Amryt Pharma Inc (LON:AMYT) (NASDAQ:AMYT) has said therapy AP103 has received an orphan drug designation from the US Food and Drug Administration (FDA) for the condition 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, Amryt said.
Orphan drug status is granted to rare diseases that affect fewer than one in 200,000 people in the US and allows a seven-year period of marketing exclusivity on approval and a waiver of some drug licence fees.
AP103 is based on the new gene therapy delivery platform that was in-licensed by Amryt in March 2018.
In a statement, Joe Wiley, Amryt’s chief executive, said: "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.”
DEB is a condition that affects 20% of people with the fragile skin condition EB and is where blisters heal with scarring that can lead to problems such as joint contractions, fusions of fingers and toes and narrowing of the oesophagus.