MeiraGTx Holdings Plc. (MGTX), Wednesday announced that the U.S. Food and Drug Administration has granted the Rare Pediatric Disease Designation to its AAV8-RK-RetGC program for the treatment of patients with Leber congenital amaurosis that results in severe vision loss in children.
The clinical-stage genetic medicines company noted the disease is an after-effect of GUCY2D mutations. Due to photoreceptor function loss, blindness emerges very early in life.
The FDA approval marks a regulatory milestone for the company, demonstrating the therapeutic potential of its technology to address severe childhood blinding conditions.
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