Those efforts are reflected in a newly revised INN application form for cell therapy substances, which was officially approved during the 70th INN Consultation in April 2020.
That version is now mandatory for all new INN applications for gene therapy (GT), cell therapy (CT) and cell-based gene therapy (CGT) substances.
“The work was initiated based on the large volumes of cell and gene therapy applications the WHO was seeing in relation to INN requests,” Massimo Dominici, medical oncology expert, CEO of Rigenerand, and a member of the WHO Expert Advisory Panel on the International Pharmacopoeia and Pharmaceutical Preparations serving the INN Expert Group, told us.
As the cell therapy market exhibits a tremendous growth potential, that will likely translate into a corresponding increase in INN applications, he said.
The INN nomenclature system was originally developed to assign INN mainly to chemically well-defined, homogeneous substances. However, with the advent of large, complex biologics such as GT, CT and CGT substances, this has changed dramatically, noted Dominici in a landmark review of the work the INN CGT working group has undertaken.
He also led a talk on this milestone work during last month’s ISCT annual meeting, held virtually.
The challenge
While INN published in the WHO Drug Information are largely substances conforming to International Union of Pure and Applied Chemistry (IUPAC) nomenclature rules, GT, CT and CGT substances cannot be defined in this way.
Instead, INN around CGT are published together with a textual definition to univocally describe their characteristics. These definitions are an integral part of the INN, and particular importance is therefore allocated to these descriptive paragraphs, as they are the basis for defining the uniqueness of a particular cell substance.