Amryt Supports Global Rare Disease Day 2021
Amryt Supports Global Rare Disease Day 2021
DUBLIN, Ireland, and Boston MA, February 25, 2020, Amryt (Nasdaq: AMYT, AIM: AMYT), a global, commercial-stage biopharmaceutical company focussed on acquiring, developing and commercializing novel treatments for rare diseases, is pleased to announce its support for Global Rare Disease Day 2021 which will take place on Sunday, February 28, 2021.
Global Rare Disease Day represents a special opportunity to hear the voice of rare disease patients across the world and to highlight the burden and impact of living with a rare disease. Globally, there are an estimated 300 million people living with rare or ultra-rare diseases of which there are more than 7,000 different types. Approximately 80% of rare and ultra-rare diseases are inherited and sadly, it is estimated that a third of rare disease patients will die before their fifth birthday.
Orphan diseases are a global health problem that affect people of all nationalities, races, ethnicities, genders, ages and socio-economic groups. Although the overall number of people affected collectively is large, rare disease patients are often neglected by research and health systems. This is even more of a challenge for patients with ultra-rare diseases, the burden for patients at the rarest end of the spectrum being even greater.
To help highlight the unique challenges of rare disease and as a mark of recognition of the importance of Global Rare Disease Day, Amryt has published a Rare and Ultra-Rare Disease Prioritization and Sustainability Report to support ongoing recognition and investment in this important and underserved area.
Dr Joe Wiley, CEO of Amryt Pharma, commented today: “We are delighted to be able to support Rare Disease Day 2021 through the publication of our Rare and Ultra Rare Prioritisation and Sustainability Report. This report, with contributions from 17 different organisations around the world, helps to highlight the challenges we face when trying to develop new medicines for rare diseases and calls for greater support for sustainable investment in finding treatments for rare and ultra-rare diseases.”
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 two orphan disease products – metreleptin (Myalept®/ Myalepta®) and lomitapide (Juxtapid®/ Lojuxta®).
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.
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, Israel and Brazil (under the trade name Lojuxta®). For additional information, please follow this link.
Amryt's lead development candidate, Filsuvez® (Oleogel-S10) is a potential treatment for the cutaneous manifestations of Junctional and Dystrophic Epidermolysis Bullosa (“EB”), a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment. Filsuvez® has been selected as the brand name for Oleogel-S10. The product does not currently have regulatory approval to treat EB.
Amryt’s pre-clinical gene therapy platform, AP103, offers a potential treatment for patients with Dystrophic EB, and is also potentially relevant to other genetic disorders.
For more information on Amryt, including products, please visit www.amrytpharma.com.
The person making this notification on behalf of Amryt is Rory Nealon, CFO/COO and Company Secretary.
Shore Capital (Edward Mansfield, Daniel Bush, John More) are NOMAD and Joint Broker to Amryt in the UK. Stifel (Ben Maddison) are Joint Broker to the company in the UK. Davy (John Frain, Daragh O’Reilly) act as Joint Broker to the company.
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.
Joe Wiley, CEO / Rory Nealon, CFO/COO, +353 (1) 518 0200, email@example.com