To legally sell or supply an Advanced Therapy Medicinal Product (ATMP) in the United Kingdom, you must obtain a marketing authorisation (MA). This process ensures that the ATMP meets the required standards of safety, quality, and efficacy.
The Medicines and Healthcare Products Regulatory Agency (MHRA) is the competent authority responsible for granting MAs in the UK and released an updated guidance "Advanced therapy medicinal products: regulation and licensing in UK" on 7 March 2025, that details 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.
ATMPs are classified into three main categories:
Gene Therapy Medicinal Products: These involve the transfer of genetic material into cells to treat or prevent diseases.
Somatic Cell Therapy Medicinal Products: These consist of cells or tissues that have been manipulated to alter their biological characteristics or are not intended to be used for the same essential function(s) in the recipient as in the donor.
Tissue-Engineered Products: These contain engineered cells or tissues applied to regenerate, repair, or replace human tissue.
Suppose there is uncertainty regarding whether a product qualifies as an ATMP or its specific classification. In that case, the MHRA offers guidance by consulting the MHRA Innovation Office and by reviewing EMA Resources. For comprehensive regulatory guidance on regenerative medicines, stakeholders can utilize the MHRA Innovation Office as a single point of contact.
The MHRA offers scientific advice services to support the development of ATMPs. This includes guidance on quality, non-clinical, and clinical aspects of product development, ensuring alignment with regulatory requirements.
To market an ATMP in the UK, a Marketing Authorisation (MA) must be obtained from the MHRA. The application process involves:
National Route: If you only intend to market your ATMP in the UK, you can apply directly to the MHRA for a national MA.
European Route (Until Further Notice): While the UK has left the European Union, there may be transitional arrangements or pathways that involve the European Medicines Agency (EMA), particularly if prior procedures have been initiated. Seek updated information from the MHRA.
For ATMPs combined with medical devices, additional regulatory considerations apply. These combination products must comply with both medicinal product and medical device regulations, necessitating coordinated assessments by relevant authorities.
Manufacturers of ATMPs in the UK are required to hold a Manufacturer’s Licence issued by the MHRA. The MHRA provides specific guidance for the manufacture of unlicensed ATMPs, such as those used in hospital exemptions. These products must be prepared on a non-routine basis, comply with specific quality standards, and be used within the same Member State.
ATMPs involving human tissues and cells must comply with the Human Tissue (Quality and Safety for Human Application) Regulations 2007. 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.
For comprehensive and up-to-date information, stakeholders are advised to consult the official MHRA guidance on advanced therapy medicinal products. Also, check out this guidance "UK-wide licensing for human medicines" that was updated recently and details on the implementation of changes to the licensing of medicines for human use in the UK following the agreement of the Windsor Framework.
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