Advanced therapy medicinal products: regulation and licensing in UK
Source: gov.uk (2025-03-06)

How to get a marketing authorisation (MA) for an advanced therapy medicinal product (ATMP) so it can be sold and/or supplied in the United Kingdom.

This page is for licensing ATMPs only. For biosimilars, see Guidance on the licensing of biosimilar products (https://www.gov.uk/government/publications/guidance-on-the-licensing-of-biosimilar-products). for PMFs and VAMFs, see Licensing plasma master files and vaccine antigen master files (https://www.gov.uk/guidance/licensing-plasma-master-files-and-vaccine-antigen-master-files).

1. Overview

An advanced therapy medicinal product (ATMP) is a medicinal product which is either:
- a gene therapy medicinal product
- a somatic cell therapy medicinal product
- a tissue engineered product
The definition of ATMP is in the Human Medicines Regulations 2012 and includes combination ATMPs.

In the UK, the MHRA is the competent authority:
- for all medicinal products, including ATMPs
- for UK manufacturers or importers of ATMPs
2. Getting a classification opinion or advice about ATMPs

If you are not sure if your product is an ATMP or which type of ATMP it is, refer to this flowchart (https://assets.publishing.service.gov.uk/media/65f962bca4d4f3001a0352cc/ATMP_Flowchart_V6.xlsx) (PDF, 32.1KB, 5 pages). If you are still uncertain, contact the MHRA Innovation Office by either:
- completing the innovation office enquiry form (http://info.mhra.gov.uk/forms/innovation_form.aspx) or
- emailing xxx@xxx.xx (mailto:xxx@xxx.xx).
The MHRA will classify your product as an ATMP where appropriate.

The European Medicines Agency (EMA) ATMP classification website (https://www.ema.europa.eu/en/human-regulatory-overview/marketing-authorisation/advanced-therapies-marketing-authorisation/advanced-therapy-classification) provides useful information, including:
- examples of products that have been classified
- a ‘Reflection paper on classification of advanced therapy medicinal products’ outlining the scientific and regulatory principles as applied to the classification of ATMPs
These are also applicable to the UK.

3. Regulatory queries on regenerative medicines (one-stop shop)

All regulatory enquiries about regenerative medicines should be made through the MHRA Innovation Office (https://www.gov.uk/government/groups/mhra-innovation-office).

The Innovation Office is the single point of contact for all the regulators involved in regenerative medicines:
- the Human Tissue Authority (HTA)
- the Human Fertilisation and Embryology Authority (HFEA)
- Health Research Authority (HRA)
- the MHRA
To contact the innovation office, either:
- complete the innovation office enquiry form (http://info.mhra.gov.uk/forms/innovation_form.aspx) or
- email xxx@xxx.xx (mailto:xxx@xxx.xx)
4. Scientific advice

You can get scientific advice from MHRA (https://www.gov.uk/guidance/medicines-get-scientific-advice-from-mhra) at any stage of ATMP development. There is usually a fee for this service. See Current MHRA fees (https://www.gov.uk/government/publications/mhra-fees/current-mhra-fees#scientific-advice-meetings-fees).

5. Apply to conduct a clinical trial for an advanced therapy medicinal product

All ATMPs must undergo clinical trials (https://www.gov.uk/government/collections/clinical-trials-for-medicines) in the same way as all other medicines. Applications for clinical trial approvals in the UK should be made to the MHRA. See Clinical trials for medicines: apply for authorisation in the UK (https://www.gov.uk/guidance/clinical-trials-for-medicines-apply-for-authorisation-in-the-uk#when-a-clinical-trial-authorisation-cta-is-needed).

6. Apply for a marketing authorisation for an ATMP

All ATMPs to be placed on the market in the UK must have a marketing authorisation (MA). The MHRA is responsible for assessing the quality, safety and efficacy of each ATMP that needs an MA. See the MHRA guidance on the application process in the UK (https://www.gov.uk/guidance/apply-for-a-licence-to-market-a-medicine-in-the-uk) for further information.

