Orchard Therapeutics nabs Orphan Drug tag for neurometabolic disease med

The FDA designates Orchard Therapeutics’ (ORTX -2.2%) OTL-203, an ex vivo autologous hematopoietic stem cell gene therapy, an Orphan drug and rare pediatric disease, for mucopolysaccharidosis type I (MPS-I), a rare neurometabolic disease caused by a deficiency of the alpha-L-iduronidase lysosomal enzyme required to break down sugar molecules.

Among the benefits of Orphan Drug status in the U.S. is a seven-year period of market exclusivity for the indication, if approved

Orchard expects to release full proof-of-concept results and initiate the pivotal study for OTL-203 in 2021.

https://seekingalpha.com/news/3592768-orchard-therapeutics-nabs-orphan-drug-tag-for-otlminus-203-for-neurometabolic-disease