Company News
RQA European QA Virtual Conference
We are thrilled to announce that our Head of Quality Assurance, Shona Ross, and two of our Senior Associates, Anna Lothian and Nicole Ainsworth will be participating in the upcoming 2025 RQA European QA Virtual Conference on the 20th-22nd May. For more information on the event, click here.
New Course Alert: ICH Good Clinical Practice (GCP) E6 (R3)
We are delighted to introduce our new ICH Good Clinical Practice (GCP) E6 (R3) course. If you are involved in clinical research or clinical trials, this course is designed to help you stay ahead of the curve. It provides comprehensive knowledge of the updated ICH GCP E6 (R3) regulations, ensuring you understand the latest developments and their implications for your role in clinical trials. Upon completion of the course, you will have a clear understanding of the end-to-end clinical drug testing process, including risk management and the considerations in the identification of critical to quality factors and quality by design.
We offer flexible training options, available for individuals or groups, allowing you to schedule your training at your convenience.
If you’re interested, please reach out to us at training@towermains.com or click here for more information.
Regulatory News
EMA Proposes Streamlined Development Pathway for Biosimilars
On 1st April, the European Medicines Agency (EMA) published a draft reflection paper proposing a simplified approach to biosimilar development. The new framework suggests that structural, functional, and pharmacokinetic data may be sufficient to demonstrate biosimilarity, potentially removing the need for large-scale clinical efficacy trials. The goal is to reduce development time and costs while maintaining safety and efficacy standards. Public consultation on the proposal is open until 30 September 2025.
EMA’s Clinical Trials Information System Recognised as WHO Primary Registry
On 3rd April, the EMA’s Clinical Trials Information System (CTIS) was officially recognised as a primary registry by the World Health Organisation (WHO). This designation confirms CTIS meets WHO standards for quality, transparency, and accessibility, allowing its data to be included in the WHO’s global clinical trials platform. The move supports international research visibility and meets key publication requirements set by major medical journals.
MHRA to Play a Pivotal Role in Ensuring the Quality of Global Biological Medicines
On 9th April, the World Health Organisation (WHO) confirmed the successful redesignation of the Medicines and Healthcare products Regulatory Agency (MHRA)’s Science and Research group as one of its Collaborating Centres for the Standardisation and Evaluation of Biologicals for the next four years.
First-ever MHRA Analysis of UK Trial Applications
On 10th April, the MHRA and University of Liverpool published the first detailed analysis of UK clinical trial applications in the British Journal of Clinical Pharmacology. It highlights strong innovation, with one in eight trials being first-in-human and 85% industry-led. However, research is heavily focused on cancer, with less attention to areas like heart disease and mental health. The report also calls for greater diversity in trial participants.
Major Overhaul of UK Clinical Trials Framework Now Official
On 11th April, the UK government enacted the most significant reform of clinical trial regulations in 20 years. Developed by the MHRA in collaboration with the Health Research Authority (HRA), the updated framework is designed to simplify trial processes, cut red tape, and improve patient safety. Key changes include a unified ethics and regulatory review system, a risk-based regulatory approach, and a goal to shorten the timeline from application to first participant from 250 to 150 days. The new system will be fully rolled out by 10th April 2026.
EMA and HMA Issue Recommendations to Strengthen EU Radiopharmaceutical Supply Chain
On 14th April, the EMA and the Heads of Medicines Agencies (HMA) issued recommendations to address vulnerabilities in the EU’s radiopharmaceutical supply chain. These medicines are crucial for diagnosing and treating conditions like cancer but face risks due to limited production capacity and reliance on non-EU sources. The guidance calls for boosting domestic manufacturing, upgrading infrastructure, and improving cross-border transport coordination to help prevent shortages and ensure patient care continuity.
New Appointment to Commission on Human Medicines as Three Members Renew Terms
On 17th April, Professor Claire Stewart was appointed to the Commission on Human Medicines (CHM) for a four-year term starting 3rd March 2025. Alongside her appointment, Professors Amanda Adler, Steve Cunningham, and Yvonne Perrie were reappointed to the CHM, with new terms beginning on 1st May 2025.
EMA Publishes EudraVigilance Expert Working Group Work Programme for 2025-2026
On 22nd April, the EMA released the EudraVigilance Expert Working Group (EV-EWG) Work Programme for 2025–2026. The programme outlines key activities, including improving the EudraVigilance system, developing policies and guidance, and supporting data analysis and signal detection. The EV-EWG will also contribute to international standardisation and data protection in pharmacovigilance.
EMA Management Board Recommends Renewal of Executive Director Emer Cooke’s Mandate
On 28th April, the EMA Management Board unanimously recommended the renewal of Emer Cooke’s mandate as Executive Director until her pension age on 1 May 2027. This recommendation follows a consultation request from the European Commission, which supports the renewal. If approved by the College of Commissioners, the proposal will return to the EMA Management Board for formal approval. The final decision is expected later in 2025.
