FDA Grants Break Through and Orphan Drug Designations for Krabbe Disease Gene Therapy

The Food and Drug Administration (FDA) announced that it has granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations for gene therapy (FBX-101, Forge Biologics, Inc.) to potentially treat Krabbe disease, a rare, inherited leukodystrophy. 

Krabbe disease is a lyosomal storage disorder caused by loss-of-function mutations in galactosylceramidase (GALC) needed to metabolize certain types of lipids such as psychosine. The disease initially manifests itself as physical delays in development, muscle weakness and irritability and affects approximately 1 in 12,500 to 100,000 people in the US. 

This gene therapy uses an adeno-associated virus (AAV) to deliver a functioning copy of the GALC gene intravenously to cells in the central nervous system and peripheral organs. A phase 1/2 clinical trial, RESKUE (NCT04693598), is actively recruiting participants for enrollment. 

“Infantile Krabbe is a progressive and devastating leukodystrophy,” said Jessie Barnum, MD, assistant professor, Department of Pediatrics, Division of Blood and Marrow Transplantation and Cellular Therapies, University of Pittsburgh Medical Center. “FBX-101 is an AAV gene therapy that has shown promising preclinical efficacy in Krabbe animal models of disease by extending survival and improving neuromuscular function when administered early in the disease course.”