What are ATMPs?
Advanced therapy medicinal products (ATMPs) are medicines for human use that are based on genes, cells or tissue engineering. This is a European classification through the European Medicines Agency (EMA).
Traditional transplants involve removing cells or tissue from the patient or from a donor and transferring them to the patient in a way that they perform the same funtion eg.
- skin taken from a healthy area and transplanted to a damaged area.
- bone marrow taken from a donor and injected into the patient to reconstiute their blood system.
Advanced Therapy Medicinal Products (ATMPs) vary from traditional transplant in at least one of the following ways;
- contains recombinant genes and for which the therapeutic effect is dependent on expression of that gene – thus all gene therapies and gene corrected cell therapies are ATMPs
- contains cells that have been taken from donor or patient and modified in a way deemed to alter their function before returning to the patient
- contains cells that have been taken from donor or 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 subclassified as GTMPs.
To be a GTMP the medicine must have both;
- an active substance which contains or consists of 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 relates directly to the recombinant nucleic acid sequence it contains or to the product of genetic expression of this sequence.
GTMPs do not include vaccines against infectious diseases.
The following are 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 or consist of cells or tissues that have been subject to substantial manipulation so that biological characteristics, physiological functions or structural properties relevant for the intended clinical use have been altered; or it contains or consists of cells or tissues that are not intended to be used for the same essential function(s) in the recipient as in the donor.
- have properties for, or be used in or 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 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:
- it contains or consists of cells or tissues that have been subject to substantial manipulation so that biological characteristics, physiological functions or structural properties relevant for the intended clinical use have been altered; or it contains or consists of cells or tissues that are not intended to be used for the same essential function(s) in the recipient as in the donor.
- it is presented as having properties for, or is used in or administered to, human beings with a view to regenerating, repairing or replacing a human tissue.
The following is 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.