Retinitis pigmentosa treatment gets orphan drug status

The FDA has granted orphan drug status to Ocugen’s recombinant lens epithelium derived growth factor 1-326 (OCU 100) for retinitis pigmentosa.

“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, Ocugen founder and chairman of its board of directors.

Ocugen also announced the signing of exclusive license agreements with the University of Colorado (CU) that allow it to continue developing OCU 100 and OCU200, an anti-angiogenic tumstatin fusion protein for treatment of wet AMD.

“OCU100 has shown the potential as a promising therapeutic agent for treating retinitis  pigmentosa by reducing protein aggregation and associated cellular stresses, which are known  to contribute to this condition,” said Ocugen scientific founder and board member Uday Kompella, PhD, a professor of pharmaceutical sciences, ophthalmology and bioengineering at CU. “With impressive preclinical data, we look  forward to progressing with a phase 1 study for safety and tolerability in patients sometime in  2015.”