Regulatory Round-up - January 2023

EUROPE

European Medicines Agency (EMA)

Sponsor Responsibilities with regard to Handling and Shipping of IMPs

The “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” came into effect on the 1^st of January 2023. This guideline lays down the principles for the two-step release and shipping of Investigational Medicinal Products (IMPs) by the Qualified Person (QP) and the Sponsor in accordance with good clinical practice (GCP) and good manufacturing practice (GMP).

End of the Transition period from Clinical Trials Directive (2001/20/EC) to Clinical Trial Regulation (EU) No 536/2014.

From the 31^st of January 2023, the one year transition period from the Clinical Trials Directive (2001/20/EC) to Clinical Trial Regulation (EU) No 536/2014 will end. All new clinical trial Applications (CTAs) in the EU must be submitted in accordance with the Clinical Trial Regulation (CTR) using the Clinical Trials Information System (CTIS). Please find further details here.

EMA has published a sponsor handbook to help sponsors using the CTIS which is continuously being updated. The revised Version 3.01 sponsor handbook provides clarifications on the transition period timelines in addition to the previous version which provided key guidance, technical information, recommendations and references for managing clinical trial transition to the CTR.

Zolgensma: fatal cases of acute liver failure

Fatal cases of acute liver failure were recently reported in patients treated with Zolgensma (onasemnogene abeparvovec), a gene therapy medicine for the treatment of spinal muscular atrophy (SMA). A direct healthcare professional communication (DHPC) will be issued by the EMA in due course. Please find details here.

Clinical Trials Information System (CTIS) training session

The EMA and the European Medicines Regulatory Network are holding training events and information sessions to support Clinical Trails Information System (CTIS) users. Sessions will be held in February, May and June 2023. A list of the upcoming events and recordings from previous training sessions can be found on the EMA’s website.

Priority Medicines (PRIME): List of products granted eligibility

During its December 2022 meeting, the CHMP reviewed 3 recommendations for ATMPs with respect to their eligibility to PRIME: 1 was granted and 2 were denied. More information can be found here.

Hemgenix receives positive CHMP opinion.

The Committee for Medicinal Products for Human Use (CHMP) has recommended granting a conditional marketing authorisation for the advanced therapy medicinal product (ATMP), Hemgenix* (etranacogene dezaparvovec), the first gene therapy for the treatment of severe and moderately severe Haemophilia B.

UNITED KINGDOM

Medicines and Healthcare products Regulatory Agency (MHRA)

Consultation outcome

Government response to consultation on proposals to support the regulation of medicines manufactured at the Point of Care

The MHRA led a joint public consultation with the Northern Ireland Department of Health in accordance with Section 45(1) of the Medicines and Medical Devices Act 2021, seeking views on a legislative proposal to introduce a new regulatory framework for the manufacture and supply of point of care (POC) products.

Comments were submitted by a variety of stakeholders from across the UK and internationally.

The new regulatory framework will be based on and link into the current regulatory systems for medicines approvals, clinical trials, evaluation of regulatory compliance at manufacturing sites and safety monitoring. It will support increased manufacture and supply of new products manufactured at POC whilst ensuring that these products keep equivalent levels of safety, quality and efficacy currently in place for medicinal products manufactured in factory-based locations.

Human Tissue Authority (HTA)

HTA publish licence fee levels for the 2023/24 business year

On the 3^rd of January 2023 HTA published licence fee levels for the 2023/24 business year. Please find further details here.

USA

Food and Drug Administration (FDA)

Dosage and Administration Section of Labeling for Human Prescription Drug and Biological Products — Content and Format

In January 2023, the FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) issued the ‘Dosage and Administration Section of Labeling for Human Prescription Drug and Biological Products--Content and Format’ draft guidance to assist applicants in developing the DOSAGE AND ADMINISTRATION section of labeling. This guidance is replacing the existing guidance issued on March 29, 2010. Comments can be submitted by 03/14/2023. Details on how to submit comments can be found here.

FDA Modernization Act 2.0

In late December 2022 President Joe Biden signed into legislation the FDA Modernization Act 2.0. This bill authorizes the use of certain alternatives to animal testing, including cell-based assays and computer models, to obtain an exemption from the Food and Drug Administration to investigate the safety and effectiveness of a drug. The bill also removes a requirement to use animal studies as part of the process to obtain a license for a biological product that is biosimilar or interchangeable with another biological product.

National Institutes of Health (NIH)

NIH study shows genotype-first approach uncovers new links to genetic conditions.

National Institutes of Health researchers have published an assessment of 13 studies that took a genotype-first approach to patient care which involves selecting patients with specific genomic variants and then studying their traits and symptoms. This study found that the genotype-first approach leads to the discovery of new links to genetic conditions. More information on this study can be found here.

Public consultations

EUROPEAN MEDICINES AGENCY (EMA)

Medicines and Healthcare products Regulatory Agency (MHRA)

Food and Drug Administration (FDA)

International Conference on Harmonisation (ICH)

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