Our weekly newsletter provides you with the latest industry news, developments and regulatory agency updates.

Company Updates

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Regulatory Updates

MHRA issues tips for staying healthy and safe this festive season

With the festive season in full swing, the Medicines and Healthcare products Regulatory Agency (MHRA), published tips to help you stay healthy and safe. These include ensuring you have enough medicine supplies, looking after your mental health and reporting any concerns with medicines or medical devices.

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MHRA guidance updates

  • Windsor Framework explainer. On 6th December, updated guidance for industry on the main changes introduced by the Windsor Framework on the supply of medicines in Northern Ireland. Click to view source
  • Apply for a licence to market a medicine in the UK. On 11th December, guidance on the process of applying for a license to market in the UK was updated, including links to further guidance documents. Click to view source

ICH announces publication of updated Q9(R1) Annex 1- Q8/Q9/Q10 Questions & Answers

On 6th December, the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Secretariat announced the publication of the updated Q9(R1) Annex 1- Q8/Q9/Q10 Questions & Answers (R5) related to ICH Q9(R1) Quality Risk Management (QRM). The ICH Assembly approved this updated Q9(R1) Annex 1 on 30th October 2024.

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FDA leaders discuss role in revising Declaration of Helsinki in new publication

On 9th December, the US Food & Drug Agency’s (FDA) Office of Global Policy and Strategy published a conversation with FDA Chief Medical Officer Dr Hilary Marston and Ann Meeker-O’Connell, director of the FDA’s Office of Clinical Policy, on the FDA’s role in the revision of the Declaration of Helsinki, the World Medical Association’s ethical principles for human studies.

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FDA guidance updates

  • Standardised Format for Electronic Submission of NDA and BLA Content for Planning Bioresearch Monitoring (BIMO) Inspections for CDER Submissions Guidance for Industry. On 6th December, the FDA published final guidance to describe the electronic submission of certain data and information in standardised formats. This information is used by the Center for Drug Evaluation and Research (CDER) in the planning of, and by the FDA’s Office of Inspections and Investigations (OII) in the conduct of, BIMO inspections.
  • Guidance for Industry: Registration and Listing of Cosmetic Product Facilities and Products. On 11th December, final guidance was published with recommendations and instructions to assist persons submitting cosmetic product facility registrations and product listings to the FDA.

Industry Updates

EMA issues meeting highlights from latest Committee for Veterinary Medicinal Products

On 6th December, the European Medicines Agency (EMA) issued meeting highlights from the Committee for Veterinary Medicinal Products (CVMP), held on the 3rd-5th December 2024. Highlights include opinions on veterinary medicinal products, the issue of a certificate for a vaccine platform technology master file (vPTMF) and the adoption of VICH GL8(R) on Stability testing for medicated premixes for release for 5 months of public consultation.

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Datopotamab Deruxtecan receives breakthrough therapy designation for advanced EGFR-Mutated NSCLC

On 9th December, AstraZeneca announced that its Datopotamab deruxtecan (Dato-DXd) has been granted Breakthrough Therapy Designation (BTD) in the US for the treatment of adult patients with locally advanced or metastatic epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC) with disease progression on or after treatment with an EGFR-tyrosine kinase inhibitor (TKI) and platinum-based chemotherapy. The FDA’s BTD is designed to accelerate the development and regulatory review of potential new medicines that are intended to treat serious conditions and address significant unmet medical needs.

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iOnctura reaches new clinical milestones in uveal melanoma

On 11th December, iOnctura, a clinical-stage biopharmaceutical company combating neglected and hard-to-treat cancers, announced promising results from the phase 1 DIONE-01 study, showcasing the clinical activity and long-term safety of roginolisib in patients with uveal melanoma. The study demonstrated that patients with uveal melanoma treated with roginolisib had a median overall survival of 16 months, significantly exceeding the 7 months observed with previous treatments. The findings support the continued development of roginolisib, with the activation of trial sites for the phase 2 OCULE-01 study already underway. This next phase will investigate roginolisib versus the investigator’s choice in second-line treatment of uveal melanoma.

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Minoryx and Neuraxpharm announce NEXUS trial success

On 12th December, Minoryx Therapeutics, a late-stage biotech specializing in therapies for orphan CNS disorders, and Neuraxpharm Group, a pharmaceutical company focused on CNS treatments, announced the completion of the NEXUS trial, with the primary endpoint achieved. The companies plan to submit a filing for EU marketing authorisation of leriglitazone for paediatric and adult patients with cerebral Adrenoleukodystrophy (cALD) by mid-2025.

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MHRA issues latest Drug Safety Update

On 12th December, the MHRA issues its December edition of the Drug Safety Update newsletter from the agency and its independent advisor, the Commission on Human Medicines.

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Syndax reports positive data for Revuforj® in AML trials

On 9th December, Syndax Pharmaceuticals, a biopharmaceutical company focused on developing cancer therapies, announced positive new data from the AUGMENT-101 trial of Revuforj® (revumenib) in relapsed or refractory (R/R) mutant NPM1 (mNPM1) acute myeloid leukaemia (AML). Additionally, the company shared results from the BEAT AML trial of revumenib combined with venetoclax and azacitidine in newly diagnosed AML patients. Revuforj is the company’s FDA-approved, first-in-class oral menin inhibitor, approved for treating R/R acute leukaemia with a KMT2A translocation in patients aged one and older.

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Issue Number: WN00192

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