Orchard Therapeutics, a commercial stage company, has been granted a Rare Pediatric Disease Designation from the US Food and Drug Administration (FDA) for its gene therapy candidate, OTL-200, for the treatment of patients with metachromatic leukodystrophy (MLD).
Gene therapy
MLD is a rare, fatal, neurodegenerative, inherited metabolic disease caused by mutations in the ARSA gene. In its late infantile and juvenile forms, which represents the majority of MLD patients, mortality at 5 years is estimated at 75% and 30%, respectively.
“This is the fourth Rare Pediatric Disease Designation awarded by the FDA to one of Orchard’s autologous ex vivo gene therapy candidates, providing significant incentives to continue expanding our pipeline,” stated Mark Rothera, president and CEO of Orchard. “MLD is a devastating disease in which most patients do not survive the first decade of life. In clinical trials, early treatment with OTL-200 has demonstrated preservation of cognitive and motor development to levels comparable with healthy individuals. We look forward to working with Telethon/Ospedale San Raffaele to bring this potentially life-changing treatment to patients as rapidly as possible.”
Orchard anticipates filing OTL-200 for market authorisation with regulatory authorities from 2019.