QLT Inc. (Nasdaq:QLTI) (TSX:QLT) ("QLT" or the "Company") today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for QLT091001, its oral synthetic retinoid, for the treatment of Leber Congenital Amaurosis (LCA). LCA is an inherited progressive retinal degenerative disease that leads to retinal dysfunction and visual impairment beginning at birth. QLT091001 is an orally administered synthetic retinoid replacement for 11-cis-retinal, which is a key biochemical component of the visual retinoid cycle.
"We are pleased to have received FDA orphan drug designation for QLT091001 as a potential treatment for LCA, as there are no FDA approved treatments for children suffering from this debilitating disease," said Bob Butchofsky, President and Chief Executive Officer of QLT. "It is an important step in the development of our oral synthetic retinoid as we continue to advance this program."
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.
Source QLT Inc.