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Alexion’s Soliris® (eculizumab) Receives Positive Opinion from the Committee for Orphan Medicinal Products for Treatment of

  Alexion’s Soliris® (eculizumab) Receives Positive Opinion from the Committee
  for Orphan Medicinal Products for Treatment of Neuromyelitis Optica (NMO)

Business Wire

CHESHIRE, Conn. -- July 16, 2013

Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced that Soliris^®
(eculizumab), the company’s first-in-class terminal complement inhibitor, has
received a positive opinion for orphan medicinal product designation from the
Committee for Orphan Medicinal Products (COMP) of the European Medicines
Agency (EMA) for the treatment of neuromyelitis optica (NMO), a
life-threatening, ultra-rare neurological disorder. The positive opinion of
the COMP has now been forwarded to the European Commission for final approval
and publication in the community register. Soliris is not approved in any
country for the treatment of patients with NMO.

“We are pleased that the COMP has provided a positive opinion regarding orphan
medicinal product status for Soliris for the treatment of patients with NMO,
particularly after having been recently granted orphan-drug designation by the
FDA,” said Martin Mackay, Ph.D., Executive Vice President and Global Head of
R&D at Alexion. “NMO is an extremely rare, chronic and debilitating disease
that can lead to paralysis, blindness and death, and there are no approved
therapies for it. Therefore, this news represents another positive step toward
meeting the needs of this underserved patient population in the EU.”

The European Commission grants orphan medicinal product status to provide
incentives to develop medicinal products to treat, prevent or diagnose
diseases or conditions that affect no more than five in 10,000 persons in the
EU. The orphan medicinal product status designation would provide Alexion with
certain benefits and incentives, including a period of marketing exclusivity.

About NMO

In patients with NMO, uncontrolled complement activation causes destruction of
myelin-producing cells, leading to severe damage to the central nervous system
(CNS), including the spinal cord and optic nerve.^1-3 The disease leads to
severe weakness, paralysis, respiratory failure, loss of bowel and bladder
function, blindness and premature death.^4-6 Patients with NMO have a
life-long exposure to the uncontrolled complement activation due to chronic
autoimmune attack, and most patients experience an unpredictable, relapsing
course of disease with cumulative disability, as each attack adds to the
neurologic disability.^5,7,8 Fifty percent of relapsing NMO patients have been
reported to sustain permanent severe disability, including paralysis and
blindness, within five years of disease onset.^9 Most NMO-related deaths
result from respiratory complications from NMO attacks.^9,10 The disease
primarily affects women, with a female to male ratio as high as a 9:1.^11

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 United States, European Union (EU) and other
countries as the first and only treatment for aHUS patients. Soliris is
indicated to inhibit complement-mediated TMA. Soliris is not indicated for the
treatment of patients with Shiga toxin E. coli-related hemolytic uremic
syndrome (STEC-HUS). Alexion is evaluating the safety and efficacy of Soliris
for the treatment of patients with STEC-HUS.

Soliris also is approved in the US, EU, 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 indicated to reduce hemolysis.

Alexion's breakthrough approach in terminal complement inhibition has 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 prescribing information on Soliris, is
available at

Important Safety Information

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 appropriate 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 were
headache (occurred mostly in the initial phase), leukopenia and meningococcal
infection. Soliris is not expected to affect the aplastic component of anaemia
in patients with PNH.

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
serving patients with severe and ultra-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 a treatment for patients with PNH and aHUS, two debilitating,
ultra-rare and life-threatening disorders caused by chronic uncontrolled
complement activation. The treatment 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 its marketed drug and is developing four other highly
innovative biotechnology product candidates, which are being investigated
across nine severe and ultra-rare disorders beyond PNH and aHUS.

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 PNH and 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
March 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.


^1 Jarius S, Wildemann B. AQP4 antibodies in neuromyelitis optica: diagnostic
and pathogenetic relevance. Nat Rev Neuro. 2010;6:383-92.

^2 Hinson SR, Romero MF, Popescu BFG, et al. Molecular outcomes of
neuromyelitis optica (NMO)-IgG binding to aquaporin-4 in astrocytes. Proc Nat
Acad Sci 2012;109(4):1245-50.

^3 Hinson SR, Pittock SJ, Lucchinetti CF, et al. Pathogenic potential of IgG
binding to water channel extracellular domain in neuromyelitis optica.
Neurology 2007;69:2221-31.

^4 Wingerchuk DM, Lennon VA, Lucchinetti CF, Pittock SJ, Weinshenker BG. The
spectrum of neuromyelitis optica. Lancet Neuro. 2007;6(9):805-15.

^5 Wingerchuk DM. Diagnosis and treatment of neuromyelitis optica. Neurologis.

^6 Wingerchuk DM, Weinshenker BG. Neuromyelitis optica. Curr Treat Options
Neurol 2008;10(1):55-66.

^7 Tuzun E, Kurtuncu M, Turkoglu R, et al. Enhanced complement consumption in
neuromyelitis optica and Behcet’s disease patients. J Neuroimmunol

^8 Kuroda H, Fujihara K, Takano R, et al. Increase of complement fragment C5a
in cerebrospinal fluid during exacerbation of neuromyelitis optica. J
Neuroimmunol 2013;254(1-2):178-82.

^9 Wingerchuk DM, Hogancamp WF,O’Brien PC, Weinshenker BG. The clinical course
of neuromyelitis optica (Devic’s syndrome). Neurology 1999;53(5):1107-14.

^10 Kitley J, Leite MI, Nakashima I, et al. Prognostic factors and disease
course in aquaporin-4 antibody-positive patients with neuromyelitis optica
spectrum disorder from the United Kingdom and Japan. Brain 2012;135(Pt

^11 Wingerchuk DM. Neuromyelitis optica. Int MS J 2006;13(2):42–50.


Alexion Pharmaceuticals, Inc.
Irving Adler, 203-271-8210
Executive Director, Corporate Communications
SmithSolve Communications, LLC
Jennifer Devine, 973-442-1555 ext.102
Rx Communications
Rhonda Chiger, 917-322-2569
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