The U.S. Food and Drug Administration (FDA) has granted orphan drug designation for AVROBIO’s AVRO lead drug AVR-RD-05 for the treatment of mucopolysaccharidosis type II (MPSII), or Hunter syndrome.
Hunter syndrome is a rare and seriously debilitating lysosomal disorder that primarily affects young boys.
The regulatory agency has granted rare paediatric disease designation for AVR-RD-05 previously, while the lead candidate is company’s fourth gene therapy to receive orphan drug designation.
FDA grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. It also provides benefits of seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.
Price Action : AVROBIO shares are trading around 12 percent higher at $1.08 on Wednesday pre-market session.