NGF Blog


AVROBIO Receives Orphan-Drug Designation from FDA for AVR‑RD‑02

AVROBIO announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for Avrobio’s investigational gene therapy, AVR-RD-02, for the treatment of Gaucher disease. Dr. Birgitte Volck says in the news release:

“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.”

Read the full press release:

Living a Better Today

Skip to content