- tamsinmarshall4
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Review of Developments in GMP and the Regulation of Medicines June 2026
EJPPS Volume 31.2C
INTRODUCTION
The topics covered in this edition of the “Update” have come from the UK, EU, USA & Australian regulatory authorities.
UK
Medicines and Healthcare products Regulatory Agency (MHRA)
· UK-wide consultation on redefining gene therapies
· MHRA invites views on proposed changes to medical device regulation
· MHRA supports Northern Ireland life sciences sector with new hub
· MHRA to support local life sciences sector in Wales
· MHRA’s largest ever seizure of unlicensed weight loss medicines
· Draft rare disease therapies regulatory framework
EU
European Medicines Agency (EMA)
· Recommendation to update the antigenic composition of authorised COVID-19 vaccines for 2026-2027
· Concept paper on the need for revision of the guideline on the development of medicinal products for the treatment of smoking
· Quality of medicines Q&A Part 2
· EMA welcomes political agreement on Critical Medicines Act
The European Directorate for the Quality of Medicines & HealthCare (EDQM)
· Council of Europe position paper highlights considerations around in-use storage time of reconstituted medicines in hospitals
· New Council of Europe Recommendation sets standards for remote and online medicine provision
· EDQM reference standards monthly newsletter – April 2026
· 23rd edition of the Blood Guide – The EDQM opens digital stakeholder consultation
· Certification monthly report of activities
· New OCABR guideline for mRNA vaccines produced with LNPs and revision of two additional OCABR vaccine guidelines
· The European Pharmacopoeia Commission intensifies its efforts on quality control guidance for preparations for inhalation and nasal administration
USA
· One-Day Inspectional Assessments to Strengthen and Expand Oversight
· ICHQ8, Q9, and Q10 - Q&A (R5)
· Bioequivalence studies with pharmacokinetic endpoints for drugs submitted under an ANDA
· Statistical Approaches to Establishing Bioequivalence
International
Australia
Therapeutic Goods Administration (TGA)
· All medicinal cannabis products supplied to Australian patients must meet quality standards
Products
· MHRA approves Linerixibat (Lynavoy) for the treatment of itch due to biliary tract disease
· MHRA approves Dawnzera for the treatment of hereditary angioedema
· MHRA approves beremagene geperpavec (Vyjuvek) for the treatment of dystrophic epidermolysis bullosa (DEB)
· MHRA- Rilzabrutinib authorised to treat adults with immune thrombocytopenia when prior treatments have been insufficient
Conferences
· GMP multistakeholder workshop on expert contributions to artificial intelligence guidance development (Annex 22)
RECENT DEVELOPMENTS IN GMP AND REGULATORY REQUIREMENTS
UK
Medicines and Healthcare products Regulatory Agency (MHRA)
UK-wide consultation on redefining gene therapies
To future-proof the sector and ensure regulation keeps up with scientific and technological developments, the MHRA is consulting on proposed changes to how gene therapies are defined in UK legislation.
Gene therapies have evolved significantly since the current definitions were established, more than a decade ago. Since then, advances in science and manufacturing technologies have prompted consideration on whether the wording remains appropriate for use.
The MHRA is consulting on updated definitions that would classify a medicinal product as a gene therapy, based on how it works.
The updated definition will not alter the approval process for gene therapies, the regulatory classification of products already licensed, or change any existing safety, quality, or efficacy standards.
MHRA invites views on proposed changes to medical device regulation
New pre-market regulatory requirements for medical devices and in vitro diagnostic devices entering the GB market have been published by the MHRA on the World Trade Organisation notification portal.
The notification provides an opportunity for WTO members to comment on the draft pre-market regulatory requirements, titled the draft Medical Devices (Amendment) Regulations 2026.
The MedTech industry, approved bodies, healthcare providers and patients are also invited to share their views on the impact of the proposed changes via an MHRA survey. Responses will inform the Impact Assessment and future implementation of the regulations.
