Orchard Therapeutics Granted FDA Orphan Drug Designation for OTL-102 for X-linked Chronic Granulomatous Disease (X-CGD)

Orchard Therapeutics Granted FDA Orphan Drug Designation for OTL-102 for X-linked Chronic Granulomatous Disease (X-CGD)

Orchard Therapeutics announced the U.S. FDA has granted Orphan Drug Designation for OTL-102, the company’s ex vivo autologous hematopoietic stem cell (HSC) gene therapy being investigated for the treatment of X-linked chronic granulomatous disease (X-CGD). The FDA may grant orphan designation to drugs and biologics intended to treat a rare disease or condition affecting fewer than 200,000 persons in the U.S.

Early academic clinical trial data for OTL-102 was recently published in Nature Medicine. The trial was led by researchers at the University of California, Los Angeles (UCLA) — including Donald B. Kohn, M.D., one of the study's lead investigators and professor of microbiology, immunology and molecular genetics at UCLA — and Great Ormond Street Hospital (UK). The data provides an analysis of safety and efficacy outcomes in nine severely affected patients with X-CGD. At 12 months post-treatment, six of seven surviving patients, all of whom were adults or late adolescents, exceeded the minimum threshold hypothesized in published literature to demonstrate potential clinical benefit, defined as 10% functioning, oxidase-positive neutrophils in circulation and have discontinued pr...

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