NEW HAVEN –(BUSINESS WIRE)– Alexion Pharmaceuticals, Inc. besteedt samen met de gemeenschap voor zeldzame ziekten aandacht aan de negende Internationale Dag voor Zeldzame Ziekten. De dag valt ieder jaar op de laatste dag van februari en vraagt aandacht voor zeldzame ziekten en hun invloed op patiënten en hun gezinnen. Werknemers van Alexion neemt deel aan evenementen over de hele wereld voor meer bewustzijn over zeldzame ziekten als kritiek probleem voor de wereldgezondheid, dat blijvend onderzoek, overheidsbeleid, onderwijs en nieuwe behandelmethodes vereist.
Het internationale thema luidt dit jaar ‘De stem van de patiënt’. De nadruk ligt daarbij op een beter begrip van de ervaringen die patiënten met zeldzame ziekten hebben. De dag vraagt aandacht voor de unieke uitdagingen waarvoor deze gemeenschap staat. Alexion steunt de hoofddoelen van Rare Disease Day 2016 met de ontwikkeling en levering van therapieën die overal ter wereld de levens van patiënten met slopende, zeldzame ziekten transformeren.
Alexion Employees Across the Globe Join Together to Support Rare Disease Day 2016
NEW HAVEN, Conn.–(BUSINESS WIRE)– Alexion Pharmaceuticals, Inc. (Nasdaq:ALXN) joins the worldwide rare disease community in honoring the ninth annual international Rare Disease Day. Rare Disease Day takes place the last day of February each year and is dedicated to raising awareness of rare diseases and their impact on patients and families. Alexion employees will participate in events around the globe to help raise awareness of rare diseases as a critical global health issue requiring ongoing research, public policy, disease education and new treatments.
This year’s global theme, “Patient Voice,” highlights the need for greater understanding of the experiences of rare disease patients and calls attention to the unique challenges faced by this community. Alexion supports the key objectives of Rare Disease Day 2016 through its mission to develop and deliver life-transforming therapies for patients around the world living with devastating and rare diseases.
“Today serves as a special milestone for Alexion as we celebrate Rare Disease Day by dedicating our new global headquarters in New Haven to the patients who inspire us every day to work with a sense of urgency to deliver medical breakthroughs where none currently exist,” said David Hallal, Chief Executive Officer of Alexion. “Alexion’s 3,000 employees around the world will participate in activities that demonstrate our commitment to transform lives and create lasting impact for the patients and families we serve today, and the generations that follow.”
Alexion’s Rare Disease Day 2016 activities include:
Today, Alexion has three innovative therapies to serve patients with four devastating ultra-rare diseases and we are partnering with governments worldwide to ensure patients have access to our life-transforming therapies. Throughout the year, Alexion’s research and development teams dedicate themselves to understanding the underlying causes of rare diseases and discovering breakthrough therapies to treat them. Alexion is currently advancing the most robust rare disease pipeline in the biotech industry, which includes 10 clinical programs and 30 pre-clinical programs, with at least four moving into the clinic in 2016. To learn more about Alexion’s R&D programs, visit www.alexion.com/pipeline.
About Rare and Ultra-Rare Disorders
In the United States, a disease is defined as rare if it affects fewer than 650 patients per million of the population. 1 The European Union definition of a rare disease is one that affects fewer than five patients per 10,000 of the population. 2 In contrast, a disease is generally considered ultra-rare if it affects fewer than 20 patients per million of the population 3 (one patient per 50,000) – and most ultra-rare diseases affect far fewer people than this.
Despite the very small numbers of patients they affect, the impact of rare and ultra-rare diseases on patients, their families, and society is profound, as many of these conditions are severe, chronic and progressive, with significant premature mortality. Patients with severe and life-threatening ultra-rare diseases often live without hope and have no effective treatments.
Alexion is a global biopharmaceutical company focused on developing and delivering life-transforming therapies for patients with devastating and rare disorders. Alexion is the global leader in complement inhibition and has developed and markets a treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two life-threatening ultra-rare disorders. In addition, Alexion’s metabolic franchise includes two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D). In addition, Alexion is advancing the most robust rare disease pipeline in the biotech industry with highly innovative product candidates in multiple therapeutic areas. This press release and further information about Alexion can be found at: www.alexion.com.
1. U.S. Food and Drug Administration. Definition of Disease Prevalence for Therapies Qualifying Under Orphan Drug Act:http://www.fda.gov/ForConsumers/ConsumerUpdates/ucm387513.htm
2. REGULATION (EC) No 141/2000 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 16 December 1999 on orphan medicinal products. http://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32000R0141&qid=1421232987002&from=EN
3. REGULATION (EU) No 536/2014 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 16 April 2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC. http://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32014R0536&qid=1421232837997&from=EN
Alexion Pharmaceuticals, Inc.