Orchard receives U.S. FDA Fast Track designation for hematopoietic stem cell gene therapy in Hurler syndrome

The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Orchard Therapeutics’ OTL-203, an investigational hematopoietic stem cell gene (HSC) therapy being developed for the potential treatment of the Hurler subtype of mucopolysaccharidosis type I (MPS-IH).

Fast Track designation is intended to expedite the development of promising medicines that address serious medical needs. Therapeutic candidates that receive Fast Track designation may be eligible for enhanced interactions with the FDA, including potentially quicker submission and review timelines.

MPS-I is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme resulting in the accumulation of glycosaminoglycans (GAGs) in multiple organs, including the eyes, ears, heart, as well as the musculoskeletal and central nervous systems. It is estimated to occur globally in 1 in 100,000 live births.

Approximately 60 percent of children born with MPS-I have the most severe subtype, MPS-IH, also called Hurler syndrome, and rarely live past the age of 10 when untreated. Current treatment options for MPS-IH include allogeneic hematopoietic stem cell transplant (HSCT) and chronic enzyme replacement therapy (ERT), both of which have significant limitations.

“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.”

OTL-203 has previously received Rare Pediatric Disease (RPD) and priority medicines (PRIME) designations from the FDA and European Medicines Agency, respectively.

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