Advanced therapy medicinal products ( (ATMPs) are medicines that are based on genes, tissues or cells which offer groundbreaking new opportunities for the treatment of disease and injury.
ATMPs can be classified into three main types:
- Gene therapy medicines: these contain genes that lead to a therapeutic, prophylactic or diagnostic effect. They work by inserting ‘recombinant’ genes into the body, usually to treat a variety of diseases, including genetic disorders, cancer or long-term diseases. A recombinant gene is a stretch of DNA that is created in the laboratory, bringing together DNA from different sources. Gene therapies may be either in-vivo therapies where genetic modification occurs inside the body or ex-vivo where genetic modification which occurs outside of the human body.
- Somatic-cell therapy medicines: these contain cells or tissues that have been manipulated to change their biological characteristics or cells or tissues not intended to be used for the same essential functions in the body. They can be used to cure, diagnose or prevent diseases.
- Tissue-engineered medicines: these contain cells or tissues that have been modified so they can be used to repair, regenerate or replace human tissue.
In addition, some ATMPs may contain one or more medical devices as an integral part of the medicine, which are referred to as combined ATMPs. An example of this is cells embedded in a biodegradable matrix or scaffold.
ATMPs are usually complex treatments so having an experienced QP onboard is essential! They may require new ways of working from more traditional sterile dosage forms and the knowledge and skills of the many staff groups involved in the manufacture and delivery to patients, is of paramount importance. ATMPs are resource intensive as they present many complex and unique risks. It is common to have complex challenges concerning manufacturing/quality, safety and efficacy requiring expertise from several areas and departments such as tissue engineering, gene therapy or cell therapy experts, biotechnology personnel, clinical and surgical staff, pharmacovigilance, risk management, medical devices and ethics departments.
A risk based approach must be taken from the very start of your project to identify the various risks associated with the clinical use of the ATMP and the risk factors inherent to the ATMP with respect to quality safety and efficacy. Some of the potential ATMP specific risks may include the following;
- infections (microbial contamination of starting materials or during processing)
- tumourigenicity (cell transformation, integration to genome)
- dedifferentiation / loss of function of the cells
- immunogenicity, rejection
- ectopic engraftment of cells to non-target tissues
- shedding from bacteria or viral vectors
- small batch and sample sizes,
- short shelf-lives,
- availability of proper animal models,
- applicability of analytical testing methods.
- High production and testing costs per batch
As well as the challenging risks associated with ATMPs, there are also complex regulatory frameworks that must be followed e.g. ATMP Regulation 1394/2007 and Directive 2001/83EC, Clinical Trials Regulation 536/2014, specific GMP guidelines for ATMPs and other relevant legislation pertaining to tissue and cell traceability, human blood components and genetically modified organisms. To add to that, there may also be differing national requirements within the EU and UK, along with a diverse interpretation of the hospital exemption in different member states.
Why use MIAS Pharma for your ATMP product
Our MIA licenses covers both gene and cell therapy products, and we have QPs who are experienced in the manufacturing and testing of ATMPs to ensure your project runs smoothly and efficiently. Many ATMPs have short shelf lives and our QPs are experienced in releasing products quickly and without all release testing being completed where applicable, to meet the requirements of short treatment windows.
For any questions get in touch with us at info@miaspharma.com
