Amryt Supports FH Awareness Day – September 24, 2022
DUBLIN, Ireland, and Boston MA, September 23, 2021, Amryt (Nasdaq: AMYT, AIM: AMYT), a global, commercial-stage biopharmaceutical company dedicated to acquiring, developing and commercializing novel treatments for rare diseases, announces its support today for FH Awareness Day 2022 which will take place on Saturday, September 24, 2021.
Dr Joe Wiley, CEO of Amryt Pharma, commented: “All of the Amryt team are pleased to support the great work of the Family Heart Foundation and FH Europe as they seek to raise awareness of this rare and devastating disease. Those with HoFH often go undiagnosed for years and raising awareness is critical to accurate and timely diagnosis that improves outcomes for patients. For our part at Amryt, we are collaborating with all stakeholders to continue to deliver therapies to patients in desperate need.”
About FH and HoFH
Familial Hypercholesterolaemia (“FH”) is an inherited genetic condition that leads to levels of cholesterol that are much higher than that of the general population.i FH is the most common genetic condition leading to early heart disease.ii Millions of people around the world do not know that they and their families are at severe risk for early heart disease, heart attacks, and even death. Nine out of ten people born today with familial hypercholesterolemia are undiagnosed. Yet, with early diagnosis and treatment, individuals diagnosed with FH can reduce their risk for heart disease by 80%.iii
Homozygous Familial Hypercholesterolaemia (“HoFH”) is a rare and severe form of FH, causing excessive levels of low-density lipoprotein cholesterol (“LDL-C”) or ‘bad cholesterol’ to accumulate in the body from conception. People with HoFH are at a continued risk of progressive atherosclerotic disease and life-threatening cardiac events, for example, heart attack, stroke, major cardiac surgery and premature cardiac death. Untreated, most patients with markedly elevated LDL-C levels develop overt cardiovascular disease before the age of 20 years, and generally do not survive past 30 years.iv
Amryt is a global commercial-stage biopharmaceutical company focused on acquiring, developing and commercializing innovative 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 four orphan disease products – metreleptin (Myalept®/ Myalepta®); oral octreotide (Mycapssa®); lomitapide (Juxtapid®/ Lojuxta®); and Oleogel-S10 (Filsuvez®).
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®) as an adjunct to diet 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. For additional information, please follow this link.
Mycapssa® (octreotide capsules) is approved in the US for long-term maintenance therapy in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide. Mycapssa® is the first and only oral somatostatin analog approved by the FDA. Mycapssa® has also been submitted to the EMA and has received a positive opinion by the CHMP recommending the approval of Mycapssa® in the European Union (EU). For additional information, please follow this link.
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, Colombia, Argentina and Japan (under the trade name Juxtapid®) and in the EU, Israel, Saudi Arabia and Brazil (under the trade name Lojuxta®). For additional information, please follow this link.
Filsuvez® (Oleogel-S10) is approved in the EU and Great Britain for the treatment of partial thickness wounds associated with junctional and dystrophic Epidermolysis Bullosa in patients 6 months and older. Filsuvez® remains under review by the FDA.
Amryt’s pre-clinical gene therapy candidate, AP103, offers a potential treatment for patients with Dystrophic EB, and the polymer-based delivery platform has the potential to be developed for the treatment of other genetic disorders.
Amryt also intends to develop oral medications that are currently only available as injectable therapies through its Transient Permeability Enhancer (TPE®) technology platform. For more information on Amryt, including products, please visit www.amrytpharma.com.
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iv Cuchel M, et al. Eur Heart J 2014;35:2146–57