FDA Names Potential Topical Gene Therapy for DEB an Orphan Drug

Forest Rayby Forest Ray

In Epidermolysis Bullosa News.

FDA Names Potential Topical Gene Therapy for DEB an Orphan Drug

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FDA Names Potential Topical Gene Therapy for DEB an Orphan Drug

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AP103, a potential topical gene therapy for dystrophic epidermolysis bullosa (DEB). “Receiving an orphan drug designation from the FDA for our gene-therapy candidate, AP103, is a significant development for patients suffering from EB and provides additional momentum to our development pipeline,” Joe Wiley, CEO of Amryt Pharma, the medication’s developer, said in a press release. Orphan drug status provides companies with incentives…

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