Review of Developments in GMP and the Regulation of Medicines February 2025

EJPPS vol 30.1B

EJPPSVol 30.1B.pdf

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INTRODUCTION

This month reported issues have come from the UK, EU, USA, Swiss, WHO and PIC/s regulatory authorities.

The topics covered in this edition of the “Update” include:

UK

Medicines and Healthcare products Regulatory Agency (MHRA)

·         New MHRA Chair outlines priorities

·         New, integrated Innovative Licensing and Access Pathway

·         Pharmacovigilance updates

·         Guidance on new Medical Devices Post-Market Surveillance requirements

·         Criminals denied £7.5m in profits from the illegal trade in medicines

·         MHRA Windsor Framework hub

EU

European Medicines Agency (EMA)

·         Guidance on GMP & GDP – Q&A

·         Q&A to Stakeholders on the implications of Regulation (EU) 2023/1182 for centrally authorised medicinal products for human use.

·         Write PMS API implementation Guide

·         Guideline on quality aspects of mRNA vaccines for veterinary use

·         Checklist for Initial Notifications for Parallel Distribution.

·         European shortages monitoring Platform fully operational for monitoring of shortages in the EU

·         One Health Approach

·         EU agencies unite to tackle azole fungicide resistance in Aspergillus fungi

·         Product Management Service (PMS) webinar on Product User Interface (PUI) edit functionalities for industry users

·         Qualification of non-mutagenic impurities - Scientific guideline

·         Clinical Trials Regulation becomes fully applicable

The European Directorate for the Quality of Medicines & HealthCare (EDQM)

·         Certification monthly report

·         Recommendation CM/Rec(2024)8 on the family approach to discuss deceased organ and tissue donation

·         Publication of BSP establishment study for hepatitis A virus coating reagent BRR

·         Implementation of the European Pharmacopoeia Supplement 11.8 – Notification for CEP holders

·         How CEP holders can avoid the rejection of notifications

·         Significant milestone in the elimination of animal tests from the Ph. Eur.

·         Three new Ph. Eur. general chapters for plastic materials for containers

·         Reference Standards.

·         First three general texts on MRNA vaccines

·         Pharmeuropa 37.1 just released

·         Amiodarone capsules monograph published in European Paediatric Formulary

Nordic Countries

·         Common nordic packages for better availability

Ireland

The Health Products Regulatory Authority (HPRA)

·         Joint packs with other member states

USA

The US Food and Drug Administration (USFDA)

·         Considerations for the Use of Artificial Intelligence To Support Regulatory Decision-Making for Drug and Biological Products

·         Interim Policy on Compounding Using Bulk Drug Substances Under Section 503A & 503B of the Federal Food, Drug, and Cosmetic Act

·         Considerations for Complying with 21 CFR 211.110

International

Switzerland

Swissmedic

·         Changes to the Full declaration form

·         Changes to the Guidance document (parallel import)

·          

World Health Organization (WHO)

·         WHO comments on United States’ announcement of intent to withdraw

·         WHO Points to consider in continuous manufacturing of pharmaceutical products

The Pharmaceutical Inspection Co-operation Scheme (PIC/S)

·         The African Union Development Agency (AUDA-NEPAD) becomes a new PIC/S Associated Partner Organisation

Products

·         MHRA-Seladelpar approved to treat adult patients in the UK with liver illness

·         MHRA Aprocitentan (Jeraygo) to treat hypertension

·         MHRA Garadacimab (andembry) approved to prevent angioedema attacks

Conferences

·         PHSS Sterile Product Conference

·         PHSS Aseptic Processing Workshop

RECENT DEVELOPMENTS IN GMP AND REGULATORY REQUIREMENTS

UK

Medicines and Healthcare products Regulatory Agency (MHRA)

New MHRA Chair outlines priorities

Professor Anthony Harnden has officially started his role as Chair of the MHRA.

In his first 100 days, his priority will be talking and listening to staff and partner organisations so he can gain a strong understanding of how to drive forward the strategic direction of the MHRA to maintain the UK as a global centre of excellence in life sciences, in the best interests of patients and the public.

