FDA grants SB-FIX orphan drug designation

Sangamo BioSciences Inc. recently announced that SB-FIX has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA).

SB-FIX is Sangamo’s zinc finger nuclease (ZFN) mediated genome editing product candidate that was designed and developed to treat hemophilia B.

"We are pleased with the FDA's decision to grant orphan drug designation to SB-FIX for hemophilia B," Sangamo EVP of Research and Development Geoff Nichol said. "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."

Sangamo has plans in place to begin a Phase 1/2 clinical study called SB-FIX-1501 this year. The study will involve adult subjects who have hemophilia B.

Orphan drug designation is granted by the FDA to investigational drugs and biologics that have been designed to treat rare diseases affecting less than 200,000 people.