Alexion Pharma Nordics AB
Providing Innovative and Life-Transforming
Therapies to Patients with Severe and Life-Threatening Rare Diseases
Alexion is a global biopharmaceutical company that is exclusively focused on discovering and developing life-transforming therapies for forgotten populations of patients with devastating and life-threatening rare conditions. We are driven because we know these patients depend on us to deliver the innovative and life-changing therapies they so desperately need.
According to the European Commission, a condition is generally considered to be rare if it affects fewer than 5 patients per 10,000 people. Ultra-rare diseases are considered to affect fewer than 20 patients per million of population (or one patient per 50,000), and most ultra-rare diseases will affect far fewer than this. There are between 6,000-8,000 rare and ultra-rare diseases that have been currently identified, and patients with these devastating conditions often have no effective treatment options and little hope.
Rare and ultra-rare diseases present particular challenges as they affect very small numbers of patients. But the impact of these rare and ultra-rare diseases on patients, their families, and society can be profound as many are severe, chronic and progressive, with high mortality rates. Ultra-rare diseases also present unique public health issues due to a lack of scientific knowledge and a limited number of physicians who are familiar with diagnosing and treating them. This often leads to misdiagnosis, delayed diagnosis and inappropriate treatment.
Alexion works closely with various physicians and patient groups to better understand the barriers that patients with rare diseases face in terms of a timely diagnosis and access to treatment. These include low awareness of the condition among healthcare professionals, poor understanding of the disease in general, lack of local expertise and few and far specialist reference centres meaning that many patients may need to travel long distances to gain access to suitable care. Alexion provides education and disease awareness programs to educate physicians about how to best diagnose two ultra-rare diseases called PNH and aHUS so that the physicians can make a rapid and accurate diagnosis, then start patients on appropriate treatment.
Alexion in the Nordics
Alexion Pharma Nordics AB is a subsidiary of Alexion Pharmaceuticals. Alexion Pharma Nordics has operations in Sweden, Norway and Denmark to serve patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two severe and life-threatening ultra-rare diseases.
Additionally, Alexion partners with leading research institutions in the Nordics, such as the world renowned Karolinska University Hospital in Sweden and the Uppsala University Hospital, to enroll patients in their clinical trials and patient registries. Alexion is currently undertaking two global trials in the area of transplantation at four centres in the Nordics, three of which are based in Sweden. Both trials are evaluating Alexion’s treatment as a way of stopping organ rejection in a particular subset of transplant patients with antibodies against donors and who have received a kidney from either a diseased or living donor, thus increasing their chances to retain the new kidney. Recruitment is currently ongoing for both these trials.
Commitment to innovation
The biopharmaceutical industry faces several challenges in the research and development of effective treatments, and these challenges are magnified for rare and ultra-rare disorders. At Alexion, our goal is to develop highly innovative and breakthrough therapies that will dramatically transform the lives of patients with rare diseases. We are the leaders in complement inhibition and have developed and market a treatment for patients with PNH and aHUS. Both of these diseases affect extremely small number of patients but are severe and devastating for patients and their families. If untreated, both of them can significantly impact the quality of lives and may also cause premature death in a significant amount of patients. Alexion’s treatment is the only one approved, and it is now approved in nearly 50 countries for the treatment of PNH and in nearly 40 countries for the treatment of aHUS.
Additionally, we are developing a strong portfolio of innovative new therapies that have the potential to be medical breakthroughs. We are currently evaluating other potential indications for our marketed drug and investigating other highly innovative product candidates across multiple severe and ultra-rare disorders. Out of these, two have already been awarded the Breakthrough Therapy designation by the US Food and Drug Agency (FDA) in 2013. The first is an enzyme replacement therapy for hypophosphatasia (HPP), a genetic, multi-system and life-threatening ultra-rare metabolic disorder. The second is an investigational candidate for MoCD Type A, a severe and genetic ultra-rare metabolic disease that can be fatal in newborns. There are currently no treatment options for patients with HPP or MoCD Type A.
In summary, at Alexion we strive to understand the needs of the patients we serve and how urgently they must be met so that patients diagnosed with a rare and devastating disease can have access to life-transforming treatments.