AVROBIO Inc. said the U.S. Food and Drug Administration granted its gene therapy AVR-RD-01 the orphan drug designation to treat Fabry disease.
The inherited disease is characterized by a defect in the GLA gene leading to a deficiency of the enzyme alpha-galactosidase A, which results in the body's inability to break down a compound called globotriaosylceramide, resulting in potentially life-threatening kidney damage, cardiac disease and early stroke.
AVR-RD-01 involves modifying certain stem cells of patients to add the functional copy of the faulty GLA gene and re-infusing them back into the blood to help produce the deficient enzyme.
The Cambridge, Mass.-based company is enrolling patients for a phase 2 as well as phase 1 study to test AVR-RD-01.
Orphan drug designation is granted by the U.S. FDA to encourage the development of therapies for treating rare diseases that affect fewer than 200,000 people in the U.S. through incentives, such as tax credits and application fee waiver.