MHRA: UK ATMP Marketing Authorisation Required for Advanced Therapies

• The MHRA is the competent authority for advanced therapy medicinal products (ATMPs) in the UK, responsible for assessing their quality, safety, and efficacy for marketing authorisation.
• From January 1, 2025, all ATMP marketing authorisation applications must be UK-wide, a change mandated by the Windsor Framework.
• Combined ATMPs, which integrate medical devices, require specific evidence that the device components meet UK Medical Devices Regulations (SI 2002 No 618, as amended).
• All ATMPs must undergo clinical trials, with applications submitted to the MHRA, and the British Pharmacopoeia provides quality standards and guidance.
• Unlicensed ATMPs can be made available in the UK through hospital exemption for non-routine preparations used in a hospital with a prescription.

From January 1, 2025, all marketing authorisation applications for advanced therapy medicinal products must cover the entire United Kingdom, eliminating the option for Great Britain-only approvals.

The UK's regulatory landscape for Advanced Therapy Medicinal Products (ATMPs) is overseen by the Medicines and Healthcare products Regulatory Agency (MHRA), which serves as the competent authority. For any ATMP to be legally sold or supplied within the United Kingdom, it must first secure a marketing authorisation (MA). The definition of an ATMP, encompassing combination ATMPs, is established within the Human Medicines Regulations 2012.

Companies developing these innovative products must engage with the MHRA early to ensure proper classification. Should there be any uncertainty regarding a product's status as an ATMP or its specific type, developers can consult a detailed five-page PDF flowchart provided by the MHRA. Further clarification can be sought directly from the MHRA Innovation Office via email or telephone, where the agency will formally classify the product as an ATMP if appropriate. Additionally, the European Medicines Agency (EMA) ATMP classification website offers valuable information that is also applicable within the UK context.

The MHRA is responsible for rigorously evaluating the quality, safety, and efficacy of each ATMP before granting a marketing authorisation. This comprehensive assessment is a prerequisite for all ATMPs intended for the UK market. Detailed guidance on the application process in the UK is available from the MHRA, outlining the necessary steps for securing approval.

A fundamental requirement for all advanced therapy medicinal products is undergoing clinical trials, mirroring the stringent processes applied to other medicinal products. Applications for these clinical trials in the UK must be submitted directly to the MHRA for approval. This ensures that ATMPs meet the necessary safety and efficacy benchmarks before they can be considered for broader market access.

Beyond clinical validation, the British Pharmacopoeia (BP) plays a crucial role in supporting innovation within advanced therapies. It provides public quality standards and non-mandatory guidance specifically designed to aid the development, manufacture, and control of ATMPs. This comprehensive support, including free downloadable ATMP-related guidance, is accessible via the British Pharmacopoeia website, offering valuable resources for developers. Furthermore, the MHRA offers scientific advice at any stage of ATMP development, though this service typically incurs a fee, as detailed in the current MHRA fee schedule.

A significant regulatory shift is set to occur from January 1, 2025, following the implementation of the Windsor Framework. From this date, it will no longer be possible to submit marketing authorisation applications for ATMPs that cover Great Britain only. Instead, all new MA applications for advanced therapy medicinal products must encompass the entire United Kingdom, mandating a UK-wide licensing approach. Additional guidance on this change, particularly concerning UK-wide licensing for human medicines, is available on GOV.UK.

For combination ATMPs, which are defined as products incorporating one or more medical devices or active implantable medical devices as integral components, specific regulatory considerations apply. The MHRA is responsible for evaluating these combined ATMPs for both marketing authorisations and UK clinical trials. An application for an MA for a combined ATMP must include robust evidence demonstrating that the integrated medical device meets the essential requirements outlined in the UK Medical Devices Regulations (SI 2002 No 618, as amended). Where available, results from an assessment by an approved body or an EU notified body should also be provided. Further guidance on the regulation of medical devices in the UK is available from the MHRA.

The MHRA Innovation Office serves as a dedicated single point of contact for all regulators involved in regenerative medicines, streamlining communication for developers. Inquiries to this office can be made via email or telephone. Beyond product classification and marketing authorisation, the MHRA also provides guidance on obtaining a manufacturer’s licence, with specific advice available through their guidance documents or by contacting gmpinspectorate@mhra.gov.uk.

In certain circumstances, unlicensed ATMPs can be made available in the UK through specific pathways. One such route is the hospital exemption, which applies to ATMPs prepared on a non-routine basis. For a product to qualify under this exemption, it must be used within a hospital setting and dispensed with a prescription. This provision offers a controlled mechanism for patient access to advanced therapies outside the standard marketing authorisation process.

Lawyers advising pharmaceutical or biotech clients developing Advanced Therapy Medicinal Products (ATMPs) in the UK must understand the MHRA's detailed requirements for marketing authorisation, including product classification, clinical trials, and the critical shift to mandatory UK-wide licensing from January 2025 due to the Windsor Framework. This guidance helps navigate compliance and market access strategies, particularly for combined ATMPs and those seeking hospital exemption.

Source: Original reporting via GOV.UK guidance