Retinitis pigmentosa treatment receives orphan designation

Aurora, CO-The FDA has granted orphan drug status to Ocugen’s recombinant lens epithelium derived growth factor 1-326 (OCU100) for the treatment of retinitis pigmentosa (RP).

“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 (the treatment), which has the potential to be the first approved therapeutic for RP,” said Shankar Musunuri, PhD, MBA, founder and chairman of the Ocugen Board of Directors.

According to Uday Kompella, PhD-Ocugen scientific founder and board member, and professor of pharmaceutical sciences, ophthalmology, and bioengineering at the University of Colorado’s Anschutz Medical Campus-the treatment has shown potential to be a promising therapeutic agent for treating RP. Dr. Kompella said it does so by reducing protein aggregation and associated cellular stresses, which are known to contribute to the condition.

“With impressive preclinical data, we look forward to progressing with a phase I study for safety and tolerability in patients sometime in 2015,” said Dr. Kompella, who is also the treatment’s inventor.

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