AVRO - AVROBIO gets FDA Rare Pediatric Disease status for Hunter syndrome treatment AVR-RD-05

AVROBIO (NASDAQ:AVRO) announces that the U.S. FDA has granted Rare Pediatric Disease Designation to AVR-RD-05 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 company’s planned investigator-sponsored Phase 1/2 clinical trial for Hunter syndrome is expected to commence in the second half of 2022. Hunter syndrome, which affects an estimated one in 100,000 to one in 170,000 males worldwide, causes devastating complications throughout the body and brain, including

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AVROBIO gets FDA Rare Pediatric Disease status for Hunter syndrome treatment AVR-RD-05