MHRA supports Northern Ireland life sciences sector with new hub
The MHRA is establishing a hub in Northern Ireland, hosted by Invest Northern Ireland.
The MHRA hub in Belfast will strengthen collaboration and engagement with local industry, academia and the healthcare sector.
This builds on the MHRA’s established partnerships with healthcare, academia and industry and ensures its work is even more strongly linked to the whole of the UK.
Northern Ireland is home to around 250 businesses in the life sciences sector, supporting 18,000 jobs and contributing £1.1 billion to the local economy.
Local stakeholders working within the healthcare, health and life sciences sectors across Northern Ireland will be able to book periodic, in-person surgery sessions with MHRA technical experts to discuss topics such as innovation pathways, clinical investigations and be signposted to further regulator support available from the MHRA throughout the product development pipeline.
MHRA to support local life sciences sector in Wales
MHRA announces new programme of events and greater engagement with industries, researchers, local NHS and government in Wales.
Wales is a leader in MedTech, AI-powered diagnostics, wound healing, regenerative medicine, cell therapy and precision medicine employing over 13,000 specialists across 300 companies.
The MHRA’s new programme of events puts regulatory expertise at the heart of the Welsh life sciences powerhouse helping the industry to move faster and further.
Three quarters of the industry’s output is for export, generating a turnover of £2.6 billion and significantly contributing to the economy.
MHRA Liaison Days will bring MHRA representatives to Wales on a regular basis to host meetings and workshops around the topics most relevant to the Welsh Life Sciences and Healthcare sectors. MHRA’s Liaison Days will include topics such as innovation pathways, clinical investigations for medtech, regulatory support and ways to engage with the MHRA throughout the product development pipeline.
MHRA’s largest ever seizure of unlicensed weight loss medicines
The raid on a Northamptonshire estate dismantled what is believed to be a major illicit manufacturing and distribution operation.
Officers from the MHRA Criminal Enforcement Unit have arrested two people after raiding a country estate near Northampton, recovering around 12,000 doses of unlicensed weight loss medicines in the largest ever seizure of such products by the agency. During the operation, two male suspects, both aged 29, were arrested on suspicion of offences under the Human Medicines Regulations 2012.
The property is believed to have been used as a large-scale facility to manufacture, assemble, and distribute unlicensed weight loss medicines, including retatrutide and tirzepatide, as well as peptide products.
Draft rare disease therapies regulatory framework
Rare diseases affect more than 3.5 million people in the UK, but only a small proportion have an approved medicinal treatment. Many rare conditions face unique scientific and practical challenges: extremely small patient populations, limited natural history data, difficulties conducting randomised controlled trials, and in some cases, individual‑ or family‑specific genetic variants. These factors make traditional regulatory routes difficult or impossible to use.
The rare disease therapies framework aims to address these challenges by introducing a single, flexible authorisation, the Investigational Marketing Authorisation (IMA), which would allow controlled early access to a therapy while further clinical and real‑world evidence continues to be generated.
MHRA are seeking feedback from manufacturers, developers, clinicians, researchers, patient organisations, carers, families and other stakeholders on our regulatory approach to rare disease therapies.
Europe
European Medicines Agency (EMA)
Recommendation to update the antigenic composition of authorised COVID-19 vaccines for 2026-2027
EMA is providing through its Emergency Task Force a recommendation with respect to the antigenic composition of authorised COVID-19 vaccines for use during the 2026-2027 vaccination campaign in the EU/EEA region.
Concept paper on the need for revision of the guideline on the development of medicinal products for the treatment of smoking
There has been a rapid development of essentially tobacco-free recreational products containing nicotine for inhalation (e.g. electronic cigarettes) and oromucosal use (e.g. snuff) in the EU. Addiction to tobacco-free nicotine products has become an increasing problem, especially in the adolescent and young adult populations. The current guideline is not intended for and is neither completely applicable for development of medicinal products for the treatment of the addiction to non-smoking nicotine products. Nevertheless, as no other guideline applies in those cases, the guideline for smoking cessation is still the most relevant. To meet the treatment needs arising from the new types of nicotine addiction, adjustments of this guideline are proposed for the document to be fully applicable for all types of nicotine dependence.
