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EU LRA Watchdog - June 2022

  

Europe Legal and Regulatory Affairs
Watchdog Update
June 2022


By:

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

Committee for Advanced Therapies (CAT)

CAT is the European Medicines Agency's (EMA) committee responsible for assessing the quality, safety and efficacy of advanced therapy medicinal products (ATMPs) and following scientific developments in the field.

Minutes of the meetings

Minutes of the meeting on 11-13 April 2022
https://www.ema.europa.eu/en/documents/minutes/minutes-cat-meeting-11-13-april-2022_en.pdf

Minutes of the meeting on 16-18 March 2022
https://www.ema.europa.eu/en/documents/minutes/minutes-cat-meeting-16-18-march-2022_en.pdf

Minutes of the meeting on 16-17 February 2022
https://www.ema.europa.eu/en/documents/minutes/minutes-cat-meeting-16-17-february-2022_en.pdf

Minutes of the meeting on 19-21 January 2022
https://www.ema.europa.eu/en/documents/minutes/minutes-cat-meeting-19-21-january-2022_en.pdf


Agendas

Agenda for the meeting on 11-13 May 2022
https://www.ema.europa.eu/en/documents/agenda/agenda-cat-agenda-11-13-may-2022-meeting_en.pdf

 

Monthly report

https://www.ema.europa.eu/en/documents/report/cat-monthly-report-application-procedures-guidelines-related-documents-advanced-therapies-december_en.pdf



Updates and regulatory news

FDA guidance documents

FDA  issues a guidance document providing considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products.
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-development-chimeric-antigen-receptor-car-t-cell-products?utm_medium=email&utm_source=govdelivery

FDA guidance documents on Human Gene Therapy Products Incorporating Human Genome Editing.
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/human-gene-therapy-products-incorporating-human-genome-editing?utm_medium=email&utm_source=govdelivery

FDA Guidance documents (Recommendations to Reduce the Possible Risk of Transmission of Creutzfeldt-Jakob Disease and Variant Creutzfeldt-Jakob Disease by Blood and Blood Components Guidance for Industry)
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/recommendations-reduce-possible-risk-transmission-creutzfeldt-jakob-disease-and-variant-creutzfeldt?utm_medium=email&utm_source=govdelivery 

 

Regulatory News

EMA has recommended granting a marketing authorisation in the European Union (EU) for Upstaza (eladocagene exuparvovec), a therapy for the treatment of adult and paediatric patients with severe aromatic L-amino acid decarboxylase (AADC) deficiency with a genetically confirmed diagnosis.
https://www.ema.europa.eu/en/news/first-therapy-treat-rare-genetic-nervous-system-disorder-aadc-deficiency

Step 4 Training presentation for E8(R1). The ICH E8(R1) General Considerations for Clinical Studies was finalised in October 2021 and sets out general principles on the conduct of clinical studies. 
https://database.ich.org/sites/default/files/ICHE8%28R1%29_Step4Presentation_2022_0408.pdf

Cell and gene therapy consulting firm (Dark Horse) submits draft AAV purity guidance to FDA. The proposed DRAFT Guidance for FDA Consideration: Testing of Adeno- Associated Viral (AAV) Vector-Based Human Gene Therapy Products for Empty Capsids During Product Manufacture.
https://darkhorseconsultinggroup.com/wp-content/uploads/2022/05/DHC_Proposed-DRAFT-Guidance-for-FDA-Consideration.pdf

 

EU-LRA highlights the Triton X-100 ban affecting medicinal products and medical devices in the EU 

Substances under the trade name ‘Triton X-100’ (CAS: 9002-93-1), covered by the group entry “4-(1,1,3,3-tetramethylbutyl)phenol, ethoxylated” (4-tert-OPnEO), have been identified in the EU as substances of very high concern (SVHC) due to their endocrine disrupting properties. The OPE substances (including Triton X-100) were placed in the Candidate list (Entry) and later in the Authorisation list (Annex XIV, Entry 42, on 13 June 2017) with a latest application date on 4 July 2019 and a sunset date on 4 January 2021. Concerning medicinal products and medical devices used for the diagnosis, treatment or prevention of COVID-19 the latest application date is 22 June 2022 and the sunset date 22 December 2023.  After the sunset date the use of Triton X-100 is no longer allowed in the EU/EEA and affected companies should approach as soon as possible their enforcement authorities and start preparing their application. There are some generic exemptions from the authorization requirement listed on the EC webpage (Authorisation - ECHA (europa.eu), however use of Triton X-100 and other OPEs in medicinal products like vaccines and gene therapy products is no longer accepted without authorization. The authorization applications submitted for use of OPEs (including medicinal products) are available at Adopted opinions and previous consultations on applications for authorisation - ECHA (europa.eu). It is highly recommended that the use of OPE in manufacture of medicinal products is replaced with a safer alternative substance or technology.”

 

Open consultations

ICH guideline Q2(R2) on validation of analytical procedures (Step 2b). Q2(R2) provides guidance and recommendations on how to  derive and evaluate the various validation tests for each analytical procedure.
https://www.ema.europa.eu/en/documents/scientific-guideline/ich-guideline-q2r2-validation-analytical-procedures-step-2b_en.pdf

Open to 31 July 2022.

ICH guideline Q14 on analytical procedure development (Step 2b).

(This guideline describes science and risk-based approaches for developing and maintaining analytical 4  procedures suitable for the assessment of the quality of drug substances and drug products)
https://www.ema.europa.eu/en/documents/scientific-guideline/ich-guideline-q14-analytical-procedure-development-step-2b_en.pdf

Open to 31 July 2022.

ICH guideline E11A on pediatric extrapolation Step 2b.
https://www.ema.europa.eu/en/documents/scientific-guideline/draft-ich-guideline-e11a-pediatric-extrapolation-step-2b_en.pdf 

Open to O6 August 2022.


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