MHRA Safety Roundup: April 2025
On 29th April, the MHRA issued its April safety round-up, the summary of the latest safety advice for medicines and medical device users
EU Publishes Materials from Session on Critical Medicines Act Proposal
On 30th April, the European Commission released materials from the online session held on 29th April regarding the proposed Critical Medicines Act. The Act seeks to ensure a stable supply of essential medicines across the EU by addressing manufacturing and supply chain challenges, with provisions for strategic projects and collaborative procurement to enhance availability.
Published Guidance
Medicines and Healthcare products Regulatory Agency (MHRA)
- 3rd April: National assessment procedure for medicines. Guidance was published on the MHRA’s national assessment procedure for marketing authorisation (MA) applications. This procedure applies to both innovative and established medicines, with distinct requirements and timelines for each.
- 3rd April: Submission and assessment timetables for innovative medicines applications. Guidance on the deadlines for initial and response submissions.
- 16th April: Risk minimisation measures for medicines. Guidance was published for healthcare professionals on risk minimisation options for medicines. Risk minimisation measures facilitate the safe and effective use of medicines by healthcare professionals, patients, and their carers or guardians.
European Medicines Agency (EMA)
- 29th April: Clinical Trials Information System. EMA introduced a new “CTIS Simplification Task Force” section on its website. This task force aims to streamline the Clinical Trials Information System (CTIS), simplifying processes and improving user experience for clinical trial sponsors and regulators.
- 30th April: ICH Q1 Guideline on stability testing of drug substances and drug products. The draft ICH Q1 guideline on stability testing for drug substances and products has been made available for public consultation. It provides key principles for stability studies, covering aspects such as stress testing, protocol design, and data evaluation, to help determine re-test periods and shelf lives for pharmaceutical products. Stakeholders are encouraged to submit their comments by 30th July 2025.
Updated Guidance
MHRA
- 2nd April: AI Airlock: the regulatory sandbox for AIaMD. Guidance was updated to include a link to a blog about AI Airlock.
- 4th April: The Innovative Devices Access Pathway (IDAP). Guidance on the Innovative Devices Access Pathway (IDAP) pilot was updated to add a note that the iDAP pilot phase and Patient and public engagement in iDAP are now closed.
- 4th April: Early Access to Medicines Scheme: Overview. New ‘Dates for submission, Day 1 and Day 45’ were added.
- 8th April: MHRA phase I accreditation scheme. Updated to add updated versions of the phase I accreditation scheme requirements, guidance on the phase I accreditation scheme and the list of accredited phase I units
- 10th April: Clinical investigations for medical devices. Updated to add new guidance for manufacturers document.
Industry News
Elekta and Azra AI Partner to Enhance Cancer Registry Operations with AI Automation
On 3rd April, Elekta announced a partnership with Azra AI to improve cancer registry operations through AI-powered automation. The collaboration aims to streamline and enhance the accuracy of cancer data collection by integrating Azra AI’s technology into Elekta’s oncology ecosystem. This move is expected to reduce the manual workload of cancer registrars, speed up reporting processes, and support better clinical decision-making, ultimately leading to improved cancer care delivery.
Eplontersen Approved to Treat Adults With Rare Inherited Nerve Disease or Polyneuropathy
On 4th April, the MHRA approved eplontersen (Wainzua) to treat adults with polyneuropathy associated with hereditary transthyretin amyloidosis (ATTRv), which is a rare progressive condition that runs in families caused by a protein called transthyretin (TTR).
AI Tool Enhances Monitoring of Multiple Sclerosis Treatment Effectiveness
On 7th April, researchers at University College London (UCL) Queen Square Institute of Neurology introduced an AI tool named MindGlide, designed to assess the effectiveness of multiple sclerosis (MS) treatments. This tool analyses brain MRI scans to detect and measure lesions and subtle changes such as brain shrinkage, providing insights into disease progression and treatment response. In a study published in Nature Communications, MindGlide demonstrated superior performance over existing AI tools, identifying abnormalities with greater accuracy and processing images in just five to ten seconds. The researchers anticipate that MindGlide will enable the use of existing brain images in hospital archives to better understand MS and how treatments affect the brain, leading to valuable insights for researchers and clinicians.
SkyCell and Microsoft Collaborate to Integrate Co-pilot into Pharma Supply Chains
On 7th April, SkyCell announced a collaboration with Microsoft to integrate its AI-powered supply chain solution, SkyMind, with Microsoft Teams and Co-pilot via Azure OpenAI Service. This integration aims to enhance pharmaceutical cold chain logistics by providing real-time shipment data, predictive insights, and automated alerts within the existing Microsoft ecosystem. The intelligent assistant, K.AI, continuously monitors shipments, assesses risks, and suggests solutions in real time, facilitating seamless communication among stakeholders and improving efficiency, compliance, and decision-making.
