Sangamo BioSciences, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to SB-FIX, the company's zinc finger nuclease (ZFN)-mediated genome editing product candidate for the treatment of hemophilia B. Sangamo expects to initiate a Phase 1/2 clinical study (SB-FIX-1501) in adult subjects with the disease in 2016.
"We are pleased with the FDA's decision to grant orphan drug designation to SB-FIX for hemophilia B," said Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president of research and development. "By enabling targeted integration of a therapeutic factor 9 gene, our ZFN-mediated genome editing approach may offer hemophilia B patients a therapeutic option that has potential advantages over conventional gene therapy approaches. We will enroll adult hemophilia patients into our first clinical trial, however, our goal is to move into pediatric patients, a population we believe could particularly benefit from a treatment that has the potential to provide lifelong expression of therapeutic levels of Factor IX protein."
"Our mission is to translate our ground-breaking science into genetic therapies that transform patients' lives," said Sandy Macrae, M.B., Ch.B., Ph.D., Sangamo's president and chief executive officer. "This is another positive step forward for the first therapeutic in vivo genome editing application cleared for clinical evaluation in humans. I am pleased with the progress that we have made to advance this program and we look forward to initiating a Phase 1/2 clinical trial by the end of 2016."
The FDA grants orphan drug designation to investigational drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and provides several benefits to drug developers, including assistance with clinical study design and drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.