He will be particularly focused on helping the MHRA:

·         Protect patient safety through robust safety surveillance systems

·         Embrace risk-proportionate regulation

·         Foster an inclusive workplace where people flourish.

New, integrated Innovative Licensing and Access Pathway

UK patients enabled access to transformative new medicines in shortest time possible via new, integrated Innovative Licensing and Access Pathway (ILAP).

It is the only example globally of an end-to-end access pathway, where a medicine developer can work collaboratively with the national health system, the regulator, and health technology assessment bodies from the early stages of clinical development.

The ILAP was first launched in January 2021 to offer developers of promising new medicines a single platform to collaborate with the MHRA and the UK Health Technology Assessment (HTA) bodies to accelerate the time taken for innovative medicines to get to patients. The UK life sciences ecosystem within which the ILAP sits has since evolved. In response to feedback from stakeholders and the recommendations of the Pro-innovation Regulation of Technologies Review, ILAP partners have worked together to refresh the pathway.  The new ILAP will bring a number of key improvements compared to the original pathway.

Pharmacovigilance updates

On 8 Jan the following aspects were updated:-

·         Pharmacovigilance system requirements

·         The locations for a Marketing Authorisation Holder (MAH) and a Qualified Person responsible for Pharmacovigilance (QPPV)

·         The MHRA approach to pharmacovigilance

Guidance on new Medical Devices Post-Market Surveillance requirements

A suite of guidance has been published by the MHRA. This guidance is designed to help medical device manufacturers understand and prepare for the new post-market surveillance (PMS) regulation. for medical devices in Great Britain (GB) which will come into force on the 16th June 2025.

Key new requirements are enhanced data collection, shorter timelines for reporting serious incidents and summary reporting to enable the MHRA and manufacturers to identify safety issues earlier, as well as clearer obligations for risk mitigation and communication to protect patients and users.

Businesses are encouraged to start using the guidance straight away so that they understand their obligations and are ready to comply with the regulations when they take effect.

These new regulations are part of wider regulatory reform and will introduce clearer and more risk-proportionate PMS requirements that improve the safety of medical devices across GB and provide certainty for manufacturers.

These new regulations will apply to medical devices, including in vitro diagnostic (IVD) devices and active implantable medical devices, in use in GB. However, the PMS requirements vary based on the risk level posed by the device to patients. The guidance will provide additional detail on these requirements to support manufacturers with their PMS activities and help to ensure their devices continue to meet appropriate standards of safety and performance.

As businesses put the new guidance into use, feedback is welcomed, and the MHRA encourages this to be provided through Trade Associations, if businesses have one, who will be working closely with the MHRA to help identify and address where additional enhancements are needed.

Criminals denied £7.5m in profits from the illegal trade in medicines

MHRA last year stopped criminals making more than £7.5 million linked to the illegal trade in medicines.

As part of the crackdown on criminal profits, more than 17 million doses of illegally traded medicines, with a potential street value of more than £40 million, were taken out of circulation last year by the MHRA and its law enforcement partners. These included painkillers, sleeping tablets and erectile dysfunction treatments.

MHRA Windsor Framework hub

The Information on the main changes introduced by the Windsor Framework were updated during January 2025. The updated sections are supplementary information for:-.

·         Pharmaceutical wholesalers, pharmacists and medicines producers

·         UK-wide licensing for human medicines:-supplementary guidance

Europe

European Medicines Agency (EMA)

Guidance on GMP & GDP – Q&A

Several changes have been made to this document during January 2025.

Q&A to Stakeholders on the implications of Regulation (EU) 2023/1182 for centrally authorised medicinal products for human use.

This is Revision 2 of the practical guidance on the applicable rules to centrally authorised medicinal products for human use intended to be placed on the market in Northern Ireland before and after the application of Regulation (EU) 2023/1182.

Revision 2 updates section 11 on the applicability of Regulation (EU) 2023/1182 and, consequently, section 5 on the deletion of the local representative for Northern Ireland and section 6 on the marketing status report.

Write PMS API implementation Guide

Version 1 of this Application Programming Interface (API) Fast Healthcare Interoperability Resources (FHIR) Product Management Service (PMS)

can be found in the ZIP file available on the EMA PMS Webpage. This FHIR Implementation Guide (IG) ZIP package is a comprehensive resource for developers, implementers, and stakeholders who are working to adopt and implement healthcare standards based on FHIR.

