FDA grants orphan drug designation, rare pediatric disease designation for gene therapy for rare neurometabolic disease

The U.S Food and Drug Administration has granted orphan drug designation and rare pediatric disease designation for OTL-203 (Orchard Therapeuticsa), an ex vivo autologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I). 

“We are pleased by the FDA’s acknowledgement of the critical and urgent need to develop additional treatments for MPS-I given the severe, life-limiting nature of the disease,” said Bobby Gaspar, M.D., PhD., chief executive officer of Orchard. “The underlying causes of lysosomal storage disorders such as MPS-I have been notably difficult to address, and we are encouraged by the early evidence of our hematopoietic stem cell gene therapy’s approach to potentially treating this condition. The orphan drug and rare pediatric disease designations provide important momentum for the OTL-203 clinical program, which we remain committed to advancing as quickly as possible for patients in need.”

Read the full press release here.