Orphan status granted for neurotropic keratitis drug

The U.S. FDA has granted orphan drug status to a recombinant human nerve growth factor (rhNGF)-based treatment for neurotrophic keratitis.

Developed by Italian biopharmaceutical company Dompé, the drug previously received orphan drug status from the FDA and the European Medicines Agency for retinitis pigmentosa.

The drug’s safety and efficacy are currently under investigation in the randomized, double-blind REPARO study in 39 centers in nine European countries. More than 170 patients with unilateral neurotrophic keratitis with grade two lesions (persistent epithelial defect) or grade three lesions (corneal ulcers) are receiving one of two doses of rhNGF or placebo. Secondary objectives include evaluation of corneal lesion healing and visual acuity improvement.