QLT091001 works as a replacement for 11-cis-retinal, a key biochemical component of the visual retinoid cycle, and is used to treat the inherited retinal degenerative diseases Leber Congenital Amaurosis (LCA) and Retinitis Pigmentosa (RP).
The EMA’s orphan drug designation program gives certain incentives to further the development of drugs for patients suffering from rare and life-threatening diseases, such as a ten-year period of market exclusivity after approval, regulatory guidance and direct access to centralized marketing authorization, fee reductions and tax credits related to development expenses.
QLT president and CEO Bob Butchofsky said the EMA recognition would propel the use of QLT091001 as a potential treatment for patients suffering from inherited blindness.