AMYT.L Regulatory News (AMYT)

AMRYT PHARMA PLC

("Amryt" or the "Company")

AP101 Receives Fast Track Designation from FDA

Amryt, a biopharmaceutical company focused on developing and delivering innovative new treatments to help improve the lives of patients with rare and orphan diseases, today announces that the U.S. Food and Drug Administration (FDA) has designated the investigation of AP101 (Oleogel-S10)  for the treatment of Epidermolysis Bullosa (EB) as a Fast Track development program. The FDA has recognised that EB is a serious disease, and that there are no FDA-approved treatments for this condition. Additionally, the FDA has noted that Amryt has generated preliminary clinical data from an ongoing Phase 3 trial, which supports continued study. 

Dr Joe Wiley, CEO of Amryt Pharma, commented: "Receiving a Fast Track designation from the FDA represents another significant step in the progress of our lead development asset - AP101 - as a potential treatment for the wound related complications of EB, a rare and life limiting condition. This designation, alongside the other significant efforts of our team globally, drives our continued progress and brings us closer to realising our vision of becoming a global leader in rare and orphan diseases." 

The Fast Track programme is designed to accelerate the development and review of products such as AP101, which are intended to treat serious diseases and for which there is an unmet medical need. Fast Track designation enables more frequent communication with the FDA and may allow for further benefit from FDA accelerated programmes such as priority review and/or rolling review. 

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About Amryt

Amryt 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.

Amryt's commercial business comprises two orphan disease products.

Juxtapid®/ Lojuxta® (lomitapide) is approved as an adjunct to a low-fat diet and other lipid-lowering medicinal products for adults with the rare cholesterol disorder, Homozygous Familial Hypercholesterolaemia ("HoFH") in the US, Canada, Columbia, Argentina and Japan (under the trade name, Juxtapid®) and in the EU (under the trade name, Lojuxta®). HoFH is a rare genetic disorder which impairs the body's ability to remove low density lipoprotein ("LDL") cholesterol ("bad" cholesterol) from the blood, typically leading to abnormally high blood LDL cholesterol levels in the body from before birth - often ten times more than people without HoFH - and subsequent aggressive and premature cardiovascular disease.

Myalept® / Myalepta® (metreleptin) is approved in the US (under the trade name, Myalept®) as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy (GL) and in the EU (under the trade name, Myalepta®) for the treatment of leptin deficiency in patients with congenital or acquired GL in adults and children two years of age and above and familial or acquired partial lipodystrophy (PL) in adults and children 12 or over for whom standard treatments have failed to achieve adequate metabolic control. Metreleptin is also approved for lipodystrophy in Japan. Generalised and partial lipodystrophy are rare disorders characterised by loss or lack of adipose tissue resulting in the deficiency of the hormone leptin, produced by fat cells and are associated with severe metabolic abnormalities including severe insulin resistance, diabetes, hypertriglyceridemia and fatty liver disease.

Amryt's lead development candidate, AP101 (Oleogel-S10), is a potential treatment for the cutaneous manifestations of Epidermolysis Bullosa ("EB"), a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment. It is currently being studied in a Phase 3 clinical trial and recently reported that unblinded interim efficacy data supported continuation of the study with a modest increase in sample size and unblinded interim safety data allowed the inclusion of children from as young as 21 days old. AP101 has been granted FDA Pediatric Rare Disease Designation. The European and US market opportunity for EB is estimated by the Directors to be in excess of $1 billion.

In March 2018, Amryt in-licenced a pre-clinical gene-therapy platform technology, AP103, which offers a potential treatment for patients with Recessive Dystrophic Epidermolysis Bullosa, a subset of EB, and is also potentially relevant to other genetic disorders. 

For more information on Amryt, including products, please visit www.amrytpharma.com

This announcement contains inside information for the purposes of article 7 of the Market Abuse Regulation (EU) 596/2014