QLT Inc. (Nasdaq:QLTI) (TSX:QLT) ("QLT" or the "Company") today announced that QLT091001, the Company's oral synthetic retinoid, has been granted orphan drug designation for the treatment of Retinitis Pigmentosa (RP) from the U.S. Food and Drug Administration (FDA). This follows the FDA grant of orphan drug designation for QLT091001 for the treatment of Leber Congenital Amaurosis (LCA) also on December 7, 2010. 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 excited to gain this second orphan drug designation status from the FDA for QLT091001, as it strengthens the synthetic retinoid program and allows for important advantages in the continued development path," said Bob Butchofsky, President and Chief Executive Officer of QLT. "We are hopeful these benefits will help us reach our goal of treating the thousands of patients suffering from these rare and sight debilitating inherited retinal diseases."
LCA is an inherited progressive retinal degenerative disease that leads to retinal dysfunction and visual impairment beginning at birth. RP also results from genetic mutations that lead to visual impairment and is characterized by degeneration of rod and cone photoreceptors, resulting in a more variable loss of vision in late childhood to adulthood.
The Orphan Drug Act allows for financial incentives to promote the development of drugs and biologics for the treatment of rare diseases or conditions which affect fewer than 200,000 patients in the United States. Incentives include a seven-year period of market exclusivity after approval for the indication, regulatory guidance, FDA fee reductions and tax credits related to development expenses.