Scientific recommendation on classification of ATMPs - February 2015 reports

By William Chin, PhD, Project Manager, EUCRAF

A series of reports were published by the Committee for Advanced Therapies (CAT) on the scientific recommendations on classification of advanced therapy medicinal products. Here is a summary of the reports presented in tabular format.

Active substance 

Finished product 
Proposed indication 
EMA/CAT conclusion Classification
A plasmid encoding a mutation-inactivated E7-E6 fusion protein from Human Papillomavirus 16 linked to the human chemokine hMIP-1α via a dimerization module derived from human IgG3 Sterile solution for injection Prevention and treatment of HPV16 induced pre-malignancies and malignancies

Consists of a genetically modified DNA plasmid containing a recombinant nucleic acid expressing a fusion protein consisting of the oncoproteins HPV E6/E7 and the cytokine human MIP 1-a. The medicinal product is administered to humans with the view of adding a genetic sequence.

Gene therapy medicinal product.
A double-stranded naked DNA plasmid encoding an inactive human telomerase reverse transcriptase protein fused to ubiquitin Sterile solution for intradermal injection followed by electroporation under local anaesthesia Immunotherapy for the treatment of various malignancies and the prevention of tumour relapse

Contains an active substance that consists of a plasmid nuclear acid sequence that is directly related to the transient local expression and processing of the Ubi-hTERT fusion protein, which subsequently specifically stimulates the patient’s immune system to mount an immune response against telomerase over-expressing tumor cells.

Gene therapy medicinal product
Bone marrow aspirate enriched in autologous mesenchymal stromal cells Bone marrow aspirate enriched in autologous mesenchymal stromal cells Repair of bone in case of bone fracture

Contains cells or tissues with biological characteristics, physiological functions or structural properties relevant for the intended regeneration, repair or replacement.

Tissue engineered product
Human retinal pigment epithelial cells derived from human embryotic stem cells
Cell suspension in cryopreservation medium
Treatment of age-related macular degeneration and Stargardt’s macular dystrophy 
The cells are substantially manipulated so that biological characteristics, physiological functions or structural properties relevant for the intended regeneration, repair or replacement are achieved.

Tissue engineered product

Autologous mature dendritic cells pulsed with tumour antigen-derived synthetic peptides

Cell suspension for injection Treatment of glioblastoma

The viable cells are substantially manipulated, so that the biological characteristics, physiological functions or structural properties relevant for the intended clinical use have been altered.

Somatic cell therapy medicinal product
Allogeneic peripheral blood mononuclear cells induced to an early apoptotic state Cell suspension for intravenous administration Prevention of graft versus host disease

The product consists of human cells and the manufacturing process includes substantial manipulation that leads to changes in the cell characteristics (early apoptotic state) relevant for its intended use.

Somatic cell therapy medicinal product
Killed spores of Bacillus subtilis incorporating a non-toxic antigen of Clostridium difficile Oral / sublingual suspension Prevention of Clostridium difficile infections in elderly patients The product does not contain or consist of human cells, tissues or genes.

Does not fall within the definition of an Advanced Therapy Medicinal Product.

Source

1. EMA, News and Events, Report, 2 March 2015, Scientific recommendation on classification of advanced therapy medicinal products: Human retinal pigment epithelial cells derived from human embryotic stem cells

2. EMA, News and Events, Report, 2 March 2015, Scientific recommendation on classification of advanced therapy medicinal products: Plasmid encoding a mutation-inactivated E7-E6 fusion protein from Human Papillomavirus 16 linked to the human chemokine hMIP-1a via a dimerization module derived from human IgG3

3. EMA, News and Events, Report, 2 March 2015, Scientific recommendation on classification of advanced therapy medicinal products: Double-stranded naked DNA plasmid encoding an inactive human telomerase reverse transcriptase protein fused to ubiquitin

4. EMA, News and Events, Report, 2 March 2015, Scientific recommendation on classification of advanced therapy medicinal products: Autologous mature dendritic cells pulsed with tumour antigen-derived synthetic peptides

5. EMA, News and Events, Report, 2 March 2015, Scientific recommendation on classification of advanced therapy medicinal products: Killed spores of Bacillus subtilis incorporating a non-toxic antigen of Clostridium difficile

6. EMA, News and Events, Report, 2 March 2015, Scientific recommendation on classification of advanced therapy medicinal products: Autologous bone marrow aspirate enriched in autologous mesenchymal stromal cells

7. EMA, News and Events, Report, 2 March 2015, Scientific recommendation on classification of advanced therapy medicinal products: Allogeneic peripheral blood mononuclear cells induced to an early apoptotic state