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FDA grants orphan drug designation to SB-FIX for hemophilia B
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The FDA granted orphan drug designation to SB-FIX for the treatment of hemophilia B, according to its manufacturer.
SB-FIX (Sangamo BioSciences) — a zinc finger nuclease–mediated in vivo genome editing approach — is designed as a single treatment strategy to provide stable and continuous production of Factor IX clotting protein for the lifetime of each patient with the disease.
“We are pleased with the FDA’s decision to grant orphan drug designation to SB-FIX for hemophilia B,” Geoff Nichol, MB, ChB, executive vice president of research and development at Sangamo, said in a press release. “By enabling targeted integration of a therapeutic Factor IX gene, our zinc finger nuclease–mediated genome editing approach may offer hemophilia B patients a therapeutic option that has potential advantages over conventional gene therapy approaches.”
A phase 1/phase 2 clinical study designed to evaluate the treatment in adults with hemophilia B is scheduled to begin later this year.
“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,” Nichol said.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.