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NICE Confirms AGNSS Positive Assessment that Eculizumab (SolirisĀ®) is a Very Effective Treatment for aHUS Patients and Produces Substantial Quality-Adjusted Life Year Gains of a Magnitude Rarely Seen

Companies mentioned in this article: Alexion Pharmaceuticals

CHESHIRE, Conn. -- (BUSINESS WIRE) -- Alexion Pharmaceuticals (Nasdaq:ALXN) is pleased that the National Institute for Health and Clinical Excellence (NICE) Evaluation Committee has acknowledged that eculizumab (Soliris®) is a very effective treatment option for patients with aHUS, and that the use of eculizumab would be of significant value to patients with this life-threatening disorder who have no other treatment options.

In an Evaluation Consultation Document (ECD) released today, the NICE Evaluation Committee confirmed that currently available supportive care for patients with aHUS has limited impact on disease morbidity and mortality, but a substantial negative effect on a patient’s quality of life. Importantly, the Committee underscored that substantial restoration of health for a very long period is achieved with ongoing treatment with eculizumab. The Committee also concluded that eculizumab produced a substantial gain in quality-adjusted life years (QALY, a measure of the number of high-quality years of life gained) of a magnitude rarely seen for a new drug treatment – let alone one for an ultra-rare disorder.

Today’s very positive assessment by NICE of the clinical value of eculizumab for patients with aHUS aligns with the two prior positive recommendations from Government – AGNSS and NHS England. In addition, NICE confirmed today that the broad interim NHS England commissioning policy for eculizumab treatment of existing and new patients with aHUS will continue.

However, Alexion is concerned that nearly 3 years after Government commenced its evaluation, and more than 1 year after eculizumab was referred to NICE, the Committee was still unable to provide a formal recommendation whether eculizumab should be nationally commissioned. Additionally, NICE has now stated that it will seek further information from NHS England on what considerations relating to the management of its specialised commissioning budget it considers should be taken into account. NICE has also asked the company to provide additional information related to budget impact. NICE has not requested any additional clinical effectiveness information from Alexion.

Currently, new and existing patients with aHUS in England are able to receive eculizumab through an interim policy commissioned by NHS England last year, and NICE confirmed today that this interim policy will remain in place pending the outcome of NICE’s appraisal. Alexion looks forward to confirmation by NICE of a final policy so that patients with aHUS in England may continue to receive treatment.

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 U.S. (2007), European Union (2007), Japan (2010) 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 indicated to reduce hemolysis. Soliris is also approved in the U.S. (2011), the European Union (2011), Japan (2013) and other countries as the first and only treatment for patients with atypical hemolytic uremic syndrome (aHUS), a debilitating, ultra-rare and life-threatening genetic disorder characterized by complement-mediated thrombotic microangiopathy, or TMA (blood clots in small vessels). Soliris is indicated to inhibit complement-mediated TMA. The effectiveness of Soliris in aHUS is based on its effects on TMA and renal function. Prospective clinical trials in additional patients, the preliminary results of which were reported at international nephrology and hematology conferences in 2013, are ongoing to confirm the benefit of Soliris in patients with aHUS. Soliris is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS). For the breakthrough innovation in complement inhibition, Alexion and Soliris have received the pharmaceutical industry's highest honors: 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 U.S. prescribing information on Soliris is available at www.soliris.net.

The full prescribing information on Soliris in Europe is available at: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/000791/WC500054208.pdf.

Important Safety Information

The U.S. product label for Soliris includes a boxed warning: "Life-threatening and fatal meningococcal infections have occurred in patients treated with Soliris. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients with complement deficiencies. Immunize patients with a meningococcal vaccine at least two weeks prior to administering the first dose of Soliris, unless the risks of delaying Soliris therapy outweigh the risk of developing a meningococcal infection. (See Serious Meningococcal Infections (5.1) for additional guidance on the management of meningococcal infection.) Monitor patients for early signs of meningococcal infections and evaluate immediately if infection is suspected. Soliris is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the Soliris REMS, prescribers must enroll in the program. Enrollment in the Soliris REMS program and additional information are available by telephone: 1-888-soliris (1-888-765-4747)."

In patients with PNH, the most frequently reported adverse events observed with Soliris treatment in clinical studies were headache, nasopharyngitis (runny nose), back pain and nausea. Soliris treatment of patients with PNH should not alter anticoagulant management because the effect of withdrawal of anticoagulant therapy during Soliris treatment has not been established. In patients with aHUS, the most frequently reported adverse events observed with Soliris treatment in clinical studies were hypertension, upper respiratory tract infection, diarrhea, headache, anemia, vomiting, nausea, urinary tract infection, and leukopenia. Please see full prescribing information for Soliris, including boxed WARNING regarding risk of serious meningococcal infection.

About Alexion

Alexion is a biopharmaceutical company focused on serving patients with severe and rare disorders through the innovation, development and commercialization 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 nearly 50 countries for the treatment of PNH, and in the United States, European Union, Japan and other countries for the treatment of aHUS. Alexion is evaluating other potential indications for Soliris in additional severe and ultra-rare disorders beyond PNH and aHUS, and is developing other highly innovative biotechnology product candidates across multiple therapeutic areas. This press release and further information about Alexion can be found at: www.alexionpharma.com.

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Safe Harbor Statement

This news release contains forward-looking statements, including statements related to potential health and medical benefits of Soliris® (eculizumab) for the treatment of patients with aHUS, pricing for Soliris in England, whether eculizumab will be nationally commissioned in England and the timing of such commissioning, and the continuation of existing programs in England that provide access to Soliris. 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 reimbursement of Soliris, 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 Annual Report on Form 10-K for the period ended Dec. 31, 2013. 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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