2022-09-21| Trials & Approvals

AVROBIO’s First-of-its-Kind Gene Therapy Receives Rare Pediatric Disease Designation

by Reed Slater
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Gene therapy-focused AVROBIO announced it received the FDA’s Rare Pediatric Disease Designation for its AVR-RD-04, a specialized gene therapy designed to treat cystinosis. AVR-RD-04 is the first and only targeted gene therapy to treat the rare genetic disorder that can shorten the affected individuals’ lifespan and quality of life. 

Targeting a Rare, Life-Threatening Childhood Disorder

Cystinosis is a rare lysosomal storage disorder characterized by an accumulation of cystine. If untreated, the disease can result in organ failure, potentially leading to early death. Only about 1,600 patients in the US, Europe, and Japan suffer from the disease. Current treatment options include vitamin supplements and the chemical compound cysteamine, which is available in eye droplet form. 

About 90% of treated cystinosis patients require a kidney transplant in the second or third decade of their lives. With modern treatments, patients with the condition can live up to 50 years or even longer. 

AVROBIO designed AVR-RD-04 to genetically modify the patient’s hematopoietic stem cells (HSCs) to express cystinosin, a protein that cystinosis patients critically lack. The treatment is currently in a Phase ½ clinical trial with six participants, studying the safety and efficacy of the therapy. The company says the drug is proving tolerable in the current trial population with very few mild or moderate adverse events. 

AVR-RD-04’s recent Rare Pediatric Disease Designation provides AVROBIO a Priority Review Voucher (PVR), which the company can use for a different product in the future. According to the FDA, recipients of a PVR are eligible to transfer the voucher by sale to third parties. The FDA implemented the Rare Pediatric Disease Designation to incentivize companies to develop therapies for the prevention and treatment of rare pediatric diseases. 

Before receiving the Rare Pediatric Disease Designation, AVROBIO’s AVR-RD-04 received the FDA’s Orphan Drug Designation in March last year. That designation comes with certain perks like tax benefits toward clinical trial costs, prescription drug user fee waivers, and marketing exclusivity upon approval.

Related Article: Bluebird’s Rare Childhood Neurodegenerative Gene Therapy Gains Accelerated Approval

AVROBIO’s Gene Therapy Ambitions

On top of AVR-RD-04, AVROBIO has four other gene therapy candidates in its pipeline. All of the products focus on rare diseases like Gaucher Disease, Hunter syndrome, and Pompe. Currently, the company has a product in Phase ½ trials to treat Gaucher Type 1, with the others in preclinical development. 

The FDA’s Rare Pediatric Disease Designation for AVR-RD-04 is another step forward in the company’s goal to bring life-saving gene therapy products to the market. AVROBIO still has a lot of work to see its aspirations come to fruition. Still, it is on the right track, leveraging revolutionary technology and ideas to develop a specialized range of products. 

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