations-clinical-trials-step-5_en.pdf

Update - EudraLex – volume 10- Clinical Trials Regulation (EU) 536/2014- version 6.2 Questions and Answers documents

https://health.ec.europa.eu/latest-updates/update-eudralex-volume-10-clinical-trials-guidelines-questions-and-answers-document-regulation-eu-2022-09-26_en

Ethical considerations for clinical investigations of medical products involving children

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/ethical-considerations-clinical-investigations-medical-products-involving-children

Human Gene Therapy for Neurodegenerative Diseases Guidance for Industry

Human Gene Therapy for Neurodegenerative Diseases; Guidance for Industry (fda.gov)

Comparability Protocols for Post approval Changes to the Chemistry, Manufacturing, and Controls Information in an NDA, AND A, or BLA.

Comparability Protocols for Postapproval Changes to the Chemistry, Manufacturing, and Controls Information in an NDA, ANDA, or BLA | FDA

Open Consultations

ICH guideline Q5A (R2) Viral safety evaluation of biotechnology products derived from cell lines of human or animal origin. Step 3 is reached.

https://www.ema.europa.eu/en/documents/scientific-guideline/ich-q-5-r2-viral-safety-evaluation-biotechnology-products-derived-cell-lines-human-animal-origin_en.pdf

Open to 10 February 2023

ICH guideline M11, clinical electronic structured harmonised protocol (CeSHarP). Step 3 is reached.

https://database.ich.org/sites/default/files/ICH_M11_draft_Guideline_Step2_2022_0904.pdf

Open to 26 February 2023

EMA guideline on the development and data requirements of potency tests for veterinary cell-based therapy products and the relation to clinical efficacy.

https://www.ema.europa.eu/en/documents/scientific-guideline/draft-guideline-development-data-requirements-potency-tests-cell-based-therapy-products-relation_en.pdf

Open to 28 February 2023