Regulatory News
UK Government to Align with EU Standards for High-Risk In Vitro Diagnostic Devices
On 10th July, the UK government announced plans to amend the Medical Devices Regulations 2002 for Great Britain by adopting the EU Common Specifications for high-risk in vitro diagnostic (IVD) devices and repealing the Coronavirus Test Device Approvals (CTDA) regulations. This alignment aims to uphold rigorous performance standards and ensure these critical IVD devices—used to test human samples for health monitoring and disease management—are supported by strong clinical evidence, while reducing regulatory burdens.
Five Non-Executive Directors Reappointed to MHRA Board
On 17th July, it was announced that five non-executive directors (NEDs) have been reappointed to the MHRA board, with the majority of their new terms starting in September 2025.
FDA Issues Warning to Companies Marketing Products Containing 7-Hydroxymitragynine
On 18th July, the FDA announced it has placed Sarepta Therapeutics investigational gene therapy clinical trials for limb girdle muscular dystrophy on clinical hold following three deaths potentially related to these products and new safety concerns that the study participants are or would be exposed to an unreasonable and significant risk of illness or injury. The FDA has also revoked Sarepta’s platform technology designation.
MHRA Publishes Consultation Outcome on Medical Devices Regulations and Market Access
On 22nd July, the MHRA issued the outcome to a consultation on four areas: International reliance; UKCA marking; In vitro diagnostic devices; and Assimilated EU law. The consultation ran from 14 November 2024 to 5 January 2025 and received 287 responses in total with the majority of the respondents agreed with the government’s proposals to remove the revocation date of the four pieces of assimilated law so that they continue to apply in Great Britain, until such time as they are replaced with updated UK law. Doing so will avoid disruption to the regulatory framework and ensure continuity for manufacturers and all who use medical devices.
MHRA Unveils Proposals to Enhance Patient Access to Leading Medical Devices and Drive UK Med Tech Growth
On 22nd July, the MHRA announced new proposals aimed at improving patient access to cutting-edge medical devices and strengthening the UK’s med tech sector. Following a public consultation on future market access routes, the proposals are designed to modernise regulation by aligning with systems in Europe and other advanced markets. The updated approach will streamline patient access to the latest technologies while supporting industrial growth by reducing duplicative regulatory burdens. The UKCA approval pathway will be refocused on first-in-market innovations, including AI-powered medical devices.
New MHRA Regulations Enable On-the-Spot Personalised Treatments for Faster Patient Care
On 23rd July, new regulations introduced by the MHRA have come into effect, streamlining the production of cutting-edge cancer therapies and personalised gene treatments directly at the point of care. In a world-first move, these rules allow for the preparation of breakthrough personalised medicines in small or individual batches—making it quicker and easier to deliver specialised treatment tailored to patients’ needs, right where they are being treated.
Sabine Scherer Elected as New Chair of EMA Paediatric Committee
On 24th July, the EMA’s Paediatric Committee (PDCO) elected Sabine Scherer of Germany as its new chair for a three-year term starting in September 2025. Dr Scherer will succeed Dr Aylward of Ireland’s Health Products Regulatory Authority (HPRA), who has held the position for the past three years.
MHRA Announces Plans to Accelerate Patient Access to Innovative Medical Devices
On 31st July, the MHRA outlined its plans to facilitate earlier access to cutting-edge medical devices that meet unmet clinical needs within the NHS. As part of this initiative, the agency will invest in developing a new Early Access service. This service will offer time-limited, conditional access to promising medical technologies prior to full regulatory approval, where there is a clear clinical need and supporting evidence of patient benefit.
MHRA Issues July 2025 Safety Roundup
On 31st July, the MHRA published its monthly safety update, summarising the latest guidance and alerts for users of medicines and medical devices.
Published Guidance
US Food & Drug Administration (FDA)
- 2nd July: Myelodysplastic Syndromes: Developing Drug and Biological Products for Treatment. The FDA issued draft guidance to assist sponsors in the clinical development of drug and biological products for the treatment of the myelodysplastic syndromes (MDS), specifically the development of drugs that are considered disease-modifying and not drugs that are considered as supportive therapy (e.g., erythropoiesis-stimulating agents).
