Open AccessNavigating Market Authorization: The Path Holoclar Took to Become the First Stem Cell Product Approved in the European Union
Author(s) -
Pellegrini Graziella,
Ardigò Diego,
Milazzo Giovanni,
Iotti Giorgio,
Guatelli Paolo,
Pelosi Danilo,
De Luca Michele
Publication year - 2018
Publication title -
stem cells translational medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.781
H-Index - 71
eISSN - 2157-6580
pISSN - 2157-6564
DOI - 10.1002/sctm.17-0003
Subject(s) - marketing authorization , authorization , product (mathematics) , agency (philosophy) , business , european union , medicine , computer security , computer science , bioinformatics , philosophy , geometry , mathematics , epistemology , biology , economic policy
Gene therapy, cell therapy, and tissue engineering have the potential to revolutionize the treatment of disease and injury. Attaining marketing authorization for such advanced therapy medicinal products (ATMPs) requires a rigorous scientific evaluation by the European Medicines Agency—authorization is only granted if the product can fulfil stringent requirements for quality, safety, and efficacy. However, many ATMPs are being provided to patients under alternative means, such as “hospital exemption” schemes. Holoclar (ex vivo expanded autologous human corneal epithelial cells containing stem cells), a novel treatment for eye burns, is one of the few ATMPs to have been granted marketing authorization and is the first containing stem cells. This review highlights the differences in standards between an authorized and unauthorized medicinal product, and specifically discusses how the manufacture of Holoclar had to be updated to achieve authorization. The result is that patients will have access to a therapy that is manufactured to high commercial standards, and is supported by robust clinical safety and efficacy data. Stem Cells Translational Medicine 2018;7:146–154