Benzinga | Nov 3, 2021 09:15AM EDT

AVROBIO Receives Rare Pediatric Disease Designation From The U.S. FDA For AVR-RD-05, A Gene Therapy For Mucopolysaccharidosis Type II Or Hunter Syndrome

AVROBIO, Inc. (NASDAQ:AVRO), 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.

The company's planned investigator-sponsored Phase 1/2 clinical trial for Hunter syndrome is expected to commence in the second half of 2022. The program was developed by Brian Bigger, Ph.D., professor of cell and gene therapy at the University of Manchester, U.K. Prof. Bigger has published preclinical data demonstrating that ex vivo lentiviral gene therapy deploying an optimized, proprietary tag has the potential to correct peripheral disease and normalize brain pathology.

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 severe cardiac and respiratory dysfunction, skeletal malformations and hearing impairment. Children with severe cases of Hunter syndrome typically show early symptoms of the disease in their toddler years and begin to regress developmentally, losing basic motor skills and cognitive function over a few years. The current standard of care is weekly enzyme replacement therapy (ERT), which can delay some health complications but does not halt overall progression of the disease and has not been demonstrated to address the central nervous system (CNS) issues. Even with ERT, people with Hunter syndrome face life-limiting symptoms and a significantly reduced lifespan.