QLT Inc. (Nasdaq:QLTI) (TSX:QLT) ("QLT" or the "Company") 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.
"We are pleased that the EMA has recognized QLT091001 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."
The EMA's Orphan Drug Designation program allows for certain incentives to promote the development of drugs and biologics for patients suffering from rare and life-threatening diseases, conditions which affect no more than five in 10,000 people in the European Union. Incentives include a ten-year period of market exclusivity after approval for the indication, regulatory guidance and direct access to centralized marketing authorization, fee reductions and tax credits related to development expenses.