Orphan drug designation for gene therapy treatment of rare genetic disease

Orchard Therapeutics Limited (“Orchard”), a clinical-stage biotechnology company dedicated to bringing transformative gene therapies to patients with orphan diseases announces today that the US Food and Drug Administration (FDA) granted Orphan Drug Designation for autologous CD34+ cells transduced with an hCD11b lentiviral vector encoding for the human N-sulfoglycosamine sulfohydrolase (SGSH) gene for the treatment of Mucopolysaccharidosis type IIIA (MPS-IIIA or Sanfilippo type A).

MPS-IIIA is a rare neurodegenerative inherited lysosomal storage disease caused by mutations in the SGSH gene. The disease, which affects children as early as two years of age, results in severe and rapidly progressive brain disease and neurological symptoms. There is no effective treatment for MPS-IIIA to date.

To develop OTL-201 Orchard is working closely with Dr Brian Bigger, Dr Simon Jones and Professor Robert Wynn under a partnership with the University of Manchester and Central Manchester University Hospitals NHS Foundation Trust.

Dr. Jesus Garcia-Segovia, Orchard’s VP Clinical Development, CNS and metabolic disorders stated:

We are very pleased to receive FDA orphan drug designation for OTL-201 currently in preclinical development for Sanfilippo type A and we are very excited with the possibility of bringing effective treatments for children suffering this condition, given the very high unmet need.

The FDA Orphan Drug Designation is granted to drugs and biologics intended for the treatment of rare diseases (those affecting fewer than 200,000 people in the United States). The designation provides sponsors with development and commercial incentives including seven years of market exclusivity in the US and certain fee exemptions and reductions.