ATMPs (Advanced Therapy Medicinal Products) offer groundbreaking treatments for diseases with unmet medical needs, like genetic disorders and cancer. These therapies include gene therapy, cell therapy, and tissue-engineered products, which provide targeted, often curative solutions.
However, challenges like complex regulations, high costs, and manufacturing difficulties limit their availability. Despite this, advancements in gene editing and regenerative medicine are driving progress. To unlock the full potential of ATMPs, streamlining regulations, improving manufacturing, and fostering collaboration between researchers and healthcare providers is essential.
The EMA had a dedicated section on ATMPs with relevant guidelines and information:
The Committee for Advanced Therapies (CAT) is an EMA committee responsible for assessing the quality, safety and efficacy of ATMPs and following scientific developments in the field.
The CAT delivers scientific recommendations on ATMP classification. Regulatory requirements differ per type of ATMP. For that reason a correct classification is critical and, in case of doubt, it is recommended to determine the right classification at an early stage in the development. More information can be found here
EMA provides formal support to ATMP developers through:
- Scientific advice and protocol assistance;
- Orphan designation;
- the micro, small and medium-sized enterprise (SME) office;
- Classification of advanced therapy medicinal products (ATMPs);
- Certification of quality and non-clinical data for SMEs.
An overview of relevant guidelines for ATMPs can be found here. Relevant legislation and guidelines regarding pharmacovigilance in the EU are applicable to ATMPs and can be found here.
The European Medicines Agency (EMA) provides procedural and guidance documents to help companies applying for a marketing authorisation for advanced therapy medicinal products (ATMPs).
Bringing a biotech solution to the market: From start-up to scaling up, this page is your EU biotech and biomanufacturing hub that provides tools and resources to help you succeed in the biotech and biomanufacturing sector, also on ATMPs.
Also see our ATMP guidebook for innovators in the Netherlands for more useful information and links
Funding
- ZonMw | The Program Pluripotent Stem Cells for Inherited Diseases and Embryonic Research (PSIDER) is designed for biomedical research with induced pluripotent stem cells (iPS).