Orchard Therapeutics gets fourth Rare Pediatric Disease designation

The US Food and Drug Administration has granted a Rare Pediatric Disease designation to the company’s gene therapy candidate OTL-200, for the treatment of patients with metachromatic leukodystrophy (MLD), the drugs developer, UK and USA-based clinical-stage biotech Orchard Therapeutics, announced today.

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 five years is estimated at 75% and 30%, respectively.

The FDA grants Rare Pediatric Disease designations for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The Rare Pediatric Disease Designation makes the program eligible for a Rare Pediatric Disease Priority Review Voucher upon approval of OTL-200 by the FDA, which can be used by the company or sold on to another drugmaker.