Advanced therapy medicinal products (ATMPs) are medicines for human use based on genes, cells or tissue engineering.
To be a GTMP the medicine must have both;
– a recombinant nucleic acid used in or administered to human beings with a view to regulating, repairing, replacing, adding or deleting a genetic sequence.
– a therapeutic, prophylactic or diagnostic effect related directly to the recombinant nucleic acid sequence it contains or to the product of genetic expression of this sequence.
The following are examples of GTMPs approved by the European Medicines Agency (EMA).
Imlygic – Unresectable Melanoma
Strimvelis – ADA
Yescarta CAR-T – non-Hodgkin lymphoma
Zalmoxis – avoiding infection after bone marrow transplantation
ATMPs are divided into four categories:
- Gene therapy drugs include genes that lead to therapeutic, preventive, or diagnostic effects. These products are mainly used by introducing recombinant genes into the patient’s body to treat a wide range of diseases, including genetic disorders, cancer, or chronic diseases. The recombinant gene is actually part of the synthetic DNA in the laboratory, which is a combination of one or more gene fragments from different sources.
- Cell therapy drugs include cells and tissues that have been manipulated to change their biological characteristics or cells and tissues that have been used for purposes other than those intended for use. These drugs can also be used to treat, diagnose, and prevent disease.
- Tissue engineering drugs: include cells and tissues that have undergone changes to regenerate, repair, and replace human tissues.
- Combination drugs: These drugs are made up of a biological drug (cell and tissue) along with other substances and compounds. Specific examples of this type of drug are cells located in scaffolding or biodegradable substrate.