The package acts as an all-in-one toolkit to ensure that healthcare data can be exchanged efficiently, accurately, and securely, in full compliance with FHIR standards.

Guideline on quality aspects of mRNA vaccines for veterinary use

This concept paper addresses the need to establish a guideline on the quality aspects of mRNA vaccines. In the area of human medicinal products, the number applications for clinical trials and marketing authorisations for mRNA containing products significantly increased over the last few years and a lot of experience with mRNA vaccines was gained during the COVID-19 pandemic. It is expected that such developments will be seen in the field of vaccines for veterinary use, too.

The concept paper is open for comment until 8 April 2025

European Shortages Monitoring Platform fully operational for monitoring of shortages in the EU

The European Shortages Monitoring Platform (ESMP) is now live with the full scope of functionalities. This will enable marketing authorisation holders (MAHs) and national competent authorities (NCAs) to directly report information on supply, demand and availability of nationally and centrally authorised medicines during crises and preparedness actions led by EMA’s Executive Steering Group on Shortages and Safety of Medicinal Products (MSSG)

The new release facilitates monitoring and management of critical medicines during public health emergencies and major events and in the context of preparedness activities.

The use of the ESMP will become mandatory for MAHs and NCAs as of 2 February 2025.

Checklist for Initial Notifications for Parallel Distribution

EMA have issued revision 5 of this document.

One Health approach

The European Medicines Agency (EMA) and four other health and environment agencies of the European Union (EU) work together to implement the One Health approach. It aims to enable the EU and its Member States to better prevent, predict, detect and respond to health threats.

EU agencies unite to tackle azole fungicide resistance in Aspergillus fungi

The extensive use of azole fungicides (azoles), particularly in some agricultural and horticultural practices, can increase the risk of Aspergillus fungi developing resistance to essential antifungal treatments. This significant finding is highlighted in a report by the five EU health and environment agencies, with support from the European Commission’s Joint Research Centre (JRC).

The Agencies collaborated under a One Health approach to address this growing threat.

Azole medicines are essential for treating aspergillosis, a serious infection caused by Aspergillus fungi. However, these fungi are increasingly becoming resistant to azole therapies, making treatment less effective.

The joint report highlights that their broad use outside human medicine, particularly in agriculture, contributes to the risk of Aspergillus becoming azole-resistant. Exposure to certain environments where azole fungicides are used or present, such as agricultural and horticultural waste and freshly cut wood, can potentially increase the risk of infection with azole-resistant Aspergillus spp.

Product Management Service (PMS) webinar on Product User Interface (PUI) edit functionalities for industry users

This is a training session for marketing authorisation holders (MAHs) to explain and showcase the edit functionalities for Product User Interface (PUI) in Product Lifecycle Management Portal.

The Product Management Service (PMS) team is hosting a training session for MAHs to explain and showcase the edit functionalities for PUI in Product Lifecycle Management Portal.

A first set of these edit functionalities will be released at the end of January 2025 for human non-centrally authorised products (non-CAPs) data for PUI industry users only - Non-CAPs are Products authorised throughout mutual recognition procedure (MRP), decentralised procedure (DCP) and national procedure (NAP).

Participation is recommended to industry stakeholders working on regulatory affairs of their respective organisations.

The session took place via WebEx and a video recording will be available after the event.

[A useful presentation is included -if you can cope with the plethora of unfamiliar acronyms! MBH]

There was also a Q&A clinic held on the topic on13 February 2025.

Qualification of non-mutagenic impurities - Scientific guideline

This draft, for public comment, reflection paper considers the safety evaluation of non-genotoxic impurities (NGI) in chemically synthesised pharmaceuticals and is intended to establish a framework to facilitate future discussions among stakeholders. Qualification of NGI may be required when data from the regular non-clinical development with the API batches is not considered sufficient. The aim of this paper is to complement currently available guidelines addressing qualification of NGI such as ICH Q3A and ICH Q3B. The reflection paper discusses different non-animal approaches, which may provide more compound-specific information than animal studies with API batches containing the impurities at low levels.

