AVROBIO Receives Orphan-Drug Designation from the FDA for AVR‑RD‑02 for the Treatment of Gaucher Disease

AVROBIO announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for the Company’s investigational gene therapy, AVR-RD-02, for the treatment of Gaucher disease. AVR-RD-02 consists of the patient’s own hematopoietic stem cells, genetically modified to express glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher disease. The Company is actively recruiting in Canada for its Phase 1/2 clinical trial of AVR-RD-02, which seeks to evaluate the safety and efficacy of the therapy in patients with Type 1 Gaucher disease.

“Under the existing standard of care, patients with Gaucher disease are bound to a lifelong infusion schedule of enzyme replacement therapies, and still experience painful and progressive symptoms such as debilitating musculoskeletal pain and fatigue,” said Birgitte Volck, MD, PhD, President of Research and Development at AVROBIO. “Orphan-drug designation recognizes the unmet need of populations with rare diseases like Gaucher where AVROBIO strives to transform lives by addressing the underlying cause of the disease with a single dose of gene therapy.”

Orphan-drug designation is granted by the FDA to drugs and biologics which 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 United States. Orphan-drug designation provides certain incentives, which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers.