Amryt Pharma plc Amryt Granted Orphan Drug Designation By The Fda For Ap103

 
TIDMAMYT 
 
 
   Amryt Granted Orphan Drug Designation by the FDA for AP103 
 
   DUBLIN, Ireland, and Boston MA, December 23, 2020, Amryt (Nasdaq: AMYT, 
AIM: AMYT), 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 today 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 <BETA>-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." 
 
   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. 
 
   Amryt's lead development candidate, FILSUVEZ(R) (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.  FILSUVEZ(R) has been selected as the brand name for the 
product. Amryt does not have regulatory approval for FILSUVEZ(R) to 
treat EB. In September and October 2020, Amryt reported positive results 
from its pivotal global Phase 3 trial in EB. The product has been 
granted Rare Pediatric Disease Designation and has also received a Fast 
Track Designation from the U.S. Food and Drug Administration. 
 
   Myalept(R) / Myalepta(R) (metreleptin) is approved in the US (under the 
trade name Myalept(R)) 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(R)) 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 years of age and above 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. 
 
   Juxtapid(R)/ Lojuxta(R) (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(R)) and in the EU and Brazil (under the trade name 
Lojuxta(R)). 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. 
 
   In March 2018, Amryt in-licensed a pre-clinical gene-therapy platform 
technology, AP103, which offers a potential treatment for patients with 
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.  The person 
making this notification on behalf of Amryt is Rory Nealon, CFO/COO and 
Company Secretary. 
 
   Forward-Looking Statements 
 
   This press release may contain forward-looking statements containing the 
words "expect", "anticipate", "intends", "plan", "estimate", "aim", 
"forecast", "project" and similar expressions (or their negative) 
identify certain of these forward-looking statements. The 
forward-looking statements in this announcement are based on numerous 
assumptions and Amryt's present and future business strategies and the 
environment in which Amryt expects to operate in the future. 
Forward-looking statements involve inherent known and unknown risks, 
uncertainties and contingencies because they relate to events and depend 
on circumstances that may or may not occur in the future and may cause 
the actual results, performance or achievements to be materially 
different from those expressed or implied by such forward-looking 
statements. These statements are not guarantees of future performance or 
the ability to identify and consummate investments. Many of these risks 
and uncertainties relate to factors that are beyond each of Amryt's 
ability to control or estimate precisely, such as future market 
conditions, the course of the COVID-19 pandemic, currency fluctuations, 
the behaviour of other market participants, the outcome of clinical 
trials, the actions of regulators and other factors such as Amryt's 
ability to obtain financing, changes in the political, social and 
regulatory framework in which Amryt operates or in economic, 
technological or consumer trends or conditions. Past performance should 
not be taken as an indication or guarantee of future results, and no 
representation or warranty, express or implied, is made regarding future 
performance. No person is under any obligation to update or keep current 
the information contained in this announcement or to provide the 
recipient of it with access to any additional relevant information that 
may arise in connection with it. Such forward-looking statements reflect 
the Company's current beliefs and assumptions and are based on 
information currently available to management. 
 
   Contacts 
 
   Joe Wiley, CEO, [phone], [e-mail] 
https://www.globenewswire.com/Tracker?data=kS56Jjwvc-MvULTTeUgo4XMo4bJWYbEAcTquaFrfpLJ9RwXZiREB4xv3Zrys20Jvx_apXkJdbpiOaRMwVpnvb1zFKjUb5bhxxNSKLeOCSxAlLEuO9u13NH8mrMoUWvuD 
 
 
   Rory Nealon, CFO/COO, [phone], [e-mail] 
https://www.globenewswire.com/Tracker?data=-fkMl7T3NdxCVsRE-nbPr5wL6-mLTylEKgJrS2ygv7BOjQ3s4hcItGn5j9lTx9Prg1dUJIbFreLKf3GBYFBzQ_CvocuC8Hhm7RJXmQgwCKBx1tx-8bX23ucjngIL6W_K 
 
 
   Edward Mansfield, Shore Capital, NOMAD, [phone], 
[e-mail] 
 
   Tim McCarthy, LifeSci Advisors, LLC, [phone], 
[e-mail] 
https://www.globenewswire.com/Tracker?data=tqUMdSSByDFeg6LYwudLOK_uKgSftji7SHn9GPDwZmfE6wbi0ATSwubke7FKPcnHna81a9aC31zKFad19DZl-64Sw0IMljBNUELrJcYDrzM= 
 
 
   Amber Fennell, Consilium Strategic Communications, [phone], 
[e-mail] 
https://www.globenewswire.com/Tracker?data=DtV0dM6hONc8AB2pJx1-VOVAbcCJ6u13t3zXD1i-oxC5pL2N86jEIsnaz-NG8ZpwUAw7qEhsURJZhaZHgPTQ6VEWfPQorPnBPFAC084ygyHyoZIzK7i_Kdd7LU-64hg_ 
 
 
 
 
 

(END) Dow Jones Newswires

December 23, 2020 02:00 ET (07:00 GMT)

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