QLT gains fast track status for drug to treat Leber's congenital amaurosis, retinitis pigmentosa
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VANCOUVER, British Columbia — The U.S. Food and Drug Administration has granted QLT two fast track designations for an orally administered synthetic retinoid replacement therapy, the company announced in a news release.
The designation applies to QLT091001 for the treatment of Leber's congenital amaurosis and retinitis pigmentosa stemming from inherited mutations in the RPE65 and LRAT genes, which are believed to hinder retinoid production.
In December 2010, the FDA granted orphan drug status to QLT091001 for Leber's congenital amaurosis and retinitis pigmentosa. In July 2011, the FDA cleared an investigational new drug application for QLT091001. Also in July, QLT gained clearance from the U.K. Medicines and Healthcare Products Regulatory Agency to proceed with clinical trials.
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QLT recently completed a phase 1b trial of QLT091001 in the treatment of Leber's congenital amaurosis. The company is proceeding with follow-up and initial re-treatment of patients and plans to complete enrollment for a retinitis pigmentosa cohort before the end of the year, the release said.
"QLT091001's fast track designation, along with its orphan drug designation, highlights the need for therapeutic options for those patients suffering from inherited retinal diseases," Bob Butchofsky, president and CEO of QLT, said in the release. "We are pleased that the FDA recognizes QLT091001 as a potential treatment for [Leber's congenital amaurosis] and [retinitis pigmentosa], and we are now working diligently to advance the development of this compound under the fast track process."作者: 601313225 时间: 2011-9-15 08:38
少见病***资格（Orphan Drug Designation）含义：
少见病就是患病人数较少的疾病，在美国是指患者少于20000人的疾病。因为患者人数少，所以研制的新药经不起市场的竞争，为了提高研制者的积极性，美国食品***管理局给予少见病***研制者更多的优惠政策和专利保护。
(备注：“少见病***”经常被错译为“孤儿药”)