Support for New Test to Monitor People at Risk of Multiple Myeloma
On 14th April, the MHRA approved eplontersen (Wainzua) to treat adults with polyneuropathy associated with hereditary transthyretin amyloidosis (ATTRv), which is a rare progressive condition that runs in families caused by a protein called transthyretin (TTR).
EU Approves Leqembi for Early Alzheimer’s Treatment
On 15th April, the European Commission authorised Leqembi (lecanemab) for early-stage Alzheimer’s patients with amyloid plaques. The approval is based on positive EMA findings, with strict monitoring for side effects like brain swelling. This treatment marks a significant step forward in targeting the root cause of Alzheimer’s.
WHO Member States Finalise Draft Pandemic Agreement, Set for May Approval
On 16th April, WHO member states concluded negotiations on a draft pandemic agreement to strengthen the global response to future health emergencies. The deal includes commitments on vaccine equity, pathogen sharing, and health system resilience. It will be considered for adoption at the World Health Assembly in May.
Patients Urged to Check Lercanidipine Blood Pressure Medication After Labelling Error
On 17th April, patients taking 10mg Lercanidipine tablets were advised to check their medication packaging due to a labelling error. Some packs were incorrectly labelled as containing 10mg tablets, but they contained 20mg tablets. The affected batch is MD4L07, expiring in January 2028, and was distributed by Recordati Pharmaceuticals.
MHRA Approves Omaveloxolone as First UK Treatment for Friedreich’s Ataxia
On 23rd April, the MHRA approved omaveloxolone (Skyclarys) as the first treatment for Friedreich’s ataxia in patients aged 16 and older. This rare condition causes progressive loss of coordination. In clinical trials, omaveloxolone showed significant improvements in neurological function. The treatment will be available as an oral capsule through NHS prescriptions, with ongoing safety monitoring.
Charcot-Marie-Tooth Association Invests $210K in Long-Read Sequencing to Boost CMT Gene Discovery
On 23rd April, it was announced that the Charcot-Marie-Tooth Association (CMTA) has invested $210,000 in a UCL-led project using long-read genome sequencing to uncover hidden genetic causes of CMT. Led by Dr. Andrea Cortese, the initiative aims to improve diagnosis for patients, especially the 50% with axonal CMT who lack a confirmed genetic explanation.
EMA Recommends First-Ever Treatment for Severe Thyroid Eye Disease
On 25th April, the EMA recommended Tepezza (teprotumumab) for approval as the first-ever treatment for moderate-to-severe thyroid eye disease (TED) in the EU. This monoclonal antibody targets the underlying cause of TED, showing significant improvement in symptoms in clinical trials. While effective, Tepezza may cause side effects, including hearing loss, and will require further assessment by the European Commission.
EMA Greenlights Vamorolone as Breakthrough Treatment for Duchenne Muscular Dystrophy
On 25th April, the EMA announced the approval of vamorolone (Agamree) as the first treatment for all Duchenne muscular dystrophy (DMD) patients, regardless of mutation type. This approval offers an alternative to corticosteroids with fewer side effects for patients aged 4 and older. Additionally, the EMA recommended givinostat (Duvyzat) for patients aged 6 and older who can walk.
Twelve Arrested in Largest-Ever Crackdown on Medicines Trafficking
On 29th April, the MHRA launched its biggest operation yet against organised medicines trafficking, arresting twelve individuals in dawn raids across the West Midlands and Northwest England. The operation, ‘Operation Subaru,’ targeted a network involved in trafficking controlled and unlicensed medicines, resulting in the seizure of hundreds of thousands of doses. The MHRA urged the public to buy medicines only from licensed pharmacies with valid prescriptions.
MHRA Approves Faster-Acting Nivolumab Injection for Cancer Treatment
On 30th April 2025, the MHRA authorised a new under-the-skin version of nivolumab (Opdivo) for the treatment of various cancers, including lung, bowel, kidney, bladder, oesophageal, skin, and head and neck cancers. This formulation allows for a 3–5-minute injection, compared to the previous 30–60-minute intravenous infusion. Clinical trials demonstrated that the subcutaneous injection delivers similar drug levels and safety profiles as the intravenous version.
Questions to Ask Yourself to Ensure Your Lab is Ready for Audit?
You have been notified that your lab has been selected for an audit by a Sponsor of your GLP studies. But how do you ensure you’re fully prepared for when the auditors arrive?
In our latest blog, Nicole Ainsworth shares a set of key questions to ask yourself to ensure thorough preparation and a smooth, successful audit.
Issue Number: WN00197
Would you like a personal copy of the Tower Mains Monthly Newsletter directly to your mailbox each month? Contact us here with the subject “Newsletter Please”