FDA grants orphan drug designation to SB-913 Genome Editing Treatment for MPS II
Release Date: 15-Apr-2017
Sangamo Therapeutics, Inc., a leading pharmaceutical company dedicated to develop gene therapies and therapeutic genome editing has recived orphan drug designation from US Food and Drug Administration for company’s genome editing product candidate, SB-913, for the treatment of Mucopolysaccharidosis Type II (MPS II), a rare lysosomal storage disorder. MPS II is caused by mutations in the gene encoding iduronate 2-sulfatase (IDS) enzyme. Using Sangamo's zinc finger nuclease (ZFN) genome editing technology, SB-913 is designed as a single treatment strategy intended to provide stable, continuous production of the IDS enzyme for the lifetime of the patient.
The FDA grants orphan drug designation to 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 may provide several benefits to drug developers, including tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.
“Global Orphan Drug Clinical Pipeline Insight” reports by kuick research gives comprehensive insight on various clinical and non-clinical aspects associated with ongoing clinical trials of 808 orphan designated drugs across the globe. The in-depth clinical insight presented in the report helps the reader to analyze and identify the various stakeholders involved in the clinical development and commercialization of orphan designated drugs in the global market. Currently there are more than 300 orphan designated drugs commercially available in the global market and around 800 drugs in clinical development phase.
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