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AVROBIO Receives Rare Pediatric Disease Designation from the U.S. FDA for AVR-RD-05, a Gene Therapy for Mucopolysaccharidosis Type II (MPSII) or Hunter Syndrome
AVROBIO, Inc., a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to AVR-RD-05, its lentiviral gene therapy for the treatment of mucopolysaccharidosis type II (MPSII), or Hunter syndrome, a rare and seriously debilitating lysosomal disorder that primarily affects young boys.
The FDA’s Rare Pediatric Disease Designation and Voucher Program is intended to facilitate the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. Companies that receive approval for a new drug application or Biologics License Application (BLA) for a rare pediatric disease may be eligible to receive a voucher for a priority review of a subsequent marketing application for a different product. The priority review voucher may be used by the company or sold to a third party.
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