AVROBIO, a US-based company, developing lentiviral-based gene therapies, has 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.
What is AVR-RD-02: 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. It is an ex vivo lentiviral-based investigational gene therapy. The stem cells are genetically modified to express functional glucocerebrosidase (GCase), which reduces levels of glucosylceramide and glucosylsphingosine, the accumulated substances which cause the symptoms of Gaucher disease.
How advanced is AVR-RD-02 development program: 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.
What is an orphan drug designation: Orphan-drug designation provides special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor. For a drug to qualify for orphan designation both the drug and the disease or condition must meet certain criteria specified in the ODA and FDA’s implementing regulations at 21 CFR Part 316. Orphan designation qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing. A marketing application for a prescription drug product that has received orphan designation is not subject to a prescription drug user fee unless the application includes an indication for other than the rare disease or condition for which the drug was designated.