Regulatory Round-up - March 2025

UNITED KINGDOM

Medicines and Healthcare products Regulatory Agency (MHRA)

MHRA implements new regulatory framework on decentralised manufacture

MHRA has provided information on the new regulations for decentralised manufacture which come into effect on 23 July 2025. Point of care and modular manufacture are collectively called decentralised manufacture. These processes allow medicinal products to be made at or close to a patient’s location for administration of the medicine. Please find details including a webinar recording of these new MHRA regulations held on 28 January 2025 here. Guidance is expected to be published in early summer 2025.

MHRA releases safety communications guidance for medicines, medical devices and other healthcare products

MHRA has published a guidance and an infographic to provide information to the public, patients and healthcare professionals on the various types of safety communications (for both medicines and medical devices) including what they are and who they are targeted at, as well as information on the decision making involved in issuing safety communications.

A National Institute for Health and Care Excellence (NICE)

NICE recommends lisocabtagene maraleucel for treating people with large B-cell lymphoma 

NICE has released its final guidance recommending Breyanzi (also known as lisocabtagene maraleucel or liso-cel) as a treatment option for individuals with large B-cell lymphoma whose cancer has either not responded to initial treatment or relapsed within 12 months. This positive recommendation marks a reversal from an initial negative draft recommendation in October 2024 following an improved commercial arrangement offered by Bristol Myers Squibb to the NHS. Breyanzi, an innovative one-time cell therapy, is now accessible through routine commissioning. Please find further information here.

Human Tissue Authority (HTA)

HTA publishes operational data

The HTA has published data about licenses, inspections and enquiries which covers activity between 1 April 2024 to 30 Sep 2024. The research sector has the highest number of active licences for the removal and storage of tissue from the living and deceased. Please find further information here.

EUROPE

European Commission (EC)

International Coalition of Medicines Regulatory Authorities (ICMRA) publishes collaborative hybrid inspection pilot (CHIP) summary report

Post-approval Chemistry, Manufacturing, and Controls (CMC) changes are critical to continued global availability of medicines to patients. Depending on the nature of the change, supporting data may need to be evaluated by the relevant regulatory authorities before implementation. However, each regional authority may have different data requirements, assessment approaches and approval timelines and may require separate inspections of the same manufacturing site (of the same changes) by different regulatory authorities. As a result, it may take years before a single modification to an approved medicinal product can be implemented globally, leading to logistical challenges and the necessity for manufacturers to maintain multiple product versions. To address the regulatory complexity, the ICMRA launched the CHIP in 2021. The initiative allowed manufacturers to submit a single CMC dossier which could undergo simultaneous hybrid inspections by multiple regulatory authorities. Please find the report outlining the outcome of this pilot.

European Directorate for the Quality of Medicines (EDQM)

EDQM On Air – The evolution of biologicals in the European Pharmacopoeia

EDQM On Air has just released a new episode on biological medicinal products discussing the history and evolution of these products, how they are at the cutting-edge of pharmaceutical science and have contributed to major breakthroughs in the treatment of smallpox, diabetes and haemophilia, as well as to promising innovations such as CRISPR-Cas9 technology and CAR T-cell therapies, making us stronger in the fight against disease. The episode can be listened to here.

European Medicines Agency (EMA)

ACT EU launches new clinical trial map in the EU

The Accelerating Clinical Trials in the European Union (ACT EU) initiative has launched a new clinical trial map which is now accessible from the public website of the Clinical Trials Information System. The map shows real-time information on clinical trials being conducted at EU trial sites and can also be filtered by therapeutic indication and country.

EMA recommends first topical gene therapy for dystrophic epidermolysis bullosa

EMA has recommended granting a marketing authorisation in the EU for Vyjuvek (beremagene geperpavec) to treat wounds in patients of all ages with dystrophic epidermolysis bullosa (DEB). DEB is a serious, rare, genetic disease caused by mutations in the collagen type VII alpha 1 chain (COL7A1) gene. It is characterised by skin fragility, blister formation, small white bumps (milia) and scarring, and is usually present at birth. Please find further information here.

