The FDA has granted orphan drug designation to Orchard Therapeutics for OTL-102, the company’s ex vivo autologous hematopoietic stem cell gene therapy being investigated for the treatment of X-linked chronic granulomatous disease (X-CGD).
The decision is based on early academic clinical trial data that demonstrated ex-vivo autologous HSC gene therapy being an ideal approach for the treatment of X-CGD, a rare, life-threatening, inherited disease of the immune system.
X-CGD is caused by mutations in the cytochrome B-245 beta chain gene encoding the gp91phox subunit of phagocytic NADPH oxidase. Patients with X-CGD are unable to kill bacteria and fungi because of the genetic defect, leading to chronic, severe infections.
In the clinical trial, an analysis of safety and efficacy outcomes in 9 severely affected patients with X-CGD was conducted. In addition, at 12 months post-treatment, 6 of 7 surviving patients exceeded the minimum threshold hypothesized in published literature to demonstrate potential clinical benefit, defined by 10% functioning, oxidase-positive neutrophils in circulation, and have discontinued preventive antibiotics.
Researchers are planning to enroll additional pediatric patients in 2020 to assess outcomes in this patient population, and to improve the efficiency of the drug product manufacturing process prior to initiating a registrational study.
Orchard Therapeutics announces FDA granted Orphan Drug Designation for OTL-102 for treatment of x-linked chronic granulomatous disease (X-CGD). Boston and London; Orchard Therapeutics: January 29, 2020 https://ir.orchard-tx.com/news-releases/news-release-details/orchard-therapeutics-announces-fda-granted-orphan-drug Accessed January 30, 2020.