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news.QLT has received orphan drug designation from the US Food and Drug Administration (FDA) for QLT091001, an oral synthetic retinoid, for the treatment of Retinitis Pigmentosa (RP).
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The designation follows the grant of orphan drug designation by the FDA for QLT091001 for the treatment of Leber Congenital Amaurosis (LCA).
QLT091001 is an orally administered synthetic retinoid replacement for 11-cis-retinal, which is a key biochemical component of the visual retinoid cycle.
QLT president and CEO Bob Butchofsky said they 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.
"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," Butchofsky said.