FDA grants orphan drug designation to gene therapy for retinal degenerative disease

The U.S. Food and Drug Administration has granted the third orphan drug designation (ODD) for OCU400 (Ocugen, Inc) in the treatment of RHO mutation-associated retinal degeneration, according to a press release.

“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. A recent preclinical study published in Nature Gene Therapy demonstrated the potency of NR2E3 to elicit broad-spectrum therapeutic benefits in early and intermediate stages of RP in five unique mouse models,” said Dr Mohamed Genead, acting Chief Medical Officer of Ocugen and Chair of Ocugen’s Retina Scientific Advisory Board in the release. “We believe OCU400 has the potential to address multiple genetic mutations associated with RP and, therefore, help a broader pool of patients,” Dr. Genead continued.