Therapy Areas: Central Nervous System
Avrobio Receives Orphan Drug Designation from the US Food and Drug Administration for AVR-RD-05, a Gene Therapy for Mucopolysaccharidosis Type II or Hunter Syndrome
14 July 2022 - - The US Food and Drug Administration has granted orphan drug designation for AVR-RD-05, US-based clinical-stage gene therapy company Avrobio, Inc's (NASDAQ: AVRO) 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.

Orphan drug designation is granted by FDA to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people in the US Orphan drug designation provides certain incentives, which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers.

Hunter syndrome is caused by a deficiency in the lysosomal enzyme iduronate-2-sulfatase, which is essential for breaking down large sugar molecules.

The gene therapy uses a patient's own hematopoietic stem cells that are transduced ex vivo with a lentiviral vector encoding the human IDS enzyme.

The company's planned collaborator-sponsored Phase 1/2 clinical trial for Hunter syndrome is expected to commence in 2023 under the company's collaboration with the University of Manchester, UK.

The program was developed by Brian Bigger, Ph.D., professor of cell and gene therapy at the University of Manchester, who has previously published preclinical data demonstrating that HSC 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, including severe neurological, cardiac and respiratory dysfunction, skeletal malformations and hearing impairment.

Children with severe cases of Hunter syndrome typically show early symptoms of the disease in infancy and childhood 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, 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 issues.

Even with ERT, people with Hunter syndrome face life-limiting symptoms and a significantly reduced lifespan.

Avrobio's pipeline is powered by its plato gene therapy platform, its foundation designed to deliver gene therapy worldwide.

It includes clinical programs in cystinosis and Gaucher disease type 1, as well as preclinical programs in Gaucher disease type 3, Hunter syndrome and Pompe disease.

The company is headquartered in Cambridge, Massachusetts.
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