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21 insights into biological & advanced therapy medicinal products (ATMPs)

Insight [in-sahyt], noun.

(The ability to have) a clear, deep, and sometimes sudden understanding of a complicated problem or situation.

See deeper into biological and advanced therapy medicinal products (ATMPs) at just a glance with this free, easy to read, downloadable booklet.

ATMP Consulting

CeutiQus offers cutting-edge strategic translation consultancy in innovative, biological, regenerative and advanced therapy medicinal product (ATMPs). We support the whole development life cycle with clinical, regulatory and quality guidance.

There are four main types of ATMPs or ATIMPs:

1) Gene therapy products (GTMP).   

2) Somatic cell therapy products (sCTMP). 

3) Tissue-engineered products (TEP). 

4) Combination ATMPs (cATMP). 

What is an Advanced Therapy Medicinal Product (ATMP) or Advanced Therapy Investigational Medicinal Product (ATIMP)?

An Advanced Therapy Medicinal Product (ATMP) is a novel and broad type of medicine used to treat diseases by using advanced technologies that involve manipulating genes, cells or tissues.

Advanced Therapy Medicinal Products (ATMPs)

These advanced therapy medicinal products (ATMPs) are a broad class of  therapies that are usually developed for the treatment of serious and life-threatening diseases, where conventional treatments are either unavailable or have failed to provide a satisfactory outcome.

ATMPs are highly complex medicines and are therefore subject to strict regulations to ensure their safety and efficacy. They require a rigorous process of development, testing, and authorisation from a regulatory authority before they can be marketed to the public.

The ATMP may be either a licensed commercially available medicine with a marketing authorisation (MA) or an investigational medicinal product (IMP), also known as an ATIMP, that is being clinically investigated in clinical trials prior to a marketing authorisation application (MAA).

Gene therapy products

A gene therapy medicinal product (GTMP) is a biological medicine that both:

 – contains a recombinant nucleic acid as its active substance, used to regulate, repair, replace, add, or delete a genetic sequence in the body. 

– has a therapeutic, prophylactic, or diagnostic effect directly related to the nucleic acid sequence it contains, or to the product of the genetic expression of this sequence.

GTMPs are hence medicines that contain genes or genetic material that is designed to correct or replace a defective or missing gene in a patient’s cells. 

NB. GTMPs do not include vaccines against infectious diseases.

Examples of gene therapy medicinal product (GTMPs)

Some examples of gene therapy medicinal product (GTMPs) include a medicine that uses a recombinant nucleic acid to:

– repair a genetic mutation that causes cystic fibrosis.
 
– add a gene that produces a protein that helps to fight cancer.
 
– delete a gene that causes a rare blood disorder. 

Gene therapy medicinal products may be further categorised as either in vivo or ex vivo.

In vivo gene therapy

In vivo gene therapy is a type of gene therapy where genetic material is delivered directly to cells in the body.

Example of an in vivo gene therapy medicinal product

One example of an in vivo gene therapy is Luxturna (voretigene neparvovec-rzyl). Luxturna is an AAV-based gene therapy product that is used to treat inherited retinal dystrophy (IRD), a group of genetic diseases that cause blindness. Luxturna works by replacing a mutated gene (RPE65) that is responsible for the production of a protein that is essential for vision.

Ex vivo gene therapy

Ex vivo gene therapy is a type of gene therapy where cells are removed from a patient’s body, genetically modified, and then returned to the patient’s body. 

Example of an ex vivo gene therapy medicinal product

An example of ex vivo gene therapy is the genetic modification of CD34+ haematopoietic stem cells (HSCs) with the Adenosine deaminase (ADA) gene to treat ADA-SCID. CD34+ HSCs are a type of stem cell that can give rise to all types of blood cells. The ADA gene is responsible for the production of an enzyme that helps to break down toxins in the body. These toxic substances can damage the DNA of T cells and B cells, which prevents them from developing properly. As people with ADA deficiency are unable to produce this enzyme, this can lead to a number of serious health problems, including severe combined immunodeficiency (SCID). In ex vivo gene therapy for ADA deficiency, CD34+ HSCs are removed from a patient’s body and genetically modified to produce the ADA enzyme. The modified cells are then returned to the patient’s body, where they can produce the enzyme in the immune system cells and help to correct the deficiency. Ex vivo gene therapy has been shown to be an effective treatment for ADA deficiency. In clinical trials, this ex vivo gene therapy has been shown to be able to cure ADA deficiency in about 90% of patients.

Somatic cell therapy products

Somatic cell therapy medicinal products are biological products that contain cells or tissues that have been manipulated to change their biological characteristics, or cells or tissues that are not intended to be used for the same essential functions in the body.

These products are used to treat, prevent, or diagnose diseases through the pharmacological, immunological, or metabolic action of their cells or tissues.

Examples of somatic cell therapy products

Examples include cells of the immune system e.g. T cells, B cells, dendritic cells, natural killer cells, macrophages etc.

Other examples include stem cells which are undifferentiated cells that have the potential to develop into a variety of different cell types.

