The FDA has granted orphan drug designation for chemically induced photoreceptor-like cells to treat retinitis pigmentosa. In a process developed by CiRC Biosciences, cells are chemically converted within two weeks from one cell type (fibroblasts) into photoreceptor-like cells (cones or rods in the retina). These cells were studied using animal models with retinitis pigmentosa and demonstrated “partial restoration of pupil reflex and visual function.” These cells are also in development for other applications such as macular degeneration and advanced retinitis pigmentosa. Orphan drug designation means that, upon request of a sponsor, the FDA granted a special status to drugs and biological products intended to treat a rare disease, a condition that affects fewer than 200,000 people in the United States. This status also provides the sponsor with tax reductions and a seven-year window of exclusivity to develop a cure for the rare disease with that drug.
What this means for Usher syndrome: If proven successful, cell therapy with these chemically induced photoreceptor-like cells may become a possible cure for retinitis pigmentosa.