OCU400 receives third orphan drug designation from FDA
The FDA has granted a third orphan drug designation for OCU400 to treat RHO mutation-associated retinal degeneration, according to a press release from Ocugen.
The mutation is part of the retinitis pigmentosa group of disorders. RHO mutations make up about 12% of patients with retinitis pigmentosa in the U.S.
OCU400 consists of a copy of a nuclear hormone receptor gene delivered to cells in the retina using an adeno-associated viral vector, the release said. When the gene is expressed in the retina, it may help reset retinal homeostasis, in addition to potentially stabilizing cells, stopping photoreceptor degeneration and halting vision loss.
“OCU400, comprising the nuclear hormone receptor gene NR2E3, has the potential to help modulate numerous biological pathways that function in maintaining the health of the retina,” Mohamed Genead, MD, acting chief medical officer and chair of the retina scientific advisory board at Ocugen, said in the release.
The other ODDs are for NR2E3 and CEP290 mutation-associated retinal degeneration, the release said.