Clinical Trials Regulation becomes fully applicable

The Clinical Trials Information System now supports submission, assessment and oversight of all trials in the EU. From 31 Jan 2025, all clinical trials in the European Union, including ongoing trials that were approved under the previous legal framework, the Clinical Trials Directive (CTD), are governed by the Clinical Trials Regulation (CTR).

This marks the end of a three-year transition period, during which more than 5,000 clinical trials were transitioned to the CTR through submission to the Clinical Trials Information System (CTIS), the single-entry point for sponsors and regulators for the submission and assessment of applications for clinical trials in the EU.

Remaining trials that are ongoing after 30 January and that were not moved to the new system may be subject to corrective measures

applied by EU Member States. Transition procedures are no longer available and sponsors of ongoing CTD trials are required to submit a new application via CTIS.

The European Directorate for the Quality of Medicines & HealthCare (EDQM)

Certification monthly report

EDQM’s Certification of Substances Department has published its monthly report up to the end of December 2024

[The usual / useful graphics report MBH]

Recommendation CM/Rec(2024)8 on the family approach to discuss deceased organ and tissue donation

The Committee of Ministers of the Council of Europe adopted this instrument, which promotes a family-centred approach to discussing organ and tissue donation after a person’s death.

This recommendation was adopted to help solve a very simple problem, but for which there is no simple solution. Efforts to save and improve tens of thousands of lives come up against the same obstacle year after year: the insufficient supply of donated organs and tissues.

The measures aim to improve consent rates for donation, and thus the availability of often long-awaited organs and tissues to treat patients in need, and support families during the grieving process.

[An unusual topic for our Update, but one which I personally strongly support. It is also one in which all of us, should we so wish, can ultimately become involved. MBH]

Publication of BSP establishment study for hepatitis A virus coating reagent BRR

The Biological Standardisation Programme (BSP) recently established batch 2 of the Ph. Eur. Hepatitis A virus Coating Reagent Biological Reference Reagent (BRR) for use in the in vitro potency assay of hepatitis A vaccines by ELISA.

The BRR was established in an international collaborative study (BSP165) and adopted by the European Pharmacopoeia Commission at its session in November 2023.

The outcome of the study, the materials and methods used and the statistical analysis of the results are detailed in an online report.

Implementation of the European Pharmacopoeia Supplement 11.8 – Notification for CEP holders

Supplement 11.8 of the European Pharmacopoeia (Ph. Eur.) is now available. Holders of Certificates of suitability to the monographs of the Ph. Eur. (CEPs) are invited to update their applications according to the revised monographs that will be implemented on 1 July 2025.

How CEP holders can avoid the rejection of notifications

In order to facilitate the acceptance of proposed changes in a timely manner, CEP holders are reminded that the EDQM Guideline on requirements for revision/renewal of Certificates of Suitability to the European Pharmacopoeia Monographs (PA/PH/CEP (04) 2, 7R corr) lists different notifications and associated conditions, and that any change not classified as a notification or a major change should be classified as a minor change by default (with the exception of editorial changes for which specific guidance is given in the policy document). Any submission of a notification which includes changes not classifiable as a notification will be rejected and the changes will then need to be resubmitted using the correct classification (with associated documentation and fee).

Significant milestone in the elimination of animal tests from the Ph. Eur.

At its 180th session in November 2024, the European Pharmacopoeia Commission (EPC) decided to suppress the general chapters on Pyrogens (2.6.8), Histamine (2.6.10) and Depressor substances (2.6.11) from the European Pharmacopoeia (Ph. Eur.), all of which require the use of animals. It was also agreed to revise 14 monographs to delete either the reference to the histamine chapter or the requirement to eliminate or minimise substances that lower blood pressure.

Present in the Ph. Eur. since its first edition, these animal tests were added to ensure the safety of parenteral medicines, before the development of modern analytical techniques and GMP conditions.

