Alexion’s Soliris® (eculizumab) Receives Orphan Drug Designation for the Treatment of Neuromyelitis Optica (NMO)

  Alexion’s Soliris® (eculizumab) Receives Orphan Drug Designation for the
  Treatment of Neuromyelitis Optica (NMO)

Business Wire

CHESHIRE, Conn. -- June 27, 2013

Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced that Soliris^®
(eculizumab), the company’s first-in-class terminal complement inhibitor, has
been granted an orphan drug designation by the U.S. Food and Drug
Administration (FDA) for the treatment of neuromyelitis optica (NMO), a
life-threatening, ultra-rare neurological disorder. In a Phase 2 study
presented at the 2012 annual meeting of the American Neurological Association
(ANA), Soliris treatment was associated with a significant reduction in the
frequency of relapses (recurring attacks) in patients with severe, relapsing
NMO.^1 Soliris is not approved for the treatment of patients with NMO.

The FDA, through its Office of Orphan Products Development (OOPD), grants
orphan status to drugs and biologic products that are intended for the safe
and effective treatment, diagnosis, or prevention of rare diseases or
disorders that affect fewer than 200,000 people in the U.S. Orphan drug
designation provides a drug developer with certain benefits and incentives,
including a period of marketing exclusivity if regulatory approval is
ultimately received for the designated indication.

“There are no approved therapies for patients with NMO, an extremely rare and
debilitating disease that can lead to paralysis, blindness and death,” said
Martin Mackay, Ph.D., executive vice president and global head of R&D at
Alexion. “This orphan drug designation is a positive step toward understanding
and meeting the needs of this underserved patient population. In clinical
trials to date, terminal complement inhibition with Soliris appeared to
significantly reduce the attack rate in patients with severe, refractory
relapsing NMO.”

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.^2-4 The disease leads to
severe weakness, paralysis, respiratory failure, loss of bowel and bladder
function, blindness and premature death.^5-7 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.^6,8,9 Fifty percent of relapsing NMO patients have been
reported to sustain permanent severe disability, including paralysis and
blindness, within five years of disease onset.^10 Most NMO-related deaths
result from respiratory complications from NMO attacks.^10,11 The disease
primarily affects women, with a female to male ratio as high as a 9:1.^12

Phase 2 Data in Patients with NMO

The Phase 2 study reported at the ANA 2012 annual meeting and subsequently
published in The Lancet Neurology^13 was a single-arm, open-label,
investigator-initiated trial in 14 women with severe, relapsing NMO who were
treated for one year. The study met its primary efficacy endpoint, reduction
in annualized relapse rate, with high degrees of clinical and statistical
significance: a decline in the median annualized attack rate from 3.0 attacks
per year pre-Soliris treatment to 0 attacks per year during 12 months of
chronic Soliris treatment (p<0.0001). After 12 months of treatment, 86% (12 of
14) of these severely affected patients were completely attack-free.^1

Additionally, Soliris was associated with significant improvements in key
secondary endpoints. The median expanded disability status scale (EDSS) score
improved from 4.3 pre-treatment to 3.5 after 12 months of treatment with
Soliris (p<0.01). Importantly, all patients experienced either improvement or
stability in all key outcome measures, including EDSS, ambulatory function as
measured by the Hauser Ambulation Index, and visual function as measured by
visual acuity. Soliris was generally well-tolerated, with the three most
common adverse events being headache, nausea, and dizziness. ^ One case of
meningococcal sepsis occurred. The patient made an uneventful recovery and
restarted treatment with eculizumab to complete the study.^1

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., E.U., 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 U.S., E.U., and other countries as the
first and only treatment for patients with atypical hemolytic uremic syndrome
(aHUS), a genetic, life-threatening, ultra-rare disease characterized by
complement-mediated thrombotic microangiopathy (TMA, the formation of blood
clots in small vessels).

Soliris is not approved for the treatment of NMO in any country. 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 NMO, STEC-HUS, and
other complement-mediated diseases.

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

Soliris is generally well tolerated in patients with PNH and aHUS. In patients
with PNH, the most frequently reported adverse events observed with Soliris
treatment in clinical studies were headache, nasopharyngitis (runny nose), and
back pain. 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, and diarrhea.

The U.S. product label for Soliris also 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 2 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 (5.2). Enrollment in the Soliris REMS program and
additional information are available by telephone: 1-888-soliris

Please see full prescribing information for Soliris, including boxed WARNING
regarding risk of serious meningococcal infection.

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. This press
release and further information about Alexion Pharmaceuticals, Inc. can be
found at:


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 Pittock SJ, McKeon A, Mandrekar JN, Weinshenker BG, Lucchinetti CF,
Wingerchuk DM. Pilot clinical trial of eculizumab in AQP4-Ig-G-positive NMO.
Presented at the 2012 annual meeting of the American Neurological Association,
Boston, MA, October 9, 2012: Abstract T1826.

^2 Jarius S, Wildemann B. AQP4 antibodies in neuromyelitis optica: diagnostic
and pathogenetic relevance. Nat Rev Neuro. 2010;6:383-92.

^3 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.

^4 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.

^5 Wingerchuk DM, Lennon VA, Lucchinetti CF, Pittock SJ, Weinshenker BG. The
spectrum of neuromyelitis optica. Lancet Neuro. 2007;6(9):805-15.

^6 Wingerchuk DM. Diagnosis and treatment of neuromyelitis optica. Neurologis.

^7 Wingerchuk DM, Weinshenker BG. Neuromyelitis optica. Curr Treat Options
Neurol 2008;10(1):55-66.

^8 Tuzun E, Kurtuncu M, Turkoglu R, et al. Enhanced complement consumption in
neuromyelitis optica and Behcet’s disease patients. J Neuroimmunol

^9 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.

^10 Wingerchuk DM, Hogancamp WF,O’Brien PC, Weinshenker BG. The clinical
course of neuromyelitis optica (Devic’s syndrome). Neurology

^11 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

^12 Wingerchuk DM. Neuromyelitis optica. Int MS J 2006;13(2):42–50.

^13 Pittock SJ, Lennon VA, McKeon A, et al. Eculizumab in AQP4-Ig-G-positive
relapsing neuromyelitis optica spectrum disorders: an open-label pilot study.
Lancet Neurol 2013;12(6):554-62.


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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