Regulatory Round-up - May 2025

UNITED KINGDOM

Medicines and Healthcare products Regulatory Agency (MHRA)

National assessment procedure for medicines

The MHRA published guidance on the new national assessment procedure for marketing authorisation (MA) applications on the 4^th of April. The guidance applies to both innovative and established medicines and describes how the MHRA will deliver decisions on MA applications within the 210 day target to support timely access to medicines. A webinar was held on the 6^th of May to provide an overview of the guidance and highlight the key process changes. Please find the video recording of this webinar here.

First MHRA Board meeting held in Scotland, underlining agency’s commitment to regional health equality and growth

The board of the MHRA has met in Scotland for the first time on the 20^th May 2025 to reinforce the agency’s commitment to delivering its health and innovation priorities across the UK. The aim of this meeting was to discuss the MHRA’s commitment to delivering the agency’s priorities in Scotland and served as an opportunity for the MHRA to listen to the experiences and ideas of attendees. Please find further information here.

EUROPE

European Directorate for the Quality of Medicines (EDQM)

European Pharmacopoeia Commission (EPC) adopts mycoplasma general chapter and monographs, updated to incorporate latest analytical developments

The EPC has adopted the general chapter 2.6.7. Mycoplasmas and 11 monographs which had been revised to reflect the new European Pharmacopoeia (Ph. Eur.) approach to the detection of mycoplasma contaminants. The revised general chapter specifies that both the culture method and the indicator cell culture method or, alternatively, a Nucleic acid amplification techniques (NAT method) should be used conjointly to ensure the detection of both “cultivable” and “non-cultivable” mycoplasmas, unless otherwise prescribed in a monograph or unless justified by a risk assessment and authorised by the competent authority. It has also been clarified that, whenever possible, the samples should contain both cells and supernatant. Please find further information here.

European Medicines Agency (EMA)

Good Manufacturing Practice / Good Distribution Practice Inspectors Working Group (GMP/GDP IWG) Concept paper on the revision of Part IV Guidelines on Good Manufacturing Practice specific to Advanced Therapy Medicinal Products

The EMA released for public consultation a concept paper outlining the rationale, objectives, and proposed changes for updating Part IV of the EU Guidelines on Good Manufacturing Practice (GMP) specific to Advanced Therapy Medicinal Products.The proposed revisions focus on several key areas:

• Alignment with Revised Annex 1
• Integration of ICH Concepts
• Adaptation to Technological Advancements
• Updates on Cleanroom and Barrier Systems
• Legal References and Definitions

 The deadline for consultation is 8^th July 2025.

External guidance on the implementation of the EMA Policy 0070 on the publication of clinical data for medicinal products for human use

The EMA has published a guidance on the policy of clinical data for medicinal products for human use (hereafter referred to as ‘Policy 0070’), in accordance with Article 80 of Regulation (EC) No 726/2004. Policy 0070 was adopted by the EMA Management Board on 2^nd October 2014 and subsequently published on the EMA website. Policy 0070 came into force on 1^st January 2015. Phase 1 of the policy pertains to publication of clinical reports only. Clinical reports and individual patient data are collectively referred to as “clinical data”. This guidance has been prepared to assist applicants in preparing packages for publication and the general principles applied.

Committee for Medicinal Products for Human Use (CHMP) positive opinion: Aucatzyl

On 22^nd May 2025, the CHMP adopted a positive opinion, recommending the granting of a conditional MA for the medicinal product Aucatzyl, genetically engineered cell therapy, intended for the treatment of adults from 26 years of age with relapsed or refractory B cell precursor acute lymphoblastic leukaemia. Please find further information here.

USA

Food and Drug Administration (FDA)

Health and human services (HHS), FDA issue request for information (RFI) on Deregulatory plan to lower costs and empower providers

The FDA has announced the launch of a public RFI to identify and eliminate outdated or unnecessary regulations. The initiative is part of a broader federal effort to reduce regulatory burdens and increase transparency, in alignment with President Trump’s Executive Order 14192, “Unleashing Prosperity Through Deregulation.”

Under the Executive Order, the HHS will implement the following measures:

• The 10-to-1 rule: For every new regulation introduced, at least ten existing regulations must be eliminated.
• Regulatory cost cap: The total cost of all new regulations in fiscal year 2025 must be significantly less than zero.
• Expanded scope: The order applies not only to formal regulations but also to guidance documents, memoranda, policy statements, and similar directives.
• Radical transparency: HHS will publish annual reports detailing estimated regulatory costs and the specific rules being offset, promoting greater transparency and accountability.

Please find further information here.

FDA approval: ZEVASKYN

The FDA has approved prademagene zamikeracel (Zevaskyn), an autologous cell sheet-based gene therapy, indicated for treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa. The disorder results from a defect in the COL7A1 gene, which prevents the production of a crucial type of collagen responsible for anchoring the upper and lower layers of the skin. Please find further information here.

INTERNATIONAL

International Conference on Harmonisation (ICH)

ICH Q5(R2) "viral safety evaluation of biotechnology products derived from cell line of human or animal origin

The ICH has published ICH Q5A(R2) “Viral Safety Evaluation of Biotechnology Products Derived from Cell Lines of Human or Animal Origin” guideline training materials. Modules 0-3 are designed to be targeted to Industry and Regulatory audiences to clarify and illustrate key concepts specific to Viral Safety that have been updated in the ICH Q5A(R2) guideline revision, which was adopted in November 2023 and to support implementation of the guideline by use of examples (for illustrative purposes).

Public consultations 

European Medicines Agency (EMA) 

 Food and Drug Administration (FDA)

 International Council for Harmonisation (ICH)

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