Avrobio Receives Rare Pediatric Disease Designation from the U.S. FDA for First-In-Class Gene Therapy for Gaucher Disease

AVROBIO, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to AVR-RD-02, a first-in-class investigational gene therapy that genetically modifies patients' own
hematopoietic stem cells (HSCs) to treat Gaucher disease, a rare lysosomal disorder that can lead to multiorgan pathology, clinical morbidity and early mortality.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 (NDA) 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.