- 15th July: Small Volume Parenteral Drug Products and Pharmacy Bulk Packages for Parenteral Nutrition: Aluminum Content and Labeling Recommendations. The FDA issued draft guidance to clarify the key factors in calculating the aluminium content to ensure that the total aluminium exposure in parenteral nutrition (PN) does not exceed an acceptable threshold. It also provides FDA’s recommendations regarding the aluminium concentration limits for small volume parenterals (SVPs) packaged as single doses or SVPs packaged in pharmacy bulk packages (PBPs).
- 17th July: Development of Cancer Drugs for Use in Novel Combination – Determining the Contribution of the Individual Drugs’ Effects. The FDA issued draft guidance providing recommendations for characterising the safety and effectiveness of individual drugs used in novel combination regimens for treating cancer, with a focus on demonstrating the “contribution of effect” – how each drug contributes to the overall treatment benefit observed in patients.
- 21st July: E21 Inclusion of Pregnant and Breastfeeding Women in Clinical Trials. This guidance provides FDA’s recommendations for the appropriate inclusion and/or retention of pregnant and/or breastfeeding women in clinical trials and facilitate the generation of robust clinical data that allow for evidence-based decision making on the safe and effective use of medicinal products by these women and their healthcare providers (HCPs).
- 30th July: Medical Device User Fee Small Business Qualification and Determination. This newly published guidance details the types of information an entity seeking the waiver may submit for FDA’s consideration in determining whether to grant the Small Business Registration Fee Waiver, including entities that are in jurisdictions without a national taxing authority.
Updated Guidance
MHRA
- 15th July: AI Airlock: the regulatory sandbox for AIaMD. Updated to reflect that the pilot phase of the AI Airlock is complete, and that applications for phase 2 have now closed
- 15th July: Access, new active substance and biosimilar work sharing initiatives. Ekterly (sebetralstat) was added to the overview of MHRA NASWSI approvals table.
- 16th July: MHRA fees. Various fees sections were updated to reflect increases from 16th July 2025:
- 16th July: Clinical investigations for medical devices. A link was added to updated SME information (relating to fees).
- 17th July: MHRA guidance on the use of real-world data in clinical studies to support regulatory decisions. Updated to reflect closing of consultation period.
- 30th July: COVID-19 test validation approved products. The ‘Products approved under the Medical Devices Coronavirus Test Device Approvals Amendment Regulations’ CSV file was updated.
FDA
- 18th July: Formal Meetings Between the FDA and Sponsors or Applicants of BsUFA Products Guidance for Industry Final guidance was issued to provide recommendations to industry on formal meetings between FDA and sponsors or applicants relating to the development and review of biosimilar or interchangeable biological products regulated by the Center for Drug Evaluation and Research (CDER) or the Center for Biologics Evaluation and Research (CBER).
- 25th June: Small Volume Parenteral Drug Products and Pharmacy Bulk Packages for Parenteral Nutrition: Aluminum Content and Labelling Recommendations Guidance was issued intended to clarify the key factors in calculating the aluminum content to ensure that the total aluminum exposure in parenteral nutrition (PN) does not exceed an acceptable threshold. It also provides FDA’s recommendations regarding the aluminum concentration limits for small volume parenterals (SVPs) packaged as single doses or SVPs packaged in pharmacy bulk packages (PBPs).
Industry News
MHRA Becomes First Regulator to Approve Elinzanetant for Menopausal Symptoms
On 8th July, the MHRA became the first regulatory authority globally to approve elinzanetant for treating moderate to severe vasomotor symptoms—such as hot flushes—linked to menopause. The decision is supported by findings from the OASIS clinical trial program, which enrolled over 1,400 women aged 40 to 65 across multiple countries. Results showed that a daily 120 mg dose of elinzanetant significantly reduced the frequency and severity of hot flushes and night sweats over a period of 26 to 52 weeks, compared to a placebo.
Proximie and HistoSonics Announce US Partnership to Transform Liver Tumour Treatment
On 16th July, Proximie, a global health technology leader focused on digitising operating rooms, announced a U.S. partnership with HistoSonics, the company behind the Edison® Histotripsy System and innovative non-invasive histotripsy therapy platforms for treating liver tumours.
