The European Medicines Agency granted an orphan designation to Sangamo Therapeutics and Pfizer’s SB-525, a gene therapy candidate for hemophilia A.
SB-525 uses a recombinant virus to deliver a human factor VIII cDNA construct and a proprietary, synthetic liver-specific promoter to the nucleus of liver cells with a single infusion.
A Phase I/II clinical trial is currently evaluating SB-525 in adults with hemophilia A. Initial data from the study are expected in late 2017 or early 2018. The FDA granted the therapy a Fast Track designation last month.