Orchard Therapeutics' gene therapy nabs accelerated review in Europe for neurometabolic disease
Sep. 28, 2020 7:19 AM ETOrchard Therapeutics plc (ORTX)ORTXBy: Vandana Singh, SA News Editor4 Comments
- The European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to Orchard Therapeutics' (NASDAQ:ORTX) OTL-203, an ex vivo autologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I).
- MPS-I is a rare neurometabolic disease caused by a deficiency of the alpha-L-iduronidase lysosomal enzyme required to break down sugar molecules.
- Recently, OTL-203 received US Orphan Drug designation for the indication.