2007-04-27

CHMP Adopts Positive Opinion for Alexion's Soliris(TM) in Europe


Cheshire, Connecticut (ots/PRNewswire) -

- CHMP recommends marketing authorization for Soliris(TM)(eculizumab) to treat all patients with paroxysmal nocturnalhemoglobinuria (PNH) -

- First medicinal product to receive positive opinion from CHMPunder Accelerated Assessment Procedure -

Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced thatthe Committee for Human Medicinal Products (CHMP) of the EuropeanMedicines Agency (EMEA) adopted a positive opinion recommendingmarketing authorization for Soliris(TM) (eculizumab) for thetreatment of patients with paroxysmal nocturnal hemoglobinuria (PNH).Based upon the CHMP's positive recommendation, marketingauthorization by the European Commission is expected in two to threemonths.

Soliris, a designated orphan medicinal product, is intended toreduce hemolysis (destruction of red blood cells) in patients withparoxysmal nocturnal hemoglobinuria (PNH). The CHMP recommended abroad label for Soliris covering all patients with PNH. According tothe adopted CHMP opinion, "Soliris (eculizumab) is indicated for thetreatment of patients with paroxysmal nocturnal haemoglobinuria(PNH)." An additional sentence describing the PNH patient populationthat was studied in the Phase III clinical trials states, "Evidenceof clinical benefit of Soliris in the treatment of patients with PNHis limited to patients with history of transfusions." Soliris waspreviously approved by the U.S. Food and Drug Administration on March16, 2007 for the treatment of PNH to reduce hemolysis, and iscurrently being marketed in the United States.

The CHMP evaluated Soliris under the European AcceleratedAssessment procedure, the fastest evaluation timeframe for fullapproval awarded by EMEA. According to the EMEA, Soliris is the firstmedicinal product to receive a positive CHMP opinion within theAccelerated Assessment procedure.

There are no therapies specifically available for the treatment ofPNH in Europe. PNH is a rare, disabling and life-threatening blooddisorder defined by chronic red blood cell destruction, or hemolysis.Hemolysis can cause one or more of the following symptoms in patientswith PNH: severe anemia, disabling fatigue, recurrent pain, shortnessof breath, pulmonary hypertension, intermittent episodes of darkcolored urine (hemoglobinuria), kidney disease, impaired quality oflife and blood clots (thromboses).

"Soliris brings real hope to people who live daily with thedevastating effects of PNH," said Leonard Bell, MD, chief executiveofficer of Alexion Pharmaceuticals. "The positive opinion adopted byCHMP is an important step in Alexion's mission to improve PNHpatients' lives in Europe and around the globe. Importantly, theadopted CHMP opinion recommends marketing authorization for Soliristo treat all patients with PNH."

CHMP based its opinion on clinical data from three multi-nationalclinical studies involving 195 patients. In these studies Solirisreduced hemolysis in every treated patient, thereby reducingsymptoms, stabilizing hemoglobin and significantly reducingtransfusions. Soliris patients reported markedly less fatigue andimproved health-related quality of life. Additionally, there werefewer thrombotic events during Soliris treatment than during the sameperiod of time prior to treatment.

A summary of the CHMP opinion can be accessed athttp://www.emea.europa.eu .

Important Safety Information

Soliris is generally well tolerated. The most frequent adverseevents observed in clinical studies were headache, nasopharyngitis (arunny nose), back pain and nausea. Treatment with Soliris should notalter anticoagulant management because the effect of withdrawal ofanticoagulant therapy during Soliris treatment has not beenestablished.

In the United States, the product label for Soliris also includesa boxed warning: "Soliris increases the risk of meningococcalinfections. Vaccinate patients with a meningococcal vaccine at least2 weeks prior to receiving the first dose of Soliris; revaccinateaccording to current medical guidelines for vaccine use. Monitorpatients for early signs of meningococcal infections, evaluateimmediately if infection is suspected, and treat with antibiotics ifnecessary." Two out of 196 vaccinated PNH patients treated withSoliris experienced a serious meningococcal infection.