One Biosciences Secures €15 Million Series A to Advance Clinical-Grade Single-Cell Profiling
On 17th July, precision oncology techbio company One Biosciences announced the closing of a €15 million Series A funding round. The round was led by Redmile Group and Blast, with additional support from Galion.exe, Invus, Adamed Technology, Sofinnova Partners, Polytechnique Ventures, and Kima Ventures. The funding will accelerate the development of the company’s clinical-grade single-cell tumour transcriptomic profiling platform.
eXmoor Pharma and Signadori Bio Partner to Advance Next-Gen Monocyte-Based Cell Therapy for Solid Tumours
On 17th July, eXmoor Pharma announced a collaboration with French biotech firm Signadori Bio to accelerate the development of gene-modified, monocyte-derived macrophage cell therapies aimed at treating solid tumours.
MHRA Approves UK’s First Needle-Free Emergency Treatment for Anaphylaxis
On 18th July, the MHRA approved adrenaline (epinephrine) nasal spray (EURneffy) to be used for the emergency treatment of serious allergic reactions, known as anaphylaxis. It is intended for use in adults and children who weigh 30 kg (about 66 pounds) or more.
MHRA’s 2024–25 Annual Report and Accounts and Impact Report Show Progress on Safety and Regulatory Excellence
On 21st July, the MHRA published its 2024–25 Annual Report and Accounts, and accompanying Impact Report, demonstrating how we have enhanced patient safety across the UK, restored our performance to ensure we are meeting regulatory timelines, and sharing our success in enabling access to life-changing medical products.
MHRA Issues Essential Advice on Protecting Your Medicines During Extreme Heat
On 23rd July, the MHRA reinforced essential advice for anyone using medicines or medical devices. Advice includes checking devices in the heat, being smart about sun-sensitising medicines and to report any safety issues via the yellow card scheme.
FDA Investigating Death of 8-Year-Old Boy Who Received Elevidys
On 25th July, the FDA announced it is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy. The agency has requested and received voluntary suspension of product distribution as it investigates the safety concerns.
EMA Recommends Approval of Aqneursa for Niemann-Pick Type C Disease
On 25th July, the EMA recommended granting marketing authorisation for Aqneursa (levacetylleucine) in the EU to treat neurological symptoms of Niemann-Pick type C (NPC) disease. The treatment is intended for adults and children aged six and older who weigh at least 20 kg. Aqneursa may be used alongside miglustat or as monotherapy for patients who cannot tolerate miglustat.
EMA Backs First Reformulation of Inhaled Medicine Using Eco-Friendly Propellant
On 25th July, the EMA recommended a compositional change for Trixeo Aerosphere and its duplicate, Riltrava Aerosphere, to replace their current gas propellant with a low global warming potential (GWP) alternative. The new propellant offers a 1,000-fold reduction in GWP while maintaining similar physical characteristics to the original, marking a significant step toward more environmentally sustainable inhaled medicines.
EMA Releases Recording of CTIS Training Material Redesign Webinar
The EMA published the video recording of the Clinical Trials Information System (CTIS) bitesize talk on the redesign of the CTIS training material for sponsor users. The event, held on the 9th of July 2025, was an opportunity to become acquainted with the revised Sponsor Handbook, which will serve as the definitive reference for sponsor users operating within CTIS. Effective 9 July, it will supersede the existing sponsor training modules.
EMA Shares Highlights from 30th Anniversary Scientific Conference
The EMA has published the video recording of the Clinical Trials Information System (CTIS) bitesize talk focused on the redesign of training materials for sponsor users. Held on July 9th, 2025, the event introduced the updated Sponsor Handbook, which will become the primary reference guide for sponsors using CTIS. As of July 9th, the new handbook replaces the previous sponsor training modules.
MHRA Approves Tofersen for Rare Inherited Form of Motor Neurone Disease
On 28th July, the MHRA approved tofersen (Qalsody) on 22nd July 2025 to treat adults with amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 (an enzyme called superoxide dismutase 1) gene, a rare, inherited form of motor neurone disease (MND).
NHS Launches Ground-breaking Review to Address LGBT+ Health Inequalities
On 29th July, the NHS announced the initiation of its first-ever review aimed at tackling health disparities faced by LGBT+ individuals, focusing on improving access to mental health services, cancer screening, and primary care.
Our Short Guide to Clinical QMS
A Quality Management System (QMS) is a structured framework implemented to manage and maintain an organisation’s quality objectives and expectations. It is pivotal in ensuring participant safety, data integrity, and compliance with regulatory standards.
In this short guide, Senior Associate, Rita Silvestre, introduces the fundamentals of QMS, focusing on its application in clinical settings.
Issue Number: WN00200
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