Tissue-engineered products

Tissue-engineered products (TEPs) are medicines that contain “engineered” cells or tissues used to regenerate, repair, or replace human tissues.

Examples of TEPs include artificial skin for burns, cartilage implants for arthritis, tissue-engineered bladders for bladder diseases and bone implants for bone fractures and injuries.

Tissue-engineered products can contain cells or tissues from humans or animals, and they can be viable or non-viable. They may also contain other substances, such as cellular products, biomolecules, biomaterials, chemical substances, scaffolds, or matrices.

NB. Products that contain only non-viable cells and/or tissues are not considered tissue-engineered products.

Cells or tissues are considered “engineered” if they have been changed in a way that makes them better able to regenerate, repair, or replace human tissues.

The changes must be significant enough to improve the cells or tissues’ biological characteristics, physiological functions, or structural properties.

Some changes, such as those listed in Annex I to Regulation (EC) No. 1394/2007 (the Advanced Therapy Medicinal Products (ATMPs) Regulation), are not considered to be significant enough to make the cells or tissues “engineered.”

Cells or tissues are also considered “engineered” if they are not intended to be used for the same essential function or functions in the recipient as in the donor.

Examples of tissue-engineered products (TEPs)

Some examples of engineered cells or tissues include:

– Skin cells that have been genetically modified to be resistant to scarring.

– Heart muscle cells that have been grown in a lab and then transplanted into a patient.

– Cartilage cells that have been harvested from a patient’s knee and then cultured in a lab to repair a damaged joint.

They are a relatively new type of medicine, and they are still under development but have the potential to revolutionise the way we treat diseases and injuries. They could be used to replace damaged or diseased tissues of the human body. Tissue-engineered products could also be used to grow new organs (organ regeneration), such as kidneys and livers. They are a promising new area of medicine, and they have the potential to improve the lives of billions of people.

Combination ATMP products

A combined advanced therapy medicinal product (cATMP) is an advanced therapy medicinal product (ATMP) that contains one or more medical devices.

To be classed as a combined ATMP product the cATMP must meet the following conditions:

– The medical device must be an integral part of the product

– The cellular or tissue part of the product must contain viable cells or tissues.

– The cellular or tissue part of the product containing non-viable cells or tissues must be liable to act upon the human body with action that can be considered as primary to that of the devices referred to.

What is an ATMP Qualified Person (QP)?

An ATMP Qualified Person (QP) is a person who has been named on a manufacturing license to ensure that an Advanced Therapy Medicinal Product (ATMP) is manufactured, tested, and released for use in patients in accordance with the appropriate regulations and quality standards.

The QP is responsible for certifying that each batch of an ATMP (or ATIMP) has been manufactured and tested in compliance with the requirements of the marketing authorisation (MA) or clinical trial authorisation (CTA), Good Manufacturing Practice (GMP) and any other relevant regulations of each relevant jurisdiction.

As an example, the QP must ensure that the manufacturing facility and quality management systems (QMS) are in compliance with Good Manufacturing Practice (GMP) regulations, for example, that any deviations or non-conformities are investigated and resolved.

This includes, amongst many other activities, verifying that the patient-safety critical manufacturing processes have been suitably validated, that the product has been tested for safety, identity, potency, and purity, and that the appropriate documentation has been completed and reviewed.

To become an ATMP QP, a person must have a degree in a relevant scientific discipline such as pharmacy and a significant level of experience in pharmaceutical manufacturing, testing, quality assurance, and regulatory affairs. The QP must also be registered with the relevant regulatory authority in the country where the ATMP is being manufactured or marketed.

An ATMP Qualified Person (QP), for example Dr Jasbir Rattu, is a person who has been designated to ensure that an Advanced Therapy Medicinal Product (ATMP) is manufactured, tested, and released for use in patients in accordance with the appropriate regulations and quality standards.

The QP is responsible for certifying that each batch of an ATMP (or ATIMP) has been manufactured and tested in compliance with the requirements of the marketing authorisation (MA) or clinical trial authorisation (CTA) and other relevant regulations.

In addition, the QP must ensure that the manufacturing facility and quality management systems (QMS) are in compliance with Good Manufacturing Practice (GMP) regulations e.g. that any deviations or non-conformities are investigated and resolved.

This includes verifying that the manufacturing process has been suitably validated, that the product has been tested for identity, potency, and purity, and that the appropriate documentation has been completed and reviewed.

To become an ATMP QP, a person must have a degree in a relevant scientific discipline such as pharmacy and a significant level of experience in pharmaceutical manufacturing, quality assurance, and regulatory affairs.

The QP must also be registered with the relevant regulatory authority in the country where the ATMP is being manufactured or marketed and have expert knowledge of the product and processes for which he or she takes responsibility.

Who is an ATMP Qualified Person (QP)? Dr Jasbir Rattu is a highly experienced ATMP Qualified Person (QP)

THE CALLING TO BIOLOGICAL AND ADVANCED THERAPY MEDICINAL PRODUCTS (ATMPS)

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