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.
It follows an orphan drug designation for the drug for the treatment of Leber’s congenital amaurosis.
QLT091001 is an orally administered synthetic retinoid replacement for 11-cis-retinal, which is a key biochemical component of the visual retinoid cycle.
Orphan drug designation is granted by the FDA to drugs that address an unmet clinical need and treat diseases affecting less than 200,000 patients in the US.
Retinitis pigmentosa is a set of hereditary retinal diseases, characterised by degeneration of rod and cone photoreceptors, while Leber’s congenital amaurosis is an inherited progressive retinal degenerative disease that results in retinal dysfunction and visual impairment beginning at birth.