AMYT Amryt Pharma Plc

 
TIDMAMYT 
 
 
   Amryt Receives Reimbursement Approval from NICE for Myalepta(R) 
(metreleptin) in England and Wales 
 
   DUBLIN, Ireland, and Boston MA, January 20, 2021, Amryt (Nasdaq: AMYT, 
AIM: AMYT), a global, commercial-stage biopharmaceutical company 
dedicated to acquiring, developing and commercializing novel 
therapeutics to treat patients suffering from serious and 
life-threatening rare diseases, is pleased to announce that the National 
Institute for Health and Care Excellence ("NICE") has approved 
Myalepta(R) (metreleptin) for reimbursement in England and Wales as an 
adjunct to diet as replacement therapy for the treatment of leptin 
deficiency in patients with congenital or acquired general lipodystrophy 
(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.  Myalepta(R) received marketing authorisation from the 
European Medicines Agency ("EMA") in 2018. 
 
   Dr Joe Wiley, CEO of Amryt Pharma, commented today: "We are very excited 
to report NICE's approval of Myalepta(R) which will now give all England 
and Wales based patients suffering from lipodystrophy the opportunity to 
access Myalepta(R). Today's news represents further progress in our 
efforts to grow the geographic reach of our commercial products in both 
existing and new territories." 
 
   Rebecca Sanders, CEO of Lipodystrophy UK, commented today: 
"Lipodystrophy UK are ecstatic that NICE has approved metreleptin for 
the treatment of partial and generalised lipodystrophy. We have worked 
tirelessly with all stakeholders involved in this appraisal to ensure 
the voice of patients and their carers was heard. We are grateful to the 
clinical experts at the National Specialist Treatment Centre at 
Addenbrooke's Hospital, Amryt and NICE for their hard work and 
collaborative spirit throughout the appraisal. Lipodystrophy UK will 
continue to advocate for our community to ensure better treatment and 
care for everyone affected by lipodystrophy." 
 
   Professor Sir Stephen O'Rahilly, Director, MRC Metabolic Diseases Unit, 
University of Cambridge and Hon. Consultant Physician, Addenbrooke's 
Hospital, Cambridge commented today: "Patients with lipodystrophy can 
suffer severe metabolic complications as a result of being deficient in 
the fat cell hormone, leptin.  I am delighted that metreleptin, 
currently the only form of leptin replacement therapy, will now be 
available as a treatment for appropriate NHS patients with 
lipodystrophy. The National Severe Insulin Resistance Service at 
Addenbrooke's Hospital, Cambridge, where many of these patients are 
supported, has worked tirelessly with Amryt and NICE to assist them in 
reaching  a fair and equitable solution to providing this medication to 
the patients who need it.  Without the commitment and effort of people 
with lipodystrophy, working through their support group Lipodystrophy UK, 
none of this would have been possible. Today is a vindication of all 
their hard work." 
 
   About Lipodystrophy - Generalised (GL) and partial lipodystrophy (PL) 
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. 
 
   About Metreleptin - 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. 
 
   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, +353 (1) 518 0200, joe.wiley@amrytpharma.com 
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   Rory Nealon, CFO/COO, +353 (1) 518 0200, rory.nealon@amrytpharma.com 
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   Edward Mansfield, Shore Capital, NOMAD, +44 (0) 207 468 7906, 
edward.mansfield@shorecap.co.uk 
 
   Tim McCarthy, LifeSci Advisors, LLC, +1 (212) 915 2564, 
tim@lifesciadvisors.com 
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   Amber Fennell, Consilium Strategic Communications, +44 (0) 203 709 5700, 
fennell@consilium-comms.com 
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