QLT Inc. today announced that QLT091001, an oral synthetic retinoid, has received positive opinions for two distinct Orphan Drug Designations by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) for the treatment of the inherited retinal degenerative diseases, Leber Congenital Amaurosis (LCA) and Retinitis Pigmentosa (RP). Positive opinions by the COMP precede official designations of QLT091001 as an orphan drug by the EMA. This follows the recent orphan drug designations by the U.S. Food and Drug Administration for QLT091001 for the treatment of LRAT and RPE65 genetic mutations in both LCA and RP.

QLT091001 is an orally administered synthetic retinoid replacement for 11-cis-retinal, which is a key biochemical component of the visual retinoid cycle, and is under investigation for the treatment of LCA and RP.

Click here ^[1] for the full press release.