Orchard announces U.S. FDA clearance of IND application for OTL-203

Orchard Therapeutics announced the U.S. Food and Drug Administration, FDA, has cleared its Investigational New Drug, IND, application for OTL-203, a hematopoietic stem cell, HSC, gene therapy being developed for the treatment of the Hurler subtype of mucopolysaccharidosis type I, MPS-IH. The company expects to initiate a global registrational trial evaluating the efficacy and safety of OTL-203 compared to standard of care in the second half of 2023. "Based on data from the proof-of-concept trial, treatment with a single administration of OTL-203 has the potential to address a range of multisystemic manifestations of MPS-IH," said Leslie Meltzer, Ph.D., chief medical officer of Orchard Therapeutics. "Our recent interactions with the FDA have been productive and we look forward to advancing this registrational study designed to generate the data necessary to enable global regulatory submissions and potentially provide an urgently needed new treatment option for the MPS-IH community." The study is a multi-center, randomized, active controlled clinical trial designed to evaluate the efficacy and safety of OTL-203 in patients with MPS-IH compared to standard of care with allogeneic hematopoietic stem cell transplant. A total of 40 patients with a confirmed diagnosis of MPS-IH who meet the study inclusion criteria will be randomized 1:1 to receive either OTL-203 or allogeneic HSCT. The study is powered to demonstrate superiority of OTL-203 over HSCT. The primary endpoint, which will be measured at two years post-treatment, comprises a composite of clinically meaningful outcomes, including death, the need for rescue treatment, treatment failure, immunological complications, as well as severe cognitive and growth impairment. Secondary endpoints include biochemical markers, additional clinical assessments, as well as safety and tolerability. The company expects to open up to six sites in the United States and Europe.