PEDIATRIC GENE THERAPY: CONSIDERATIONS FOR PLANNING, EXECUTION, & LONG-TERM FOLLOW-UP

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Executive Summary


The European approvals of Glybera® in 2012 and Strimvelis® in 2016 seem likely to be the vanguard of the therapeutic revolution heralded by the decoding of the human genome. Both of these treatments originated from academic centers of excellence and became the subjects of agreements with pharmaceutical companies (Uniqure/Chiesi and GSK, respectively) with the experience to navigate the manufacturing infrastructure and regulatory approvals processes that led to the products’ commercialization. The EMA has indicated the level of follow-up it expects to see for these patients, and the FDA’s perspective will likely be revealed with the first US approvals of gene therapies anticipated in 2017.

 

Authors

Harris Dalrymple, PhD (Medicine), PhD (Law) Executive Director, Global Scientific Affairs, Vice Chair, Center for Pediatric Clinical Development, PRA Health Sciences

Sarah Rose, Clinical Scientist, Global Scientific Affairs, PRA Health Sciences

Mark Sorrentino, Vice President, Center for Pediatric Clinical Development