The MHRA has clarified the UK licensing pathway for microbiome medicines, explaining how developers can pursue licensing under existing rules.
Microbiome-based medicines work by modulating, restoring or replacing the human microbiome.
The MHRA said the products fall within the existing UK medicines framework under the Human Medicines Regulations 2012.
Depending on their characteristics, they may be regulated as biological medicinal products or, in some cases, as advanced therapy medicinal products, known as ATMPs.
No microbiome-based medicinal product currently has UK marketing authorisation, although some countries have authorised a small number of donor-derived microbiota products for Clostridioides difficile infection.
The MHRA said developers should consider product characterisation, manufacturing consistency, control of differences between production batches, safety assessments and the generation of robust clinical evidence.
Safety assessments should also consider the potential risk of antimicrobial resistance.
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 regulator is encouraging developers to use its Innovation Office and Scientific Advice services early in development to clarify which regulatory framework applies, agree proportionate development strategies and reduce investment risks ahead of pivotal trials.
The paper also clarifies existing arrangements for faecal microbiota transplantation, or FMT, in the UK.
FMT is currently used for specific indications, particularly recurrent C. difficile infection.
It can be supplied through clinical trials or as an unlicensed medicine under MHRA “specials” manufacturing arrangements or extemporaneous preparation.
The MHRA said those existing routes remain under the direct responsibility of the prescribing clinician and are unaffected by the position paper.
The regulator also highlighted international regulatory developments, including the EU’s Substances of Human Origin Regulation, which will introduce a distinct framework for intestinal microbiota interventions in the EU.