Three new Ph. Eur. general chapters for plastic materials for containers

After several years of work by the experts of Group 16 (Plastic materials, plastic containers and closures), three new general chapters on plastic materials were adopted by the European Pharmacopoeia Commission at its 180th session in November 2024. These general chapters – Cyclo-olefin polymers (3.1.16), Cyclo-olefin copolymers (3.1.17) and Styrene block copolymers (3.1.18) – are the first new texts to be published in section 3.1. Materials used for the manufacture of containers since Polyethylene terephthalate (3.1.15) in 2001, when the European Pharmacopoeia (Ph. Eur.) was in its 4th edition. They are also the first texts in section 3.1 to refer to the general chapter on Extractable elements in plastic materials for pharmaceutical use (2.4.35), published in September 2024. With the addition of cyclo-olefin polymers, cyclo-olefin copolymers and styrene block copolymers, the Ph. Eur. now covers a total of 14 different materials used in the manufacture of containers for pharmaceutical uses.

Reference Standards.

3 new Ph. Eur. reference standards and 15 replacement batches released in December 2024

First three general texts on MRNA vaccines

At its 180th session in November 2024, EPC adopted three new general texts relating to the production and quality control of mRNA vaccines and their components, namely:

·         mRNA vaccines for human use (5.36), covering mRNA packaged in lipid nanoparticles, i.e. mRNA-LNP medicinal products;

·         mRNA substances for the production of mRNA vaccines for human use (5.39), covering mRNA active substances used in the manufacture of mRNA vaccines;

·         DNA templates for the preparation of mRNA substances (5.40), covering the linear DNA template used as starting material for the preparation of mRNA substances.

This new series of Ph. Eur. texts on mRNA vaccines is the result of the collaborative work and active engagement of the Ph. Eur.’s mRNAVAC Working Party – a large and dynamic group comprising specialists representing industry, academia, licensing authorities and national control laboratories from around the world.

Pharmeuropa 37.1 just released

All new European Pharmacopoeia (Ph. Eur.) texts and texts that have undergone technical revisions are published in Pharmeuropa for public consultation.

The deadline for comments on Pharmeuropa 37.1 is 31 March 2025.

Amiodarone capsules monograph published in European Paediatric Formulary

A new monograph, Amiodarone hydrochloride, capsules (5-200 mg), developed by the European Paediatric Formulary (PaedF) Working Party, been added to the European Paediatric Formulary.

With the publication of this monograph, the Formulary (which is not legally binding) has now reached an important milestone of 10 published monographs.

Nordic Countries

Common nordic packages for better availability

Starting from the new year, a pilot project will introduce English-language common nordic packages. This aims to improve the availability by simplifying production and distribution.

The Nordic medicines agencies are collaborating on a project to increase the number of common Nordic packages. The project will test how English-language common Nordic packages will work across all Nordic countries: Denmark, Finland, Iceland, Norway, and Sweden. 

The goal is to enhance supply security and availability to essential hospital products in all Nordic countries, ensuring that Nordic patients have access to more medicines. 

There has for a long time been a shortage of several essential medicines in the Nordic markets. Each Nordic country, being a relatively small market, is less attractive than larger markets. This issue often affects medicines used by small and specific patient groups that are rare but vital. Securing enough medicines becomes more challenging when separate packages in different languages are required. Currently, there is a requirement for national languages on packages, and there is extensive use of common Nordic packages that includes various Nordic languages. The new pilot explores using only English for packages and the printed package leaflets for certain selected, particularly vulnerable medicinal products. The intention is not for healthcare personnel and patients to have to read the English package leaflet.

Package leaflets in all the Nordic languages will be digitally available 

[An excellent initiative. This should facillitate the scheduling of production runs for relatively low offtake markets. MBH]

Ireland

The Health Products Regulatory Authority (HPRA)

Joint packs with other member states

In relation to the above article on common Nordic packages, HPRA have commented that adding Irish product details to such packs could help maximise the availability of medicines in Ireland.

MAHs can avail of this in cases where:

·         the product information text is fully harmonised, for example through MRP/DCP

·         no additional Irish blue box elements are required

MAHs can then submit a national article 61.3 notification to add required authorisation details such as the marketing authorisation holder and PA number to such packs.

This will result in a joint pack and further flexibility for companies. HPRA can also support this approach for centralised products, however reviews of centralised mock-ups are within the remit of the EMA.

