US gene therapy company Avrobio has secured orphan-drug designation for AVR-RD-02, its investigational gene therapy, for the treatment of Gaucher disease, from the US Food and Drug Administration (FDA).

AVR-RD-02 is made up of the patient’s own hematopoietic stem cells, which are modified genetically to express glucocerebrosidase (GCase), an enzyme that is deficient in Gaucher disease – a rare genetic disorder that impacts several organs and tissues in the body.

The ex vivo lentiviral-based investigational gene therapy has been designed to give durable and potentially life-long therapeutic benefit for Gaucher disease patients through a single dose of their own hematopoietic stem cells.

The FDA orphan drug designation is given to drugs and biologics which are being developed for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that impact less than 200,000 people in the US. The designation comes with certain incentives, which could cover tax credits for the cost of clinical trials and prescription drug user fee waivers.

Commenting on the orphan drug designation for AVR-RD-02 gene therapy, Birgitte Volck – Avrobio President of Research and Development, said: “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.

“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.”

Avrobio said that it is actively enrolling patients in Canada for its phase 1/2 clinical trial of AVR-RD-02, which aims to assess the safety and efficacy of the investigational gene therapy for the treatment of Type 1 Gaucher disease.

  AVR-RD-02, Avrobio, Birgitte Volck, Gaucher disease, Gene therapy company, US FDA, US Food and Drug Administration