Naples, Florida-based Attralus, Inc. announced receipt of US FDA orphan drug designation for zamubafusp alfa (AT-02) for the treatment of immunoglobulin light chain (AL) amyloidosis, a rare and progressive multiorgan disease for which no approved therapy currently targets amyloid removal directly.
The designation may provide development incentives including tax credits, user-fee exemptions, and, if ultimately approved for the indication, seven years of US orphan exclusivity.
The AL amyloidosis orphan drug designation is the fourth such designation zamubafusp alfa has received globally. The drug previously received FDA orphan drug designation for transthyretin-associated amyloidosis (ATTR), and the European Medicines Agency’s Committee for Orphan Medicinal Products adopted positive opinions for orphan medicinal product designations for zamubafusp alfa in both ATTR and AL amyloidosis. In the EU, orphan drug designation provides a 10-year period of marketing exclusivity following product approval, along with incentives including protocol assistance from the EMA and direct access to the centralized authorization procedure.