Регулятор Великобритании MHRA утвердил четкие правила регистрации препаратов на основе микробиома

The Medicines and Healthcare products Regulatory Agency has published a position paper clarifying how microbiome‑based medicines can be made available for patients in the UK. The paper encourages developers to pursue UK licensing pathways for this emerging class of therapies. Microbiome‑based medicinal products are medicines that work by modulating, restoring or replacing the human microbiome. They represent a promising new category of therapies with the potential to address areas of significant unmet clinical need such as antimicrobial resistance. The MHRA’s position is that MBMPs fall within the scope of the existing UK medicines regulatory framework set out in the Human Medicines Regulations 2012. This provides developers with a clear route to licensing while maintaining the same robust standards of quality, safety and efficacy that apply to all medicines in the UK. Depending on their characteristics, MBMPs may be regulated as biological medicinal products or may meet the criteria for advanced therapy medicinal products. No MBMP currently holds a UK marketing authorisation. Some countries have authorised a small number of donor‑derived microbiota products for Clostridioides difficile infection. The position paper sets out key scientific and regulatory steps developers should consider, including product characterisation, manufacturing consistency, control of batch‑to‑batch variability, safety assessment including antimicrobial resistance risk, and generation of robust clinical evidence. Julian Beach, Executive Director of Healthcare Quality and Access at the MHRA, said: “Microbiome-based medicinal products represent one of the most interesting and fast-moving areas in medicine today. Our position paper gives developers the clarity they need to bring these innovations forward with confidence, while ensuring patients continue to be protected by the same rigorous standards that underpin all licensed medicines in the UK. “The UK’s existing, proportionate and evidence-based licensing frameworks are ready to support this field. Early engagement with us is the best way for developers to navigate it successfully.” The MHRA is encouraging developers to make use of its Innovation Office and Scientific Advice services at an early stage to clarify the applicable regulatory framework, agree proportionate development strategies and de‑risk investment decisions ahead of pivotal trials. The position paper also clarifies existing arrangements for faecal microbiota transplantation in the UK. FMT is currently used for specific indications, most notably recurrent Clostridioides difficile infection, and is supplied either through clinical trials or as an unlicensed medicine under MHRA ‘specials’ manufacturing arrangements or extemporaneous preparation. These routes remain under the direct responsibility of the prescribing clinician and are unaffected by the publication. The MHRA also notes ongoing developments internationally, including the EU’s Substances of Human Origin Regulation, which will introduce a distinct framework for intestinal microbiota interventions in the EU.