World Health Organization selects ISCT journal Cytotherapy(R) to publish novel ATMP official nomenclature initiative

World Health Organization selects ISCT journal Cytotherapy(R) to publish novel ATMP official nomenclature initiative

Official INN assignments to provide proper classification for ATMPs, ensuring safe dispensing and identification by healthcare providers

Vancouver, Canada, April 6, 2021 -The International Society for Cell & Gene Therapy (ISCT), the global society of clinicians, researchers, regulators, technologists, and industry partners dedicated to the translation of cell and gene therapy into safe and effective therapies to improve patients’ lives, today announces that the World Health Organization (WHO) has selected ISCT’s official journal Cytotherapy® to publish a landmark reference document on nomenclature in advanced therapy medicinal products (ATMPs). To achieve the aims set out in the initiative, Massimo Dominici, a former President for ISCT, has been appointed to the WHO Expert Advisory Panel on The International Pharmacopoeia and Pharmaceutical Preparations, serving the International Nonproprietary Names (INN) Expert Group.

The nomenclature developed for ATMPs will harmonize with the wider INN nomenclature. This was established by WHO in 1953 to ensure all medicinal products are globally recognized by a unique name. This unique standard naming program makes communication more precise and avoids prescribing errors. As a result, standard naming practices for ATMPs can identify therapeutic agents within the product.

“The number of ATMPs, and specifically cell and gene therapies, moving through clinical development towards commercialization is accelerating. These therapies, and the resultant potential patients that can be treated, means harmonized nomenclature is now critical,” said Raffaella Balocco, Head of the International Nonproprietary Names (INN) programme, WHO. “Working with cell and gene therapy in the INN, working group on CT and GT, has been essential for the development of harmonized nomenclature schemes. As the global organization representing all stakeholders across the cell and gene therapy sector, the involvement of ISCT and the Cytotherapy® Journal ensures that the WHO INN nomenclature will be utilized for the benefit of all those involved in the sector. This includes academia, developers, commercial organizations, investors, regulators, practitioners, and patient associations. The WHO nomenclature will result in ATMPs that are easily identifiable, and therefore contribute to safe and appropriate prescription and delivery across these groups.

Official INN assignments developed by WHO will aid pharmacovigilance by distinguishing products approved through official regulatory channels from those unproven cell and gene therapies that are marketed without regulatory oversight. This will prove a vital development for ISCT and its Presidential Task Force (PTF) on the Use of Unproven and/or Unethical Cell and Gene Therapies with its industry leading role to characterize unproven and unethical cell and gene interventions, and promote safe and effective practices worldwide.

ISCT will showcase the pivotal role of this WHO publication by dedicating a spotlight session on the topic, in its virtual ISCT 2021 Annual Meeting, the meeting building cross-sector consensus to translate the next wave of cell and gene therapies to patients. This will enable ISCT to gather input from its membership across the sector. The summit will be held as a virtual event between May 26-28, 2021. The headline theme for the 2021 Annual Meeting is ‘Orchestrating Global CGT Translation: Building Consensus for the Path Forward’. It will provide the platform for the sector to coordinate and harmonize the development, manufacture, regulation and delivery of therapies to patients of all indications.

“This optimized WHO nomenclature program is a vital step in the clinical translation of ATMPs. This will aid the rapid development of cell and gene therapy technologies that are bringing an unprecedented number of products to clinical trial, and hopefully, the clinic world-wide,” said Massimo Dominici, Past President, ISCT, and Member of the WHO Expert Advisory Panel on The International Pharmacopoeia and Pharmaceutical Preparations serving the INN Expert Group. “In addition, this will be a major additional tool for the fight against unproven cell and gene therapies. Official categorization through nomenclature will enable the field and global scientific societies, like ISCT, to continue spearheading efforts to ensure only therapies developed through official regulatory pathways, with clinically proven safety and efficacy, reach patients.”

The INN nomenclature system that is being developed for ATMPs relies on specific key elements. These include prefixes to describe the uniqueness of each product, common suffixes to identify distinct pharmacological groupings, and infixes, syllables placed between the prefix and suffix to provide more information about the products origin, mode of action, etc. Currently, the number of ATMP-specific infixes includes 57 unique terms and this number continues to expand. The number of infixes demonstrates the scope of challenges involved in developing a harmonized nomenclature system to define cell and gene therapy products. It also provides a benchmark of what the WHO INN Programme has achieved so far in the harmonization of definitions, and in the development of a revised INN application form for cell therapy substances.

About the International Society for Cell & Gene Therapy

Established in 1992, the International Society for Cell & Gene Therapy (ISCT) is a global society of clinicians, regulators, researchers, technologists and industry partners with a shared vision to translate cell and gene therapy into safe and effective therapies to improve patients’ lives worldwide.

ISCT is the global leader focused on pre-clinical and translational aspects of developing cell and gene-based therapeutics, thereby advancing scientific research into innovative treatments for patients. ISCT offers a unique collaborative environment that addresses three key areas of translation: Academia, Regulatory and Commercialization. Through strong relationships with global regulatory agencies, academic institutions and industry partners, ISCT drives the advancement of research into standard of care.

Comprised of over 2,400 cell and gene therapy experts across five geographic regions and representation from over 60 countries, ISCT members are part of a global community of peers, thought leaders and organizations invested in cell and gene therapy translation. For more information about the society, key initiatives and upcoming meetings, please visit: https://isctglobal.org, @ISCTglobal.