United States of America

The US Food and Drug Administration (USFDA)

Considerations for the Use of Artificial Intelligence To Support Regulatory Decision-Making for Drug and Biological Products

This guidance provides recommendations to sponsors and other interested parties on the use of artificial intelligence (AI) to produce information or data intended to support regulatory decision-making regarding safety, effectiveness, or quality for drugs. Specifically, this guidance provides a risk-based credibility assessment framework that may be used for establishing and evaluating the credibility of an AI model for a particular context of use (COU).

Interim Policy on Compounding Using Bulk Drug Substances Under Section 503A & 503B of the Federal FD&C Act

FDA is announcing the availability of these final guidances for industry.

The guidances finalize the draft guidances of the same title issued in December 2023 and replaces the final guidances of the same titles issued in January 2017.

Under section 503A of the FD&C Act, bulk drug substances used in compounding must either be components of FDA-approved drug products, the subject of an applicable United States Pharmacopeia (USP) or National Formulary (NF) monograph, or appear on a list established by FDA through rulemaking of bulk drug substances that can be used in compounding. Under section 503B of the FD&C Act, bulk drug substances used in compounding must either appear on a list published by FDA in the Federal Register of bulk drug substances for which there is a clinical need, or they must be used to compound drug products that appear on FDA’s drug shortage list at the time of compounding, distribution, and dispensing.

FDA has also opened two public dockets (FDA-2015-N-3534 and FDA-2015-N-3469) where interested persons can nominate bulk drug substances for inclusion on the lists of bulk drug substances that can be used to compound drug products under sections 503A and 503B of the FD&C Act.

Considerations for Complying with 21 CFR 211.110

This guidance, when finalized, will describe considerations for complying with the requirements in 21 CFR 211.110 to ensure batch uniformity and drug product integrity. In addition, this guidance discusses related quality considerations for drug products that are manufactured using advanced manufacturing. It also discusses how manufacturers can incorporate process models into commercial manufacturing control strategies.

This guidance applies to the manufacture of human drug products, including biological products, and animal drug products.

Advanced manufacturing is a term for an innovative pharmaceutical manufacturing technology or approach that has the potential to improve the reliability and robustness of the manufacturing process and supply chain and increase timely access to quality medicines for the American public. Advanced manufacturing can integrate novel technological approaches, use established techniques in an innovative way, or apply production methods in a new domain where there may be limited experience or no defined best practices. Advanced manufacturing can potentially be used for new or currently marketed large or small molecule drugs.

International

Switzerland

Swissmedic

Changes to the Full declaration form

Listing the full qualitative composition of aromatic substances in human and veterinary medicinal products is no longer mandatory

Extensive experience gained during the implementation of the full declaration has shown that knowledge of the full qualitative composition of flavours and fragrances (aromatic substances) with complex compositions does not provide any clear added value for the assessment at Swissmedic.

In particular, this means that flavouring manufacturers no longer need to submit confidential documents directly to Swissmedic – a complex process that was often necessary in the past for reasons of confidentiality.

Changes to the Guidance document (parallel import)

Market surveillance measures based on safety signals or quality defects in the original medicinal product must also be implemented for parallel imported medicinal products

World Health Organization (WHO)

WHO comments on United States’ announcement of intent to withdraw

The World Health Organization regrets the announcement that the United States of America intends to withdraw from the Organization.

The United States was a founding member of WHO in 1948 and has participated in shaping and governing WHO’s work ever since, alongside 193 other Member States, including through its active participation in the World Health Assembly and Executive Board.

WHO hopes the United States will reconsider and looks forward to engaging in constructive dialogue to maintain the partnership between the USA and WHO, for the benefit of the health and well-being of millions of people around the globe.

WHO Points to consider in continuous manufacturing of pharmaceutical products

This draft document (DRAFT WORKING DOCUMENT QAS/24.957) is open for comment through to the end of March 2025.

Continuous manufacturing (CM) is a relatively new concept in pharmaceuticals manufacture.The guidelines consider how to transition from traditional batch processing into CM, including technological complexities, workforce training, and regulatory hurdles.