Following the implementation of the Windsor Framework on 1 January 2025, applications for MAs covering Great Britain only will no longer be possible. All MA applications for ATMPs must be UK-wide. Refer to additional guidance: UK-wide licensing for human medicines - GOV.UK (https://www.gov.uk/government/publications/uk-wide-licensing-for-human-medicines).

7. Combination ATMPs

A combination ATMP includes 1 or more medical devices or active implantable medical devices as integral components of the product. The MHRA evaluates combined ATMPs for MAs and UK clinical trials.

The application for an MA for a combined ATMP which contains a medical device should include evidence that the device meets the essential requirements set out in the UK Medical Devices Regulations (SI 2002 No 618, as amended) and, where available, the results of an assessment by an approved body or an EU notified body. See the MHRA guidance on the regulation of medical devices in the UK (https://www.gov.uk/guidance/regulating-medical-devices-in-the-uk) for further information.

8. Applying for a manufacturer’s licence for an ATMP

For advice about getting a manufacturer’s licence from the MHRA, refer to the MHRA guidance on licences to manufacture (https://www.gov.uk/government/collections/licences-to-manufacture-or-wholesale-in-medicines) or email xxx@xxx.xx (mailto:xxx@xxx.xx).

9. Manufacture of unlicensed ATMPs in the UK

There are 2 ways in which unlicensed ATMPs can be made available in the UK:
- hospital exemption
- the ‘specials’ scheme
Hospital exemption applies for ATMPs which are prepared on a non-routine basis and are used in a hospital with a prescription for a specific patient. To apply for a hospital exemption complete the hospital exemption form (https://www.gov.uk/government/publications/medicines-application-forms-for-a-manufacturer-licence) and email it to xxx@xxx.xx (mailto:xxx@xxx.xx).

See guidance on the UK’s arrangements under the hospital exemption scheme (https://assets.publishing.service.gov.uk/media/67c9ca8aae2aa47d2f5ae3e6/26_a_Guidance_on_the_UK_s_arrangements_under_the_hospital_exemption_scheme_clean__1_.pdf) (PDF, 164 KB, 7 pages).

The ‘specials’ scheme applies to all types of medicinal products. Refer to the MHRA guidance on applying for licences (https://www.gov.uk/guidance/apply-for-manufacturer-or-wholesaler-of-medicines-licences).

Manufacturers need a manufacturer’s licence from the MHRA to use either scheme.

See non-routine guidance on ATMPs (https://assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/xx-xxxx-xxxx/Non-routine_guidance_on_ATMPs.pdf) (PDF, 661KB, 3 pages).

10. Human tissues and cells in ATMPs

If tissues and cells are being used as starting materials in a medicinal product, the donation, procurement and testing of the cells are covered by the The Human Tissue (Quality and Safety for Human application) Regulations 2007. Under this regulation there are two competent authorities.

The Human Fertilisation and Embryology Authority (HFEA) (http://www.hfea.gov.uk/) is the competent authority for the use of gametes and embryos, which may be used in the derivation (development) of cells in the manufacture of ATMP.

The Human Tissues Authority (HTA) (https://www.hta.gov.uk/) is the competent authority for the licensing and inspection for all other tissues and cells. If a human embryonic stem cell line is derived in the UK, a sample of the resulting cell line must be deposited at the UK Stem Cell Bank.

Once the starting materials have been made available, medicines legislation applies and is regulated by the MHRA. Advice on the regulatory routes is available from the Innovation Office (https://www.gov.uk/government/groups/mhra-innovation-office).

The MHRA and Human Tissues Authority (HTA) work closely together in this area.

HTA and the MHRA have agreed that the collection of blood as a starting material for an ATMP can be carried out under either a tissues and cells licence or a blood establishment licence. See the joint HTA and MHRA position (https://www.hta.gov.uk/guidance-professionals/regulated-sectors/human-application/regenerative-medicine-and-regulation).

11. Blood and blood components in medicinal products

If blood and blood components are used as starting materials in a medicinal product, the donation and testing are covered by the Blood Safety and Quality Regulations 2005. The competent authority for blood product regulation in the UK is the MHRA.