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NHS England Commissions Soliris for the Treatment of Atypical Haemolytic Uraemic Syndrome (aHUS).

WEYBRIDGE, England, September 12, 2013 /PRNewswire/ --

Commissioning decision follows positive Clinical Priorities Advisory Group (CPAG) recommendation and will enable initial national funding of Soliris for children and adults with aHUS in England

CPAG positive recommendation reflects 2 [sup.nd] independent body to issue a positive recommendation to fund Soliris for children and adults with aHUS in England

NHS England decision provides initial national patient a cc ess to Soliris as Ministers of Health have placed another hurdle to patient care with referral to the 3 [sup.rd] independent body asked to review the only approved aHUS treatment; the National Institute for Health and Care Excellence (NICE) decision anticipated in 2014

Alexion Pharma UK, a subsidiary of Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN), today announced that Soliris[sup.](eculizumab) is now commissioned by NHS England for the treatment of children and adults with atypical haemolytic uraemic syndrome (aHUS). The Department of Health has stated that this policy, which follows a very similar and positive recommendation from both the Advisory Group for National Specialized Services (AGNSS) and the Clinical Priorities Advisory Group (CPAG) in 2013, will be an initial national policy providing patient access to Soliris, pending evaluation and a final decision by what would be the 3[sup.rd] independent body - the National Institute for Health and Care Excellence (NICE) - which is currently anticipated in spring 2014.

Soliris was referred to NICE by Ministers of Health as the first topic for evaluation in its Highly Specialised Technologies Programme. In the interim, and according to NHS England, given the serious nature of the disease, NHS England will fund eculizumab for the treatment of new patients with aHUS (defined as those with a functioning kidney) and for existing patients who are on dialysis and are suitable for a kidney transplant. A commissioning for evaluation scheme will be developed for patients who are not suitable for transplant. NHS England will not routinely commission eculizumab for patients currently diagnosed with aHUS who have not received approval for eculizumab from an existing commissioning body.[sup.[] [sup.1] [sup.]]

Alexion looks forward to the completion and conclusion of the NICE evaluation, which is expected in spring 2014, so that patients with aHUS in England may continue to receive treatment.

aHUS is an ultra-rare, hereditary condition that is severe, life-threatening and chronic, and progressively damages vital organs, often leading to sudden and catastrophic damage to the kidney, brain, heart, and other vital organs, and premature death.[sup.[] [sup.2] [sup.]] [sup.,] [sup.[] [sup.3] [sup.]] [sup.,] [sup.[] [sup.4] [sup.]] It occurs when small blood clots form in blood vessels throughout the body.[sup.[] [sup.2] [sup.]] [sup.,] [sup.[] [sup.3] [sup.]] aHUS affects both adults and children and often presents at a very young age.[sup.[] [sup.5] [sup.]] Sixty-five percent of all patients with aHUS require kidney dialysis, have permanent kidney damage or die within the first year after diagnosis.[sup.[] [sup.6] [sup.]] [sup.,] [sup.[] [sup.7] [sup.]]

"aHUS is a disease that is both ultra-rare and life-threatening. As such, aHUS affects very few people but is extremely devastating for those patients and their families. We are very pleased that NHS England has approved initial national funding and patient access to the only approved treatment of aHUS in England, and look forward to confirmation by NICE of a final policy," said Mark Barrett, Country Manager, Alexion Pharma UK.

Soliris was approved in the European Union for the treatment of adult and paediatric patients with aHUS in November 2011.

References:

1. NHS England (2013). Clinical commissioning policy statement: Eculizumab for atypical haemolytic uraemic syndrome. Available at http://bit.ly/17W9vTc. Last accessed 11 September 2013.

