Amryt Pharma received orphan drug designation by the US FDA for AP103

▴ Amryt Pharma received orphan drug designation by the US FDA for AP103
AP103 is based on Amryt's gene-therapy platform technology and offers a potential treatment for patients with DEB

Amryt, a global, commercial-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from serious and life-threatening rare diseases, is pleased to announce that the U.S. Food and Drug Administration (“FDA”) has granted orphan drug designation for AP103 for the treatment of Dystrophic Epidermolysis Bullosa ("DEB"). AP103 is based on Amryt's gene-therapy platform technology and offers a potential treatment for patients with DEB, a subset of EB.

The FDA’s Office of Orphan Products Development (“OOPD”) grants orphan drug designation to support the development of medicines for rare diseases, that affect fewer than 200,000 people in the United States. Orphan drug designation may allow Amryt to be eligible for a seven-year period of U.S. Marketing exclusivity upon approval of AP103 and a waiver of the Prescription Drug User Fee Act (“PDUFA”) filing fees, subject to certain conditions.

AP103 is based on a new gene therapy delivery platform, in-licensed by Amryt in March 2018, that utilises a non-viral delivery vector, HPAE (Highly Branched Poly β-Amino Ester), designed to deliver the correct collagen VII gene into skin cells.

Joe Wiley, CEO of Amryt Pharma, commented: "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. Unlike other gene therapies that rely on viral vectors, AP103 is based on a novel polymer-based topical delivery platform, which we believe offers potential advantages in the gene-therapy field and has potential to be used in other genetic skin conditions.”

Amryt Pharma is a biopharmaceutical company focused on developing and delivering innovative new treatments to help improve the lives of patients with rare and orphan diseases. Amryt comprises a strong and growing portfolio of commercial and development assets.

Tags : #AmrytPharma #LatestNewsonAmrytPharma24thDec #LatestPharmaNews24thDec #USFDAGrantsOrphanDrugDesignation #DystrophicEpidermolysisBullosa #UnitedState #NewGeneTherapy

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