Deadline for comments 30 August 2026.
Quality of medicines Q&A Part 2
The Section 'Reduced testing of incoming starting materials' updated 29 May 2026.
EMA welcomes political agreement on Critical Medicines Act
EMA welcomes the provisional agreement reached by the European Parliament and the Council of the European Union on the proposed Critical Medicines Act (CMA), which represents an important step in strengthening the resilience, security and sustainability of the European Union’s supply of critical medicines.
The European Directorate for the Quality of Medicines & HealthCare (EDQM)
Council of Europe position paper highlights considerations around in-use storage time of reconstituted medicines in hospitals
The Council of Europe’s Committee of Experts on Quality and Safety Standards in Pharmaceutical Practices and Pharmaceutical Care (CD‑P‑PH/PC), co-ordinated by the EDQM, has published a position paper on the in‑use storage time of reconstituted parenteral medicines. This document touches upon current practices in hospital settings and highlights the need to close the gap between regulatory requirements and clinical practice with the aim to support safe, accessible and cost‑effective patient care.
The position paper draws on the findings of a survey carried out in 2024 among hospital pharmacists across Council of Europe member states, which examined hospital practices related to the in‑use storage times of reconstituted medicines for parenteral administration. The survey found that, in certain situations, hospital pharmacies may set longer in‑use storage times than those indicated in the summary of product characteristics (SmPC) for a variety of reasons.
New Council of Europe Recommendation sets standards for remote and online medicine provision
This legal instrument is aimed at supporting appropriate, equitable and safe provision of medicines in an increasingly digital healthcare landscape. The EDQM, co-ordinated this initiative and the Recommendation CM/Rec(2026)7 provides member states with a coherent framework to enhance the provision of medicines while maintaining high standards of quality, safety and public health protection. It addresses the significant changes in healthcare delivery in recent years, characterised by the expanding use of digital technologies, remote services and online platforms. This transformation accelerated during the COVID‑19 pandemic, which demonstrated both the potential of remote and online provision of medicines to improve access for patients and the need for clear, robust safeguards to protect the quality of care and patient safety.
EDQM reference standards monthly newsletter – April 2026
13 new European Pharmacopoeia reference standards and 16 replacement batches released in April 2026
23rd edition of the Blood Guide – The EDQM opens digital stakeholder consultation
The Blood Guide is going digital for its 23rd edition. From 26 May to 3 July 2026, the fully revised text will be presented on a new digital platform for stakeholder consultation, enabling a more streamlined, transparent and user‑friendly process.
Access to the platform will be provided to stakeholders to allow for the submission of comments on the revised text. This consultation phase will be followed by a review process, before ultimate adoption of the finalised text by the European Committee on Blood Transfusion (CD-P-TS).
The 23rd edition of the Blood Guide is scheduled for publication in a fully digital format on a dedicated platform during the second quarter of 2027.
Certification monthly report of activities
The latest monthly activity report (End of April 2026) for the Certification of Substances Department (DCEP) is now available.
New OCABR guideline for mRNA vaccines produced with LNPs and revision of two additional OCABR vaccine guidelines
EDQM has published a new Official Control Authority Batch Release (OCABR) guideline covering all mRNA vaccines produced using lipid nanoparticles (LNPs), and two revised guidelines covering a new acellular pertussis vaccine and a new herpes zoster vaccine formulation (formerly the Varicella (rDNA) vaccine guideline). The guidelines were adopted by the full EU OCABR Network and have been in force since 1 May 2026.
The new guideline establishes a harmonised framework for the co-ordinated, independent control of all mRNA vaccines produced with LNPs intended for the European market by defining the tests to be performed by Official Medicines Control Laboratories (OMCLs) in the EU OCABR Network and providing a template for the summary protocol for batch release to be provided by the vaccine manufacturers / marketing authorisation holders.
