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FDA grants orphan drug status to oral medication for Leber's congenital amaurosis, retinitis pigmentosa
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VANCOUVER, British Columbia — The U.S. Food and Drug Administration has granted QLT orphan drug designation to further develop an oral medication for treating Leber's congenital amaurosis and retinitis pigmentosa, QLT announced.
The compound, QLT091001, is an orally administered synthetic retinoid replacement for 11-cis-retinal, a crucial biomechanical factor in the visual retinoid cycle.
Under the Orphan Drug Act, QLT will be eligible to claim 7-year market exclusivity if the compound is approved for treating Leber's congenital amaurosis and/or retinitis pigmentosa. In addition, the company will receive FDA-based regulatory guidance, fee reductions and tax credits stemming from developmental expenses, according to a press release from the company.
"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," Bob Butchofsky, president CEO of QLT, said in a release announcing the orphan drug designation in the retinitis pigmentosa indication. "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."