EudraVigilance releases 2024 annual report for the European Parliament, the Council and the Commission

EudraVigilance, the European system for managing and analysing information on suspected adverse drug reaction reports, is the tool that the EMA and national competent authorities use to monitor the safety of all investigational and authorised medicines in the EU. It complements the routine benefit-risk re-evaluation of authorised medicines performed by the EMA’s Pharmacovigilance Risk Assessment Committee through the assessment of periodic safety update reports and risk management plans. Please find the annual EudraVigilance report for 2024 here.

Drug Information Association (DIA)

EU regulators report steady progress in real-world data (RWD) acceptance at DIA Europe 2025 conference

The DIA European annual meeting took place in Basel, Switzerland from 18-20 March 2025. During a panel discussion, representatives from the EMA, Germany’s Federal Institute for Drugs and Medical Devices (BfArM) and the pharmaceutical industry (specifically Sanofi) discussed the progress made in using RWD for regulatory procedures and the importance of RWD in informing decision making for new drug approvals as well as improving drug safety data collection. Please find further information here.

USA

Food and Drug Administration (FDA)

FDA issues approval of Encelto

The FDA has approved revakinagene taroretcel-lwey (brand name, Encelto) for the treatment of adults with idiopathic Macular Telangiectasia type 2 (MacTel). It is the first FDA-approved treatment for Macular Telangiectasia type 2, a neurodegenerative retinal disease that affects the macula, causing a gradual deterioration in central vision. It is characterised by changes in the macular capillary network and atrophy of the neurosensory retina. Encelto employs encapsulated cell therapy technology to deliver continuous doses of ciliary neurotrophic factor to the retina. Please find further information here.

FDA issues joint guidance on Institutional Review Board (IRB) written procedures

The Department of Health and Human Services Office for Human Research Protections and the FDA have issued a final guidance on IRB Written Procedures to enhance human subject protection and reduce regulatory burden. This is part of efforts to harmonise the agencies' regulatory requirements and guidance for human subject research. This guidance aims to assist staff at institutions and IRBs who are responsible for preparing and maintaining written procedures to ultimately protect the rights and welfare of human subjects.

INTERNATIONAL

International Conference on Harmonisation (ICH)

ICH M11 updated Technical Specification document and Step 2 presentation now available

The ICH M11 (on clinical electronic structured harmonised protocol) updated version of the Technical Specification (TS) document, which incorporates comments received during the first public consultation, has reached Step 2b of the ICH process and now enters the second round of public consultation. The updated template will not open for public comment. The M11 Expert Working Group has also prepared a Step 2 informational presentation which clarifies information on the TS that was included in the initial Step 2 presentation, ensuring alignment with the updated TS. The updated TS Step 2 document, template and presentation are available for download on the M11 page.

Federal Commission for the Protection Against Health Risks (COFEPRIS)

COFEPRIS issues updated Good Manufacturing Practice (GMP) guidelines

Mexico’s COFEPRIS has issued GMP guidelines (N.B. this webpage can be translated from Spanish). These guidelines aim to facilitate the recognition of international regulations and reduce administrative barriers for the pharmaceutical industry.

Under the new guidelines, Mexico will: 

• accept GMP certifications issued by regulatory agencies in Brazil, Argentina, Canada, Colombia, Chile, Cuba and the US,
• implement the full digitization of processes,
• align with the international Pharmaceutical Inspection Cooperation Scheme (PIC/S) and the United States-Mexico-Canada Agreement (USMCA),
• recognise international standards from WHO as well as ISO 13485:2016 certification for medical devices.

Pharmaceuticals and Medical Devices Agency (PMDA)

PMDA notice

PMDA has opened its first office in the US, as part of a push to support drug developers that want to launch medicines in Japan.

PMDA Washington D.C. Office is now offering general consultation services to assist companies and related parties understand Japanese regulatory process and procedures on reviews and post-marketing safety measures. PMDAprovides the followingservices:

• Information on regulations or procedures under the Act on Securing Quality, Efficacy and Safety of Products including Pharmaceuticals and Medical Devices (Act No. 145 of August 10, 1960) of Japan
• A general explanation of basic matters for practical regulatory application of new seeds for drugs, medical devices, and regenerative medicine products
• An explanation of PMDA's services, and support in selecting one or more consultation categories provided by PMDA's review team in Japan

Please find further information here.

Public consultations

Medicines and Healthcare products Regulatory Agency (MHRA)  

European Medicines Agency (EMA) 

Food and Drug Administration (FDA)  

Click here to download the PDF