FDAnews Drug Daily Bulletin

Sangamo, Pfizer Receive EMA Orphan Designation for Hemophilia Gene Therapy

June 13, 2017

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.

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