The FDA has granted Amryt Orphan Drug Designation for AP103

Amryt (Nasdaq: AMYT) announces the US Food and Drug Administration (FDA) has authorized AP103 as an orphan drug to treat Dystrophic EpidermolysisBullosa (DEB).  The AP103 drug was developed by Amryt, a gene-therapy company, to treat patients with DEB, a subset of EB. 

The FDA’s Office of Orphan Products Development (OOPD) grants orphan drug designations to medicines that are being developed for rare diseases affecting fewer than 200,000 Americans. If Amryt is granted orphan drug designation, it may be granted a seven-year period of marketing exclusivity in the US in exchange for approval of AP103 and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees, subject to certain conditions.



AP103 is based on a novel gene therapy delivery platform, which Amryt in-licensed in March 2018. That delivers the collagen VII gene into skin cells using a non-viral vector, HPAE (Highly Branched Poly -Amino Ester).


Joe Wiley, CEO of Amryt Pharma, commented: The FDA designation of our gene-therapy candidate, AP103, as an orphan drug is an important advancement for patients suffering from EB, and adds momentum to our development pipeline.  Unlike other gene therapies, which rely on viral vectors and topical delivery platforms, AP103 is based on a novel polymer-based topical delivery platform, which has potential applications in other genetic skin conditions and the gene therapy field whole.

Amryt is a biopharmaceutical company that develops and delivers innovative new treatments to improve the lives of patients suffering from rare diseases. Amryt consists of a growing portfolio of commercial and development assets.

LEAVE A REPLY

Please enter your comment!
Please enter your name here