FDA Names Potential Topical Gene Therapy for DEB an Orphan Drug

FDA Names Potential Topical Gene Therapy for DEB an Orphan Drug

282049

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…

You must be logged in to read/download the full post.