Retinoid shows promise

Vancouver, BC-The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has given an oral synthetic retinoid (QLT091001, QLT Inc.) positive opinions for two distinct orphan drug designations for the treatment of the inherited retinal degenerative diseases, Leber congenital amaurosis (LCA) and retinitis pigmentosa (RP), according to the company.

The action comes on the heels of the retinoid being designated an orphan drug by the FDA for the treatment of LRAT and RPE65 genetic mutations in both LCA and RP. The orally administered synthetic retinoid replacement for 11-cis-retinal, a key biochemical component of the visual retinoid cycle, is under investigation for the treatment of LCA and RP.

“We are pleased that the EMA has recognized [the retinoid] as a potential treatment for the thousands of patients who suffer from inherited blindness by granting positive opinions for these orphan drug designations for the treatment of LCA and RP,” said Bob Butchofsky, president and chief executive officer of QLT. “These are critical steps in our development and regulatory plans, and we will continue to work diligently to develop this potential treatment, and look forward to working with the EMA closely as this program moves forward.”