Following the approval of , the related OCABR guidelines were revised to ensure the efficient independent batch release of the new vaccine products.
The European Pharmacopoeia Commission intensifies its efforts on quality control guidance for preparations for inhalation and nasal administration
At its session in November 2025, the European Pharmacopoeia Commission tasked the Inhalanda Working Party (INH WP) with including an Abbreviated Impactor apparatus together with associated procedure(s) in general chapter 2.9.18.
The Ph. Eur. monograph on Nasal preparations (0676) will also be revised to include a requirement to test the uniformity of delivered doses for nasal preparations that are supplied in single-dose containers.
The elaboration of a new general chapter on the assessment of the mass of particles/droplets smaller than 10 µm in nasal preparations (2.9.56) is ongoing. The new text will describe a specifically designed inlet for nasal preparations and reference equipment from updated general chapter 2.9.18.
While this work is ongoing, the INH WP invites input on these topics from any interested parties (e.g. experts from regulatory agencies and industry worldwide).
United States of America
The US Food and Drug Administration (USFDA)
One-Day Inspectional Assessments to Strengthen and Expand Oversight
US FDA has announced that it is piloting one-day inspectional assessments, as part of a broader initiative to make its inspectional resources more targeted and efficient.
As part of this pilot, which launched in April, the agency is conducting shorter, focused screening assessments to complement standard FDA inspections.
For the FDA, the ability to conduct shorter, targeted assessments allows for broader surveillance coverage, enabling the agency to assess more facilities and gather critical insights without compromising regulatory rigor. For industry, these assessments can provide timely feedback while minimizing operational disruption, particularly for lower-risk establishments.
Though overseas inspections are not specifically mentioned, if you, or your company are involved in the supply of product to the USA, then you may be well advised to prepare for FDA to turn up unexpectedly at your facility for one of these inspections. They could possibly be tacked easily onto an inspectors overseas schedule.
[See the recent Impact statement on the PHSS website. I have also been informed that FDA inspectors have recently performed more than one un-announced, but not 1 day, overseas inspection following on from other overseas non-inspection activity in at least one overseas country MBH]
ICHQ8, Q9, and Q10 - Q&A (R5)
FDA has now published this document as a Final Guidance for Industry,
Bioequivalence studies with pharmacokinetic endpoints for drugs submitted under an ANDA
This guidance provides recommendations to applicants planning to submit bioequivalence (BE) information in abbreviated new drug applications (ANDAs) as well as amendments and supplements to ANDAs. In addition, this guidance describes how to meet the BE requirements set forth in the Federal Food, Drug, and Cosmetic Act (FD&C Act) and FDA regulations.
This guidance is applicable to immediate-release and modified-release oral dosage forms. It is also applicable to non-orally administered drug products in which reliance on systemic exposure measures is suitable for establishing BE (e.g., transdermal delivery systems and certain rectal and nasal drug products). This guidance will also be useful to applicants planning BE studies intended to be conducted during the postapproval period for changes to a drug product approved under an ANDA
Statistical Approaches to Establishing Bioequivalence
This guidance discusses statistical approaches for BE comparisons and focuses on how to use these approaches both generally and in specific situations.
This guidance finalizes the draft guidance of the same title issued on December 5, 2022, and replaces the guidance of the same title issued on February 2, 2001.
International
Australia
Therapeutic Goods Administration (TGA)
All medicinal cannabis products supplied to Australian patients must meet quality standards
TGA is aware of recent media reports regarding questionable medicinal cannabis products being supplied in Australia.
The TGA would like to confirm that regardless of whether cannabis oil or cannabis flower medicine comes from overseas or Australia, it must comply with the relevant quality standard.
