Orchard Therapeutics receives U.S. fast track designation for OTL-203

Orchard Therapeutics announced the U.S. Food and Drug Administration, FDA, has granted Fast Track designation to OTL-203, an investigational hematopoietic stem cell gene therapy being developed for the potential treatment of the Hurler subtype of mucopolysaccharidosis type I. “At Orchard Therapeutics, we are applying our HSC gene therapy platform to indications where we believe it is clinically differentiated and has the greatest potential to make a paradigm-shifting impact on the treatment landscape,” said Leslie Meltzer, Ph.D., chief medical officer. “We are encouraged that OTL-203 has been granted Fast Track designation because new treatment options are urgently needed for children with MPS-IH, as the current standard of care is associated with significant morbidity and mortality. Moreover, existing treatment options do not adequately address the broad range of clinical manifestations of the disease, including those that are most burdensome and have a high impact on quality of life. We look forward to initiating our global registrational trial in December to evaluate the efficacy and safety of OTL-203 compared to allogeneic HSCT.”