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 Agencyâauthorization 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 âhospital exemptionâ 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;7:146â154.
Navigating Market Authorization: The Path Holoclar Took to Become the First Stem Cell Product Approved in the European Union / Pellegrini, Graziella; Ardigò, Diego; Milazzo, Giovanni; Iotti, Giorgio; Guatelli, Paolo; Pelosi, Danilo; De Luca, Michele. - In: STEM CELLS TRANSLATIONAL MEDICINE. - ISSN 2157-6564. - 7:1(2018), pp. 146-154.
Data di pubblicazione: | 2018 |
Data di prima pubblicazione: | 27-dic-2017 |
Titolo: | Navigating Market Authorization: The Path Holoclar Took to Become the First Stem Cell Product Approved in the European Union |
Autore/i: | Pellegrini, Graziella; Ardigò, Diego; Milazzo, Giovanni; Iotti, Giorgio; Guatelli, Paolo; Pelosi, Danilo; De Luca, Michele |
Autore/i UNIMORE: | |
Digital Object Identifier (DOI): | http://dx.doi.org/10.1002/sctm.17-0003 |
Rivista: | |
Volume: | 7 |
Fascicolo: | 1 |
Pagina iniziale: | 146 |
Pagina finale: | 154 |
Codice identificativo ISI: | WOS:000419115400016 |
Codice identificativo Scopus: | 2-s2.0-85039163338 |
Codice identificativo Pubmed: | 29280318 |
Citazione: | Navigating Market Authorization: The Path Holoclar Took to Become the First Stem Cell Product Approved in the European Union / Pellegrini, Graziella; Ardigò, Diego; Milazzo, Giovanni; Iotti, Giorgio; Guatelli, Paolo; Pelosi, Danilo; De Luca, Michele. - In: STEM CELLS TRANSLATIONAL MEDICINE. - ISSN 2157-6564. - 7:1(2018), pp. 146-154. |
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