ORTX - Orchard Therapeutics gains FDA fast track status for Hurler syndrome therapy

2023-11-30 07:24:17 ET

Orchard Therapeutics ( NASDAQ: ORTX ) announced on Thursday that its investigational hematopoietic stem cell (HSC) gene therapy has received Fast Track Designation from the U.S. Food and Drug Administration for the potential treatment of the Hurler subtype of mucopolysaccharidosis type I (MPS-IH).

Previously reported results from an ongoing single-center proof-of-concept (PoC) study of investigational OTL-203 showed extensive metabolic correction, continued cognitive, motor, and physical development, as well as early improvements in skeletal health.

In addition to the biochemical, neurological and skeletal results previously reported, study investigators presented favorable outcomes for other disease manifestations not effectively addressed by the current standard of care at the European Society of Cell and Gene Therapy 30th Annual Congress last month.

Buoyed by promising results observed in the PoC study, the firm is initiating a multi-center, randomized clinical trial to test the efficacy and safety of OTL-203 in MPS-IH patients compared to allogeneic hematopoietic stem cell transplant (HSCT). The global registrational trial is expected to commence in December 2023.

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