US FDA grants orphan drug designation to Attralus Therapeutic’s zamubafusp alfa for the treatment of AL amyloidosis

Attralus, Inc., a clinical stage biopharmaceutical company developing transformative medicines to improve the lives of patients with systemic amyloidosis, announced that the US Food and Drug Administration (FDA) has granted orphan drug designation for zamubafusp alfa (AT-02) for the treatment of light chain (AL) amyloidosis, a rare, progressive, debilitating, and often fatal condition.

Zamubafusp alfa, the company’s lead pan-amyloid removal candidate, has been evaluated in a completed phase 1 study and is currently being studied in an ongoing phase 2 open-label trial, both of which enrolled patients with AL amyloidosis.

“We are pleased to have received orphan drug designation from the U.S. FDA for zamubafusp alfa in AL amyloidosis,” said Gregory Bell, M.D., chief medical officer at Attralus. “Current approved therapies for AL target light-chain production, reducing the formation of new amyloid, but there is a significant unmet need for new therapies that can remove existing toxic amyloid fibrils that cause organ damage and mortality.”

The FDA’s Orphan Drug Designation applies to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. Orphan drug designation provides sponsors with benefits designed to support the development of drugs and biologics for small patient populations with unmet medical needs and include tax credits for clinical costs, exemptions from certain FDA fees, and the potential for seven years of marketing exclusivity.