Prior to beginning Soliris therapy, all patients and theirprescribing physicians in the United States, and following approvalthose in Europe as well, will be enrolled in the Soliris SafetyRegistry which is part of a special risk management program thatinvolves initial and continuing education and long-term monitoringfor detection of new safety findings.

Please see full prescribing information for Soliris in the UnitedStates at http://www.soliris.net.

About PNH

PNH is an acquired genetic blood disorder defined by hemolysis, inwhich patients' red blood cells are destroyed by complement, acomponent of the body's immune system. PNH is a rare disease thataffects an estimated 8,000 to 10,000 people in Europe and NorthAmerica. Approximately ten percent of all patients first developsymptoms at 21 years of age or younger. PNH develops without warningand can occur in men and women of all races, backgrounds and ages.PNH often goes unrecognized, with delays in diagnosis often rangingfrom one to more than 10 years. PNH has been identified more commonlyamong patients with diseases of the bone marrow, including aplasticanemia (AA) and myelodysplastic syndrome (MDS). In patients withthromboses of unknown origin, PNH may be an underlying cause.

Prior to approval of Soliris, there were no therapies specificallyavailable for the treatment of PNH. PNH treatment was limited tosymptom management through periodic blood transfusions, non-specificimmunosuppressive therapy and, infrequently, bone marrowtransplantations - a high-risk and painful procedure used as a lastresort.

About Alexion

Alexion Pharmaceuticals is a biotechnology company working todevelop and deliver life-changing drug therapies for patients withserious and life-threatening medical conditions. Alexion marketsSoliris(TM)(eculizumab) in the United States for the treatment ofparoxysmal nocturnal hemoglobinuria (PNH). Alexion is engaged in thediscovery and development of therapeutic products aimed at treatingpatients with severe disease states, including hematologic diseases,cancer and autoimmune disorders. Alexion applied for marketingauthorization with the European Medicines Evaluation Agency (EMEA)for Soliris in September 2006, and in April, 2007 the Committee forHuman Medicinal Products of the EMEA adopted a positive opinionrecommending marketing authorization for Soliris for the treatment ofPNH. This press release and further information about AlexionPharmaceuticals, Inc. can be found at: http://www.alexionpharm.com.

This news release contains forward-looking statements, includingstatements related to the timing and anticipated results ofregulatory authorities' decisions with respect to marketingapplications for Soliris, potential benefits and commercial potentialof Soliris, and estimates of the number of PNH patients.Forward-looking statements are subject to factors that may causeAlexion's results and plans to differ from those expected, includingrequests by regulatory authorities for additional information ordata, timing and evaluation by regulatory agencies of ourapplications, the need for additional research and testing, decisionof regulatory authorities not to approve (or to materially limit)marketing of Soliris, delays in arranging satisfactory manufacturingcapability, inability to acquire funding on timely and satisfactoryterms, delays in developing or adverse changes in commercialrelationships, the possibility that results of clinical trials arenot predictive of the safety and efficacy of Soliris, the risk thatthird parties won't agree to license any necessary intellectualproperty to us on reasonable terms, the risk that third party payorswill not reimburse for the use of Soliris at acceptable rates or atall, the risk that estimates regarding the number of PNH patients areinaccurate and a variety of other risks set forth from time to timein Alexion's filings with the Securities and Exchange Commission,including but not limited to the risks discussed in Alexion's AnnualReport on Form 10-K for the year ended December 31, 2006, and in ourother filings with the Securities and Exchange Commission. Alexiondoes not intend to update any of these forward- looking statements toreflect events or circumstances after the date hereof, except when aduty arises under law.

Web site: http://www.alexionpharm.com http://www.soliris.net http://www.emea.europa.eu

ots Originaltext: Alexion Pharmaceuticals, Inc.Im Internet recherchierbar: http://www.presseportal.de

Contact:Leonard Bell, M.D., Chief Executive Officer of Alexion Pharmaceuticals, Inc., +1-203-272-2596; or Media, David Patti, Makovsky + Company, +1-212-508-9623, or dpatti@makovsky.com; or Investors, Rhonda Chiger of Rx Communications Group, LLC, +1-917-322-2569, or rchiger@rxir.com

Alexion Pharmaceuticals, Inc.

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