Ocugen’s OCU100 gets FDA orphan drug designation for treatment of retinitis pigmentosa

Ocugen, Inc. and the University of Colorado today announced exclusive license agreements that allow for Ocugen to continue developing two drug candidates for the treatment for ophthalmology indications, and that one of the assets, OCU100, recombinant lens epithelium derived growth factor 1-326 (LEDGF1-326), received orphan-drug status from the U.S. Food and Drug Administration for treatment of retinitis pigmentosa (RP), a rare eye disease.
“Orphan drug designation from the FDA’s Office of Orphan Products Development is a significant milestone that will allow Ocugen to accelerate the clinical development of OCU100, which has the potential to be the first approved therapeutic for retinitis pigmentosa,” said Shankar Musunuri, PhD, MBA, founder and chairman of the Ocugen Board of Directors.
Ocugen scientific founder and board member Uday Kompella, PhD, a professor of Pharmaceutical Sciences, Ophthalmology and Bioengineering at CU’s Anschutz Medical Campus, is the inventor of OCU100. Ocugen licensed all assets related to LEDGF, including LEDGF1-326 and OCU200, an anti-angiogenic tumstatin fusion protein, to be developed for treatment of wet age-related macular degeneration (AMD) from the University of Colorado in March.
Retinitis pigmentosa is a rare eye disease caused by inherited gene mutations that lead to retinal degeneration affecting approximately 100,000 people...
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Source: News Medical