FX.co ★ MeiraGTx's AAV8-RK-RetGC Program Gets FDA's Rare Pediatric Disease Designation

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.