The Pharmaceutical Inspection Co-operation Scheme (PIC/S)

The African Union Development Agency (AUDA-NEPAD) becomes a new PIC/S Associated Partner Organisation

With a shared dedication to safeguarding public health and safety, PIC/S and AUDA-NEPAD have decided to establish a cooperation agreement in the field of Good Manufacturing Practice (GMP) standards and quality systems for pharmaceutical products and Active Pharmaceutical Ingredients (API).

The scope of the cooperation agreement includes :

·         Facilitating pre-accession and accession processes to PIC/S for National Regulatory Authorities (NRA) members of the African Medicines Agency (AMA);

·         Training of inspectors in the field of GMP;

·         Participating at each other’s meetings.

With the active contribution of other stakeholders, collaboration activities will aim at supporting capacity and confidence building among African NRA and at guiding them on their journey toward becoming PIC/S Participating Authorities.

An official welcome will be given to AUDA-NEPAD at the next PIC/S Committee meeting, which will take place in Geneva on 14 and 15 April 2025.

Products

[This section makes reference to some of the most notable new products approved during the past month and focuses on approvals of medicines for which there is a previously unmet need and / or where approvals have been made using shared information from other trusted regulators.MBH]

Seladelpar approved to treat adult patients in the UK with liver illness

MHRA has approved the medicine seladelpar (Livdelzi) in adults for the treatment of a liver illness called Primary Biliary Cholangitis (PBC), including pruritus. It works by controlling liver problems in the body and making the liver less inflamed.

MHRA states “We’re assured that the appropriate regulatory standards for the approval of this medicine have been met. As with all products, we will keep its safety under close review.”

The new marketing authorisation was granted to Cymabay Ireland Limited.

Aprocitentan (Jeraygo) to treat hypertension

MHRA has approved the medicine aprocitentan (Jeraygo) to treat hypertension (high blood pressure) in adults whose blood pressure cannot be adequately controlled by at least three other medicines (also known as resistant hypertension).

Aprocitentan belongs to the class of medicines called endothelin receptor antagonists. It works by helping to stop the blood vessels from tightening. As a result, the blood vessels relax, and blood pressure is lowered.

The new marketing authorisation was granted in 106 days on 8 January to Idorsia Pharmaceuticals Deutschland GmbH under International Recognition Procedure (IRP) Route B. The Reference Regulator (RR) was the European Medicines Agency (EMA),

Garadacimab (andembry) approved to prevent angioedema attacks

MHRA has approved garadacimab (brand name Andembry) for patients aged 12 years and older with hereditary angioedema (HAE) to prevent angioedema attacks. HAE is a rare condition that causes fluid to build up throughout the body, triggering sudden and repeated serious swelling. HAE is a condition that often runs in families, but some people may not have a family history.

The new marketing authorisation was granted on January 24th 2025 to CSL Behring GmbH

This national approval has been granted through an ACCESS work-sharing procedure. The ACCESS consortium* is a medium-sized coalition of regulatory authorities that work together to promote greater regulatory collaboration and alignment of regulatory requirements.

*The MHRA is part of the Access Consortium along with the Therapeutic Goods Administration, Health Canada, Health Sciences Authority of Singapore and Swissmedic.

Conferences

PHSS Sterile Product Conference 2025

This major PHSS conference will address the key challenges in sterile product manufacture and processing of intermediates that require low bioburden control following risk-based initiatives from a regulatory, Pharmaceutical/ Bio-pharmaceutical industry and healthcare pharmacy perspective.

Networking dinner & AZ site tour 25 June, - conference 26 June 2025

PHSS Aseptic Processing Workshop

March 25–26, 2025 | Woodland Grange, Leamington Spa

The Workshop provides two days of intensive, CPD-accredited training on best practices in aseptic manufacturing and contamination control. The program includes keynote presentations and interactive, small-group workshops designed for professionals at various stages in the field of aseptic processing.

Immersive, hands-on experience in aseptic processing led by industry experts.

Enhance your understanding of GMP compliance, contamination control strategies, and environmental monitoring.

And finally…

Further information on these and other topics can be found in recent versions of the “Regulatory Update” on the PHSS website (members area) by utilising the hyperlink within that particular Update.

We hope that our readers find our reviews to be both informative and helpful in keeping up to date with pharmaceutical legislation and regulatory guidance.