Geneva, 26 July Programme on International Nonproprietary Names (INN)

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1 INN Working Doc July 2017 Distr.: GENERAL ENGLISH ONLY Nomenclature schemes for advanced therapies (substances for gene therapies, substances for cell therapies, substances for cell-based gene therapies and virus-based therapies) Geneva, 26 July 2017 Programme on International Nonproprietary Names (INN) Technologies Standards and Norms Regulation of Medicines and other Health Technologies (RHT) Essential Medicines and Health Products (EMP) World Health Organization, Geneva World Health Organization 2017 The designations employed and the presentation of the material in this publication do not imply the expression of any opinion whatsoever on the part of the World Health Organization concerning the legal status of any country, territory, city or area or of its authorities, or concerning the delimitation of its frontiers or boundaries. Dotted lines on maps represent approximate border lines for which there may not yet be full agreement. The mention of specific companies or of certain manufacturers products does not imply that they are endorsed or recommended by the World Health Organization in preference to others of a similar nature that are not mentioned. Errors and omissions excepted, the names of proprietary products are distinguished by initial capital letters. All reasonable precautions have been taken by the World Health Organization to verify the information contained in this publication. However, the published material is being distributed without warranty of any kind, either expressed or implied. The responsibility for the interpretation and use of the material lies with the reader. In no event shall the World Health Organization be liable for damages arising from its use.

2 1. Substances for gene therapies In 2005, a two-word nomenclature scheme for substances for gene therapies was formally adopted by the members of the INN Expert Group designated to deal with the selection of nonproprietary names. The 2016 updated scheme for substances for gene therapies using vectors based on recombinant nucleic acid sequences (DNA vectors, e.g. plasmid DNA, naked or complexed), genetically modified micro-organisms (bacterial vectors) or viruses (replication defective, replication competent or replication conditional viral vectors) as shown in Table 1. This scheme does not apply to gene therapies based on administration of genetically modified cells, although a vector might be used ex-vivo or in-vitro for manufacturing of those cells prior to administration. Table 1: Two-word scheme for substances for gene therapies (plasmid-, viral vector- and bacteria-based). word 1 (gene Prefix Infix Suffix and distinctive name to identify the gene using, when available, existing infixes for biological products, e.g.: -cima- cytosine deaminase -ermin- growth factor -kin- interleukin -lim- immunomodulator -lip- human lipoprotein lipase -mul- multiple gene -stim- colony stimulating factor -tima- thymidine kinase -tusu- tumour suppression -(a vowel)gene e.g. -(o)gene word 2 (vector and distinctive name to identify the viral vector type, e.g.: -adeno- adenovirus -cana- canarypox virus -foli- fowlpox virus -erpa- herpes virus -lenti- lentivirus -morbilli- Paramyxoviridae morbillivirus -parvo- adeno-associated virus (Parvoviridae dependovirus) -retro- other retrovirus -vaci- vaccinia virus -vec (non-replicating viral vector) -repvec (replicating viral vector) to identify the bacterial vector type, e.g.: -lis- Listeria monocytogenes -bac (bacteria vector) -plasmid (plasmid vector) In the case of substances for gene therapy based on non-plasmid DNA, there is no need for a second word in the 2. Substances for cell therapies 2

3 During the 63 rd INN Consultation in 2016, an INN-USAN-harmonized nomenclature scheme for substances for cell therapies was formally approved by the members of the INN Expert Group designated to deal with the selection of international nonproprietary names 1. Table 2 shows the nomenclature scheme to name all non-genetically modified substances for cell therapies, with the exception of minimally manipulated hematopoietic elements and combinations of substances, which are not named. Table 2: Nomenclature scheme for non-genetically modified substances for cell therapies. Prefix Infix1: manipulation/s (a) to specify, if appropriate, which manipulation the cells have undergone, using, when available, existing infixes for manipulation (b), e.g.: -fus- fusion to a cell Infix2: cell type to identify the primary cell type (c) using, when available, existing infixes for cell types (d) Suffix -cel (cell) (a) There may be more than one manipulation infix in the same INN, but should be avoided if possible to avoid overly long names. (b) In the case of manipulation such as cell expansion and cell activation (with cytokines/drug, etc.), there is no need for an infix; this kind of manipulation will be specified in the description. (c) Residual cells not expected to the intended function, are not named. (d) -co(n)- chondrocytes -cor- umbilical cord cells -defitem- differentiated stem cells (not fitting into any existing category) -den- dendritic cells -end(o)- endothelial cells -ep(a)- hepatocytes -fi(b)- fibroblasts -isle- islet cells -ker(a)- keratinocytes -leu- lymphocytes/monocytes/apc (white cells) (e) -mestro- mesenchymal stromal cells (MSC) -mio(b)- myoblasts -ova- ovary cells -pla(c)- placenta cells -ren- renal tubular cells -ret- retinal epithelial cells -tem- stem cells -tesi- testis cells -tu- tumor cells -ur- urothelial cells (e) The cell type infix -leu- is used to describe hematologic cell preparations that do not fit in a particular or specific cell type category. Such cell preparations may be comprised of a mixture of the various blood cell elements, a subset of blood elements such as T-, B- or NK-cells, or antigenpresenting cells (APCs) that do not fit in the definition of dendritic cells. 1 INN selected before the adoption of the present nomenclature scheme may follow different rules. 3

4 Note: Information concerning manipulation and/or modification, and the type of the cellbased therapy (i.e. allogeneic, autologous and xenogeneic), will be specified in the description of the product. 3. Substances for cell-based gene therapies During the 63 rd INN Consultation in 2016, an INN-USAN-harmonized nomenclature scheme for substances for cell-based gene therapies was formally approved by the members of the INN Expert Group designated to deal with the selection of international nonproprietary names 2. Table 3 shows the nomenclature scheme to name all genetically modified substances for cellbased gene therapies, with the exception of minimally manipulated hematopoietic elements and combinations of substances, which are not named. Table 3: Nomenclature scheme for genetically modified substances for cell-based therapies. word 1 (gene Prefix Infix Suffix to identify the gene using, when available, existing infixes for biological products or using similar infix as for the protein for which the gene codes, e.g.: -cima- cytosine deaminase -ermin- growth factor -kin- interleukin -lim- immunomodulator -lip- human lipoprotein lipase -mul- multiple gene -stim- colony stimulating factor -tima- thymidine kinase -tusu- tumour suppression -(a vowel)gene e.g. -(o)gene word 2 (cell Infix 1: manipulation/s (a) to specify, if appropriate, which manipulation the cells have undergone, using, when available, existing infixes for manipulation (b), e.g.: Infix2: cell type to identify the primary cell type (c) using, when available, existing infixes for cell types (d) -cel (cell) -fus- fusion to a cell (a) (b) (c) (d) See the footnotes (a), (b), (c) and (d) on the preceding page. 2 INN selected before the adoption of the present nomenclature scheme may follow different rules. 4

5 Note: Information concerning manipulation and/or modification, and the type of the cellbased therapy (i.e. allogeneic, autologous and xenogeneic), will be specified in the description of the product. 4. Virus-based therapies Table 4 shows the nomenclature scheme for virus-based therapies. Table 4: Nomenclature scheme for virus-based therapies. Prefix Infix 1: virus type -adeno- adenovirus -cana- canarypox virus -foli- fowlpox virus -erpa- herpes virus -lenti- lentivirus -morbilli- Paramyxoviridae morbillivirus -parvo- adeno-associated virus (Parvoviridae dependovirus) -retro- other retrovirus -vaci- vaccinia virus Infix 2: -tu- for tumoricidal Suffix -rev (therapeutic virus) 5