MeiraGTx Holdings Plc (MGTX) announced on Wednesday that the U.S. Food and Drug Administration (FDA) has awarded Rare Pediatric Disease Designation to its AAV8-RK-RetGC program. This initiative aims to treat patients diagnosed with Leber congenital amaurosis, a condition that causes significant vision impairment in children.
The company, which specializes in clinical-stage genetic medicines, explained that this condition is linked to mutations in the GUCY2D gene. These mutations lead to the loss of photoreceptor function, resulting in early-onset blindness.
This designation by the FDA marks a significant regulatory achievement for MeiraGTx Holdings, highlighting the promise of their technology in addressing severe blinding disorders in children.
