Regulatory Round-up - April 2024

UNITED KINGDOM

Medicines and Healthcare products Regulatory Agency (MHRA)

MHRA launch Regulatory Connect, the new portal for tracking licensing applications

The MHRA have launched RegulatoryConnect portal, a service that provides the capability to track Product and Process licensing applications and view live authorisation details. The RegulatoryConnect portal will enable industry users to:

• Use the applications page to track the status of an application
• Use the Current Granted View page to view live authorisation details, including status, key data and documents held against existing licences.

Further functionality and features will be added to RegulatoryConnect later in the year, including the ability to submit applications and variations to the MHRA through the service. Please find the RegulatoryConnect guidance including video here.

MHRA combined review process for clinical trial authorisation now accepts applications for combined medicine and device trials

MHRA has provided updated guidance on the process for using the Integrated Research Application System (IRAS) for Combined review authorisation of combined clinical trials for an investigational medicinal product and an investigational medical device.

British Pharmacopoeia (BP)

New ATMP guidance: T Cell and NK Cell Characterisation Assays

BP has issued a best practice guidance titled “Advanced Therapy Medicinal Products (ATMP) T Cell and NK Cell Characterisation Assays” which offers a phase appropriate validation tool to assist cell therapy programme development. The document can be downloaded here.

New members sought for the ATMP Working Party

The British Pharmacopoeia (BP) Commission is currently looking to appoint new professional members for two new task groups focussing on providing non-mandatory best practice guidance on the topics of “Encapsidated DNA characterisation” and “Primary protein sequence modifications of encapsidated DNA”. The application process closes on Friday the 17^th May 2024. The application can be accessed here.

Genomic England

Genomics England Research Summit- 9 July 2024

This one-day, in-person event aims to drive conversations on the present and future of genomic research and medicine. It will cover advances in the diagnosis of rare conditions and cancer, pharmacogenomics, therapeutic innovations and trials, and emerging technologies. Please find registration details here.

EUROPE

European Commission (EC)

New general chapter on phage therapy medicinal products (5.31) adopted and pre-published on the EDQM website

Phage therapy medicinal products (5.31) was adopted by the European Pharmacopoeia Commission (EPC) in March 2024, making this the pharmacopoeial text to be published on phate therapy. The EPC has pre-published the text on EDQM pending its publication in Supplement 11.6 (July 2024) which can be found here.

European Medicines Agency (EMA)

Adjusted fees for applications to EMA from 1 April 2024

EMA published full details of the revised fees charged for each of its services following the adoption of the Commission Regulation (EU) 2024/848. Fees payable to EMA can be found here.

Pharmacovigilance Risk Assessment Committee (PRAC) reviews new safety updates for several ATMPs

Minutes of the 5-8 February 2024 PRAC meeting included the Periodic safety update reports (PSURs) and Risk management plans (RPMs) for TECARTUS and LUXTURNA and the Risk Management Plans for CARVYKTI, KYMRIAH which are now available here.

Revised HMA/EMA guidance on personal data and commercially confidential information within marketing authorisation application (MAA) dossiers

The Heads of Medicines Agencies (HMA) and EMA have issued a revised draft of the 2012 adopted guidance document defining the common approach on what should be considered as personal data (PD) and commercially confidential information (CCI) in the MAA dossier of medicinal products for human use. This guidance document applies to information/documents on medicinal products for human use, for which the procedure has been finalised under the national, mutual recognition, decentralised and centralised procedures. Comments can be submitted until 28 June 2024.

Joint HMA/EMA Big Data Steering Group Workshop on real world evidence (RWE) methods: Harnessing Real-World Data (RWD) for Regulatory Use

EMA and the Heads of Medicines Agencies (HMA) will hold a Big Data Steering Group

Workshop on RWE methods on the 14^th of June 2024. The agenda and further information is here.

Information and Q&A session on updated centrally authorised products (CAPs) in web-based electronic application form (eAF)

EMA is hosting an Information and Q&A session to explain and showcase the changes in the product data in the web-based Human Variations eAF. This online event will take place on 7^th May 2024. Please find further information including how to register here.

Webinars on Regulatory Procedure Management (RPM) for Product Lifecycle Management (PLM)on IRIS for Industry and Network stakeholders

The RPM for PLM team is hosting an Industry webinar for Marketing Authorisation Holders (MAH) on 13^th June 2024, and a Network stakeholder webinar for network stakeholders on 18^th June 2024, to update on the progress of RPM plans, insights into the strategic direction, key milestones and implementation approach for the coming months.

EMA call for Proposals: “Medicines regulatory systems strengthening in Sub-Saharan Africa”

The EMA is supporting the development of regulatory systems on the African continent and the operationalisation of the African Medicines Agency (AMA) to strengthen the regulatory environment, enhance the capacity of the AMA, promote the adoption of common standards and guidelines, and facilitate joint assessments of medicines. The EMA has published a call for proposals from the European Medicines Regulatory Network (EMRN) to contribute to national and regional systems strengthening in Sub-Saharan Africa. Please find further information here.

USA

Food and Drug Administration (FDA)

BLA Approval: BEQVEZ for haemophilia B from Pfizer

FDA has approved fidanacogene elaparvovec-dzkt, indicated for the treatment of adults with moderate to severe haemophilia B (congenital factor IX deficiency) who: currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and, do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid.

BLA Approval of New Indication: ABECMA for multiple myeloma from Cellgene

FDA has approved the indication for idecabtagene vicleucel to include the treatment of adult patients with relapsed or refractory multiple myeloma after two or more prior lines of therapy including an immunomodulatory agent, a proteasome inhibitor, and an anti- CD38 monoclonal antibody.

Guidance for Industry: Providing Regulatory Submissions in Electronic Format: IND Safety Reports

FDA has issued a final guidance for industry entitled “Providing Regulatory Submissions in Electronic Format: IND Safety Reports which describes the electronic format sponsors will be required to use when they electronically submit IND safety reports to CDER and CBER for serious and unexpected suspected adverse reactions, as required under 21 CFR 312.32(c)(1)(i). This guidance finalizes the draft guidance of the same name published on October 30, 2019 (84 FR 58158).

FDA CBER Webinar: Considerations for the Development of CAR T Cell Products

A recording of the FDA CBER webinar: Considerations for the Development of CAR T Cell Products, held on 7^th March 2024 is now available here.

FDA Requires Boxed Warning for T cell Malignancies Following Treatment with BCMA-Directed or CD19-Directed Autologous CAR T cell Immunotherapies

FDA has determined that the serious risk of T cell malignancies, as initially reported in a November 2023 safety communication, is applicable to all currently approved BCMA-directed and CD19-directed genetically modified autologous CAR T cell immunotherapies. FDA has therefore concluded that changes to the Boxed Warning are warranted to highlight the serious risk of T cell malignancies for these therapies. Please find further information here.

OTP Town Hall: CMC Readiness for Gene Therapy BLAs

The FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) is hosting its next virtual town hall on 4^th June 2024, to answer stakeholder questions regarding the chemistry, manufacturing, and controls (CMC) information submitted with biologics license applications (BLAs) for gene therapy products. Experts from OTP’s Office of Gene Therapy CMC will be on hand to answer questions. Please find further information including how to register here.

Public consultations

European Medicines Agency (EMA)

Food and Drug Administration (FDA)

Therapeutic Goods Administration (TGA)

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