What are ATMPs?
Advanced therapy medicinal products (ATMPs) are medicines for human use based on genes, cells or tissue engineering.
This is a European classification through the European Medicines Agency (EMA).
Traditional transplants involve removing cells/tissue from the patient/donor and transferring them to the patient to perform the same function eg.
- skin taken from a healthy area and transplanted to a damaged area.
- donor bone marrow given to a patient to reconstitute their blood system.
Advanced Therapy Medicinal Products (ATMPs) differ from traditional transplant in at least one of the following ways, it may;
- contain recombinant nucleic acid (normally DNA or RNA) intended to have a therapeutic effect by regulating a genetic sequence or by introduction/modification of a genetic sequence – all gene therapies and gene corrected cell therapies are ATMPs
- contain cells that have been taken from donor/patient and modified so they are deemed to no longer be the same as the donor cells before being returned to the patient
- contains cells that have been taken from donor/patient and returned to the patient for what is deemed to be a different function
The official definitions of ATMPs from the EMA can be found in our ATMP regulatory guide here.
There are 4 types of ATMP;
– GTMP – gene therapy medicinal product
– sCTMP – somatic cell therapy medicinal product
– TEP – tissue engineered product
– cATMP – combined ATMP
Find more information on these below!
Gene Therapy Medicinal Product (GTMP)
Essentially all ’gene therapies’ are ATMPs/GTMPs. ATMPs/GTMPs do not include vaccines against infectious diseases.
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
Somatic Cell Therapy Medicinal Product (sCTMP)
sCTMPs contain cells or tissues that have altered characteristics or will not be used for the same essential functions in the body.
sCTMPs can be used to cure, diagnose or prevent diseases.
To be a sCTMP a medicine must both:
– contain cells that have been taken from donor/patient and modified so they are deemed to have new biological characteristics, physiological functions or structural properties relevant for the intended clinical use OR contains cells that have been taken from donor/patient and given to the patient in a way not intending the same function
– be administered to human beings with a view to treating, preventing or diagnosing a disease through the pharmacological, immunological or metabolic action of its cells or tissues.
The following are examples of sCTMPs approved by the EMA.
Alofisel – perianal fistulas in Crohn’s disease
Holocar – severe cornea damage
Tissue Engineered Product (TEP)
TEPs contain cells or tissues that have been modified so they can be used to repair, regenerate or replace human tissue.
A TEP has the following characteristics:
– contain cells that have been taken from donor/patient and modified so they are deemed to have new biological characteristics, physiological functions or structural properties relevant for the intended clinical use OR contains cells that have been taken from donor/patient and given to the patient in a way not intending the same function (SAME as sCTMP)
– be administered to human beings with a view to regenerate, repaire or replace a human tissue.
The following is an example of an EMA approved TEP.
Spherox – knee cartilage defects
Combined ATMP (cATMP)
Combined ATMPs contain one or more medical devices as an integral part of the medicine.
An example of a cATMP would be cells embedded in a biodegradable matrix or scaffold.
A combined ATMP must incorporate, as an integral part of the product, one or more medical devices within the meaning of Article 1(2)(a) of Directive 93/42/EECor one or more active implantable medical devices within the meaning of Article 1(2)(c) of Directive 90/385/EEC.
As well as either;
1. contain viable cells or tissues.
2. contain non-viable cells or tissues liable to act upon the human body with action that can be considered as primary to that of the devices referred to.