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]
MHRA approves Linerixibat (Lynavoy) for the treatment of itch due to biliary tract disease
MHRA has approved Linerixibat (Lynavoy) for use to treat an itch in adults with primary biliary cholangitis (PBC).
PBC is a condition where bile ducts in the liver become damaged, leading to a build-up of bile acids in the blood. This build-up is thought to cause itching. Linerixibat helps to reduce the build-up of substances, including bile acids, in the body and so reduce itching.
Linerixibat is administered via an orally ingested film-coated tablet.
A global Phase 3 clinical trial, Glisten, evaluated the safety and effectiveness of linerixibat for treating itching in patients with PBC.
The approval was granted to GlaxoSmithKline UK Limited. This product was submitted and approved via the National Procedure
MHRA approves Dawnzera for the treatment of hereditary angioedema
MHRA has approved donidalorsen (Dawnzera) for use in patients 12 years and older with hereditary angioedema (HAE) to prevent angioedema attacks. Donidalorsen is administered as an injection from a pre-filled pen under the skin.
The approval was granted to Otsuka Pharmaceutical Netherlands B.V. The product was submitted and approved via The International Recognition Procedure (IRP).
MHRA approves beremagene geperpavec (Vyjuvek) for the treatment of dystrophic epidermolysis bullosa (DEB)
Beremagene geperpavec (Vyjuvek) can be used from birth onward.
DEB is a rare genetic condition that makes the skin fragile and prone to blistering due to a fault in a gene responsible for holding skin layers together.
Beremagene geperpavec works by copying of the faulty gene into the cells of the wound to help the skin heal. The modified virus and genetic material in this medicine do not change the patient’s DNA.
Beremagene geperpavec is a gel, applied directly to wounds. The approval was granted to Krystal Biotech Netherlands, B.V.
This product was submitted and approved via International Recognition Procedure (IRP).
MHRA Rilzabrutinib authorised to treat adults with immune thrombocytopenia when prior treatments have been insufficient
ITP is a rare autoimmune disease, in which the body´s own immune system attacks and destroys platelets in the blood, causes fatigue, and increases risk of bleeding. Platelets are needed to help create clots and stop bleeding.
Rilzabrutinib works by blocking Bruton’s tyrosine kinase, a protein in the body that plays a role in the immune system. By blocking this protein, the medicine can decrease the destruction of blood platelets and thus increase the number of healthy platelets in the body. This helps reduce the risk of bleeding.
Rilzabrutinib is available as tablets to be taken by mouth twice a day.
This approval is based on a pivotal phase 3 study of 202 patients with ITP whose prior treatments had been insufficient. After 24 weeks, 23 per cent of patients receiving Rilzabrutinib achieved a stable, adequate platelet count to prevent excessive bleeding, compared with 0 per cent on placebo.
The new marketing authorisation was granted to Sanofi B.V
Conferences / webinars / workshops etc.
GMP multistakeholder workshop on expert contributions to artificial intelligence guidance development (Annex 22)
EMA's GMP / GDP Inspectors Working Group is organising a two-day workshop to help shape a risk-based approach to the use of generative artificial intelligence (AI) in medicines manufacturing.
The first day of the workshop (30 June 2026) is organised as an open session where experts present their opinions and evidence.
The second day (1 July 2026) is organised as a closed session where the Annex 22 drafting group at EMA reviews expert contributions.
EMA expects the workshop to produce a report with expert input.
EMA aims to achieve the following objectives via this workshop:
· Collect expert insights on responsible AI requirements, risk identification and mitigation approaches, and practical safeguards
· Identify key principles such as data governance, model evaluation, transparency, accountability, and human oversight
· Identify specific considerations, challenges, and real-world constraints of the pharmaceutical industry
· Identify specific approaches to responsible use of AI in GMP applications
EMA has invited AI experts active in AI research to contribute their wide range of expertise to the workshop
And finally…
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.
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.
GMP Update is compiled by Malcolm Holmes C.Chem. MRSC, a member of the PHSS Management Committee.
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