Each month, we scan the regulatory landscape to bring you the latest developments shaping pharmaceutical and biotechnology innovation. In this roundup, you’ll find key publications that caught our attention, plus a curated summary of the most important regulatory news. Our goal is to highlight what matters, cut through the noise, and keep you up to date on changes that could impact drug development and market access in the UK, Europe and the US.
Regulatory Affairs News
News: The EMA stepped up gaps in medicines for women’s health
The EMA is launching new initiatives to better integrate women’s health into medicines development and regulation, addressing gaps in prevention, diagnosis and treatment. A dedicated workshop on 28–29 September will bring together regulators, researchers, healthcare professionals, patients and industry to identify research priorities and promote more inclusive clinical research. EMA’s ongoing work focuses on improving women’s representation in clinical trials, assessing and communicating sex-specific differences in medicines, strengthening evidence on medicine use during pregnancy and breastfeeding, and expanding the use of real-world evidence through the Data Analysis and Real World Interrogation Network, DARWIN EU®. Future efforts will encourage earlier regulatory engagement, strengthen international collaboration, identify research gaps and support the development of medicines that better meet women’s specific health needs.
News: The FDA approved Casgevy for children 2 years and older with sickle cell disease
The FDA granted expanded approval of Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with sickle cell disease (SCD) and recurrent vaso-occlusive crises (VOCs), or transfusion-dependent β thalassemia (TDT). It is the first gene therapy approved for children as young as 2 years with SCD. Casgevy is a one-time, autologous stem cell therapy that uses CRISPR/Cas9 gene editing to increase foetal haemoglobin (HbF), helping prevent sickled red blood cells in SCD and eliminating the need for regular transfusions in TDT. Treatment requires full myeloablative conditioning beforehand. Approval was supported by clinical trial data in children aged 5 to under 12 years, with all evaluable SCD patients free from severe VOCs for at least 12 months and most evaluable TDT patients achieving sustained transfusion independence.
News: The FDA proposed a rule to modernise drug manufacturing registration
The FDA proposed a rule to simplify registration for distributed drug manufacturing establishments operating under a “hub-and-spoke” model by allowing them to register as a single establishment rather than individually. The proposal would also streamline updates when manufacturing units are added, relocated or removed, while requiring advance notification of relocations to improve FDA oversight. In addition, the rule would clarify registration and drug listing requirements for certain foreign manufacturers, including those producing active pharmaceutical ingredients that indirectly enter the U.S. supply chain. By increasing visibility across domestic and international manufacturing networks, the proposal aims to strengthen regulatory oversight, improve supply chain transparency and help ensure Americans have reliable access to safe, high-quality medicines.
News: The MHRA published guidance on its Innovation Accelerator programme
The MHRA published guidance on its Innovation Office, which provides regulatory support for developers of novel medicines, medical devices and technologies that challenge existing regulatory frameworks. The guidance directs developers to resources on determining whether a product is regulated as a medicine or medical device, including support for borderline and hybrid products. It also outlines available pathways for medicines development, including Scientific Advice, clinical trial guidance, licensing and early access schemes. The MHRA emphasises that the Innovation Office complements, but does not replace, formal Scientific Advice, which is available throughout product development, with fee waivers for eligible UK-based small and medium-sized enterprises.
News: The British Medical Journal reported on the impact of the US-UK trade deal
An analysis published in The BMJ suggests the UK-US pharmaceuticals trade deal could divert around £45bn from other National Health Service (NHS) services by 2036 unless additional funding is provided. The authors estimate this could contribute to 229,000 excess preventable deaths, rising to 291,000 when adult social care impacts are included, mainly affecting people with cardiovascular, respiratory, gastrointestinal diseases and cancer. While the deal secures 0% tariffs on UK pharmaceutical and medical device exports to the US for 3 years, it also commits the NHS to higher spending on branded medicines by increasing the National Institute for Health and Care Excellence’s cost-effectiveness thresholds, giving greater weight to medicine benefits, and reducing industry rebate payments under VPAG. NHS spending on new medicines is set to more than double to 0.6% of gross domestic profit by 2036, adding £44.7bn in cumulative costs. The authors call for publication of the deal’s impact assessment, arguing its projected health and economic benefits remain uncertain.
News: National Institute for Health and Care Excellence (NICE) issued new guidance on polyendocrine metabolic ovarian syndrome (PMOS)
People with PMOS should receive earlier diagnosis and an annual review to monitor symptoms, treatment and long-term health risks, according to a draft NICE guideline. Formerly known as polycystic ovary syndrome (PCOS), the new name reflects the condition’s broad hormonal, metabolic and reproductive effects. Affecting around 1 in 8 women, PMOS is often under-diagnosed despite being associated with serious health risks, including type 2 diabetes, cardiovascular disease, sleep apnoea, fatty liver disease, mental health problems and pregnancy complications. The NICE guideline recommends investigating anyone with irregular or absent menstrual cycles and signs of excess male hormones and advises that PMOS should not be ruled out after menopause. Annual reviews should assess symptoms, medicines and risks of long-term conditions, while also addressing mental health, fertility and lifestyle factors. The guideline also provides recommendations on fertility treatment and management of common symptoms, including acne, excess hair growth and sleep apnoea.
Publications that caught our eye
- MHRA’s radical rare disease proposal. Nature biotechnology.
- Discordance in orphan drug approvals between the U.S. Food and Drug Administration and the European Medicines Agency: A retrospective observational analysis. Plos Medicine.
- Assessment of United States Food and Drug Administration Approval of Cell and Gene Therapies. Clinical Pharmacology & Therapeutics.
- AI might help the NHS – but we need to build the evidence. British Medical Journal.
- Major objections in EU medicines authorisation applications: an analysis of dossier submissions by small- and medium-sized enterprises. Drug Discovery Today.
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