2013-01-23 20:43:54 -
Alexion Pharma UK, a subsidiary of Alexion Pharmaceuticals, Inc., has been informed by the National Specialised Commissioning Team (NSCT) that the Ministers of Health have decided to not follow a positive recommendation by the Advisory Group for National Specialised Services (AGNSS) when assessing Soliris® (eculizumab) as a treatment for patients with atypical haemolytic uraemic syndrome (aHUS). It is important to underscore that the UK Government developed a specific process and authorised a committee, AGNSS, to evaluate treatments addressing patients with very rare disorders, and that AGNSS concluded that “eculizumab should be routinely nationally commissioned for patients with aHUS.” AGNSS further stated that, “Eculizumab would help save lives and improve the quality of life for children (among whom the condition is
particularly prevalent) and adults with atypical haemolytic uraemic syndrome.” However, the Ministers of Health have overruled this expert committee recommendation and instead referred consideration of Soliris for the treatment of patients with aHUS to the National Institute for Health and Clinical Excellence (NICE).
aHUS is an unpredictable and life-threatening disease that affects both children and adults. More than 50% of patients with aHUS experience devastating consequences, including kidney failure, require dialysis or die within 1 year of diagnosis. 1,2 Soliris was approved by the European Commission in 2011 specifically for the treatment of patients suffering with aHUS and is the only approved treatment option available. The Health Ministers decision to refer the Soliris assessment to NICE comes over one and one-half years after the initial application was submitted by the lead physician expert at Newcastle-upon Tyne Hospitals NHS Foundation Trust, and following the positive recommendation by the authorised committee AGNSS. This action pushes the Health Ministers decision further into the future, and with an indefinite timeframe, despite abundant clinical evidence on the safety and efficacy of Soliris for these patients. Alexion is gravely disappointed by the announcement and the underlying departure by the UK Government from an agreed formal process. The significant delay in the Health Ministers decision directly threatens the lives of aHUS patients in England by causing further and unnecessary delays in access to safe and effective treatment.
Alexion is committed to working with the Health Ministers to make Soliris available to children and adults as quickly as possible, and calls upon the Ministers to accept the recommendation of the significant number of experts and lay people called upon to review Soliris without delay, and to reverse his decision and accept the recommendation of AGNSS in the best interests of aHUS patients in England.
It is important to underscore that the Government developed a specific process and authorised the committee, AGNSS, to evaluate treatments addressing patients with very rare disorders, and that AGNSS concluded that “eculizumab should be routinely nationally commissioned for patients with aHUS.” AGNSS further stated that, “Eculizumab would help save lives and improve the quality of life for children (among whom the condition is particularly prevalent) and adults with atypical haemolytic uraemic syndrome.” Specifically, in June 2011, Newcastle-upon Tyne Hospitals NHS Foundation Trust submitted an application for national commissioning of a service for patients with aHUS, and the application was accepted by AGNSS. In January 2012, Alexion submitted a full dossier of data and a complete assessment to AGNSS as part of a well-defined process for review. The dossier included strong clinical data from the aHUS registration trials, demonstrating that 100% of patients had a response to therapy and that Soliris had a significant beneficial impact on TMA and patient morbidities for patients with aHUS. Based on these data, and as communicated on January 22 by the NCST to Alexion, AGNSS recommended to the Health Minister that “eculizumab should be routinely nationally commissioned for patients with aHUS”; yet the Minister chose instead to defer the decision to a new process that is neither yet defined nor will apparently commence until April 2013 at the earliest.
In the meantime, children and adults in England suffering from a life-threatening and ultra-rare condition continue to wait while other patients with aHUS around the world are receiving treatment and hope for a better life.
aHUS is an ultra-rare, life-threatening, chronic, genetic disease that progressively damages vital organs, often leading to sudden and catastrophic damage to the kidney, brain, heart, and other vital organs, and premature death. 3-5 More than 50% of patients have kidney failure, require dialysis or die within 1 year of diagnosis. 1,2 In November 2011, the European Commission approved Soliris as the first and only treatment for children and adults with aHUS.
1. Caprioli J, Noris M, Brioschi S, et al for the International Registry of Recurrent and Familial HUS/TTP (2006). Genetics of HUS: the impact of MCP, CFH, and IF mutations on clinical presentation, response to treatment, and outcome. Blood.108:1267-1279
2. Loirat C, Garnier A, Sellier-Leclerc AL et el (2010). Plasmatherpay in atypical hemolytic uremic syndrome., Semin Thromb Hemost.
