Therapy Areas: Cardiovascular
Avrobio Receives Rare Pediatric Disease Designation from the US FDA for AVR-RD-05, a Gene Therapy for Mucopolysaccharidosis Type II or Hunter Syndrome
5 November 2021 - - The US Food and Drug Administration has granted Rare Pediatric Disease Designation to AVR-RD-05, US-based clinical-stage gene therapy company Avrobio, Inc's (NASDAQ: AVRO) 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 company said.

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 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, UK 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.