ACG-801

ACG-801 is an investigational biologic therapy consisting of recombinant human acid ceramidase (rhAC). It is being developed as the first enzyme replacement therapy specifically for Farber disease (also known as Farber lipogranulomatosis), a rare and severe lysosomal storage disorder caused by a genetic deficiency in the ASAH1 gene leading to loss of acid ceramidase activity. This deficiency results in abnormal accumulation of ceramide, causing profound macrophage-driven inflammation and multi-organ involvement including bone, joints, cartilage, immune system, central nervous system, and lungs. The disease is life-threatening with many patients dying in early childhood. ACG-801 aims to restore acid ceramidase activity and reduce pathological ceramide buildup. The drug has received Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation from regulatory agencies for Farber disease[2][5][6][10]. Preclinical studies have also suggested potential utility in cystic fibrosis and other conditions involving sphingolipid metabolism[5][7].

Other names

recombinant human acid ceramidaserhAC