2. Noris M, Remuzzi G (2009). Atypical hemolytic-uremic syndrome. N Engl J Med 361:1676-87

3. Benz K, Amann K (2010). Thrombotic microangiopathy: new insights. Curr Opin Nephrol Hypertens;19(3):242-7

4. Tsai HM (2006). The molecular biology of thrombotic microangiopathy. Kidney Int ;70(1):16-23

5. Bresin E, Daina E, Noris M, et al (2006) International Registry of Recurrent and Familial HUS/TTP. Outcome of renal transplantation in patients with non-Shiga toxin-associated hemolytic uremic syndrome: prognostic significance of genetic background. Clin J Am Soc Nephrol. 1:88-99

6. 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

7. Loirat C, Garnier A, Sellier-Leclerc AL et el (2010). Plasmatherpay in atypical hemolytic uremic syndrome., Semin Thromb Hemost. 2010;36:673-681

8. Abstract 1587 entitled "A phase II study of eculizumab in patients with atypical hemolytic uremic syndrome receiving chronic plasma exchange/infusion," presented by Dr. Chantal Loirat at the 16th Congress of the European Hematology Association, Sunday, June 12, 2011.

9. Abstract 1588 entitled "Eculizumab efficacy and safety in patients with atypical hemolytic uremic syndrome resistant to plasma exchange/infusion," presented by Dr. Chantal Loirat at the 16th Congress of the European Hematology Association, Sunday, June 12, 2011.

10. (13) Abstract 396 entitled "Eculizumab therapy for atypical hemolytic uremic syndrome in pediatric patients: Efficacy and safety outcomes from a retrospective study," presented by Dr. Giacomo Simonetti at the 16th Congress of the European Hematology Association, Friday, June 10, 2011.

11. Noris M, Caprioli J, Bresin E, et al (2010). Relative role of genetic complement abnormalities in sporadic and familial aHUS and their impact on clinical phenotype. Clin J Am Soc Nephrol. 5:1844-1859

Notes to Editors:

About aHUS

aHUS is a chronic, ultra-rare, and life-threatening disease in which a genetic deficiency in one or more complement regulatory genes causes chronic uncontrolled complement activation, resulting in complement-mediated thrombotic microangiopathy (TMA), the formation of blood clots in small blood vessels throughout the body.[sup.[] [sup.2] [sup.]] [sup.,] [sup.[] [sup.3] [sup.]] Permanent, uncontrolled complement activation in aHUS causes a life-long risk for TMA, which leads to sudden, catastrophic, and life-threatening damage to the kidney, brain, heart, and other vital organs, and premature death.[sup.[] [sup.3] [sup.]] [sup.,] [sup.[] [sup.4] [sup.]] Sixty-five percent of all patients with aHUS die, require kidney dialysis or have permanent kidney damage within the first year after diagnosis despite plasma exchange or plasma infusion (PE/PI).[sup.[] [sup.6] [sup.]] [sup.,] [sup.[] [sup.7] [sup.]] The majority of patients with aHUS who receive a kidney transplant commonly experience subsequent systemic TMA, resulting in a 90% transplant failure rate.[sup.[] [sup.5] [sup.]]

aHUS affects both children and adults. In a large group of aHUS patients, 60% were first diagnosed at younger than 18 years of age.[sup.[5]] Complement-mediated TMA also causes reduction in platelet count (thrombocytopenia) and red blood cell destruction (haemolysis). While mutations have been identified in at least ten different complement regulatory genes, mutations are not identified in 30-50% of patients with a confirmed diagnosis of aHUS.[sup.[] [sup.11] [sup.]]

About Soliris

Soliris is a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval and commercialization 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, characterized by complement-mediated hemolysis (destruction of red blood cells).

Soliris is also approved in the US, the European Union and Canada 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 characterized 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. Soliris is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).

More information including the full prescribing information on Soliris is available at:

http://www.medicines.org.uk/emc/searchresults.aspx?term=Soliris&searchtype=QuickSearch

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. 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.

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 all special warnings and precautions.

About Alexion

Alexion Pharmaceuticals, Inc. is a biopharmaceutical company focused on the development of treatments for severe and ultra-rare disorders through the innovation, development and commercialisation of innovative therapeutic products. Alexion is the global leader in complement inhibition.
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