3. Noris M, Remuzzi G (2009). Atypical hemolytic-uremic syndrome. N Engl J Med 361:1676-87
4. Benz K, Amann K (2010). Thrombotic microangiopathy: new insights.
Curr Opin Nephrol Hypertens;19(3):242-7
5. Tsai HM (2006). The molecular biology of thrombotic microangiopathy.
Kidney Int ;70(1):16-23
Notes to Editors:
Soliris is a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval and commercialisation by Alexion. Soliris is approved in the US, European Union, Japan and other countries as the first and only treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, ultra-rare and life-threatening blood disorder, characterised by complement-mediated hemolysis (destruction of red blood cells). In PNH evidence of clinical benefit of Soliris is limited to patients with a history of transfusions. Soliris is also approved in the US as the first and only treatment for patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy, a debilitating, ultra-rare and life-threatening genetic disorder characterised by complement-mediated thrombotic microangiopathy (blood clots in small vessels). The effectiveness of Soliris in aHUS is based on the effects on thrombotic microangiopathy (TMA) and renal function. Prospective clinical trials in additional patients are ongoing to confirm the benefit of Soliris in patients with aHUS. Alexion's breakthrough approach in complement inhibition has received the pharmaceutical industry's highest honours: the 2008 Prix Galien USA Award for Best Biotechnology Product with broad implications for future biomedical research and the 2009 Prix Galien France Award in the category of Drugs for Rare Diseases. More information including the full prescribing information on Soliris is available at.
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Important Safety Information
In Europe, the Summary of Product Characteristics (SmPC) for Soliris includes a special warning and precaution for use: "Due to its mechanism of action, the use of Soliris increases the patient's susceptibility to meningococcal infection (Neisseria meningitidis). These patients might be at risk of disease by uncommon serogroups (particularly Y, W135 and X), although meningococcal disease due to any serogroup may occur. To reduce the risk of infection, all patients must be vaccinated at least 2 weeks prior to receiving Soliris. PNH patients must be vaccinated 2 weeks prior to Soliris initiation. aHUS patients who are treated with Soliris less than 2 weeks after receiving a meningococcal vaccine must receive treatment with appropriate prophylactic antibiotics until 2 weeks after vaccination. Patients must be re-vaccinated according to current medical guidelines for vaccination use. Tetravalent vaccines against serotypes A, C, Y and W135 are strongly recommended, preferably conjugated ones. Vaccination may not be sufficient to prevent meningococcal infection. Consideration should be given to official guidance on the appropriate use of antibacterial agents. Cases of serious or fatal meningococcal infections have been reported in Soliris treated patients. All patients should be monitored for early signs of meningococcal infection, evaluated immediately if infection is suspected, and treated with antibiotics if necessary. Patients should be informed of these signs and symptoms and steps taken to seek medical care immediately." Physicians must discuss the benefits and risks of Soliris therapy with patients and provide them with a patient information brochure and a patient safety card.
The most common or serious adverse reactions are headache (occurred mostly in the initial phase), leukopenia and meningococcal infection.
The most common (>10%) adverse reactions reported in paediatric aHUS patients were diarrhoea, vomiting, pyrexia, upper respiratory tract infection and headache. Soliris treatment should not alter anticoagulant management. Please see Summary of Product Characteristics for full prescribing information for Soliris, including information regarding risk of serious meningococcal infection.
Alexion Pharmaceuticals, Inc. is a biopharmaceutical company focused on serving patients with severe and ultra-rare disorders through the innovation, development and commercialisation of life-transforming therapeutic products. Alexion is the global leader in complement inhibition and has developed and markets Soliris® (eculizumab) as a treatment for patients with PNH and aHUS, two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation. Soliris is currently approved in more than 40 countries for the treatment of PNH, and in the United States and the European Union for the treatment of aHUS. Alexion is evaluating other potential indications for Soliris and is developing four other highly innovative biotechnology product candidates.
Safe Harbor Statement
This news release contains forward-looking statements, including statements related to anticipated clinical development, regulatory and commercial milestones and potential health and medical benefits of Soliris® (eculizumab) for the potential treatment of patients with aHUS.
Forward-looking statements are subject to factors that may cause Alexion's results and plans to differ from those expected, including for example, decisions of regulatory authorities regarding marketing approval or material limitations on the marketing of Soliris for its current or potential new indications, and a variety of other risks set forth from time to time in Alexion's filings with the Securities and Exchange Commission, including but not limited to the risks discussed in Alexion's Quarterly Report on Form 10-Q for the period ended September 30, 2012, and in Alexion's other filings with the Securities and Exchange Commission. Alexion does not intend to update any of these forward-looking statements to reflect events or circumstances after the date hereof, except when a duty arises under law.
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