Alexion Launches Uncommon Strength Campaign to Unite and Celebrate the Courage and Resilience of People Living with Rare Diseases

– Rare Disease Community Encouraged to Share Customized Rare Disease
Hero Avatars and Update Social Media Profile Pictures to Raise Awareness
of aHUS, PNH, HPP, and LAL-D Around the World –

NEW HAVEN, Conn.–(BUSINESS WIRE)–Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the launch
of Uncommon Strength, a global campaign to raise awareness of
rare diseases through the celebration of the extraordinary resilience
and inner strength of those impacted by these diseases. Uncommon
Strength (www.UncommonStrength.com)
supports global rare disease communities, including those impacted by
atypical hemolytic uremic syndrome (aHUS), paroxysmal nocturnal
hemoglobinuria (PNH), hypophosphatasia (HPP), and lysosomal acid lipase
deficiency (LAL-D) with educational information as well as interactive
social media elements to unite the community.

People living with rare diseases, and their families, must demonstrate
“uncommon strength” as they persevere to find answers about their
diseases. Reaching a diagnosis for a rare disease can be a long and
challenging experience because the conditions are often unknown,
misunderstood, or misdiagnosed. In fact, the average time from a
person’s first symptom to receiving an accurate diagnosis of a rare
disease is nearly five years, during which he or she may visit more than
seven physicians.¹

“At Alexion, we are inspired by patients and families living with rare
diseases and recognize the inner strength and perseverance they must
have to face the ongoing challenges they encounter, such as receiving an
accurate and timely diagnosis and appropriate medical care,” said David
Hallal, Chief Executive Officer of Alexion. “To all of us at Alexion,
patients with rare diseases are true heroes, and it is our hope that the Uncommon
Strength campaign will amplify their voices and generate the much
needed awareness to reach more patients and families who are still
seeking answers.”

People impacted by rare diseases, including patients, caregivers, and
loved ones, are invited to visit www.UncommonStrength.com
to:

• Create a personal aHUS, PNH, HPP, LAL-D, or rare disease “hero avatar”
  to show their unique courage, strength, and perseverance, and support
  the rare disease community.
• Share custom hero avatars over social media or e-mail to help generate
  a broader conversation about rare diseases and inspire people across
  the globe to learn more and get involved in awareness efforts.
• Update their personal Facebook and Twitter profile pictures with a
  customized photo filter to spread the word about aHUS, PNH, HPP,
  LAL-D, or rare diseases in general.

The Uncommon Strength campaign will coincide with upcoming
awareness milestones, including:

• September 24, 2016 – aHUS Awareness Day
• September 26 – October 2, 2016 – PNH Awareness Week in
  the United States
• October 13, 2016 – World Thrombosis Day
• October 24 – 30, 2016 – HPP Awareness Week
• October 2016 – Liver Awareness Month

aHUS, PNH, HPP, and LAL-D are considered ultra-rare diseases, which are
defined as diseases that impact fewer than 20 people per million
population.² The Uncommon Strength campaign aims to
drive disease education, and inspire and connect the rare disease
community to advocate for rare disease awareness locally and globally to
ensure people living with these diseases, and their families, have the
information they need.

In addition to the campaign’s interactive hero avatar builder
application, the Uncommon Strength website features helpful
information about aHUS, PNH, HPP, and LAL-D along with access to key
resources and advocacy organizations around the world where patients and
caregivers can go for support. The following organizations are key
supporters of the Uncommon Strength campaign:

• aHUS
  • aHUS Alliance
  • aHUS UK
  • Atypical HUS Foundation
  • Selbsthilfe für seltene komplementbedingte Erkrankungen MPGN und
    aHUS e.V.
  • Aplastic Anemia and MDS International Foundation (AAMDSIF)
  • Aplastic Anemia & Myelodysplasia Association of Canada (AAMAC)
  • Canadian Association of PNH Patients
  • NPO PNH Club
  • Children Living with Inherited Metabolic Diseases (CLIMB)
  • HypoPhosPhatasia Support Association of Japan (HPPSA-J)
  • Hypophosphatasie Deutschland e.V.
  • International Coalition of Organizations Supporting Endocrine
    Patients (ICOSEP)
  • Soft Bones
  • The MAGIC Foundation
  • LAL Solace
  • AE LALD Spain
  • Latin American Rare Disease
    • Federación Colombiana de Enfermedades Raras (FECOER)
    • Federación Mexicana de Enfermedades Raras (FEMEXER)

    To learn more about aHUS, PNH, HPP, and LAL-D, build your own personal
    rare disease hero avatar, and help spread awareness by getting involved
    in the Uncommon Strength campaign, visit www.UncommonStrength.com.

    About Atypical Hemolytic Uremic Syndrome (aHUS)

    aHUS is a chronic, ultra-rare, and life-threatening disease in which a
    life-long and permanent 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.^3,4 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.^3,5 Seventy-nine
    percent of all patients with aHUS die, require kidney dialysis or have
    permanent kidney damage within three years after diagnosis despite
    plasma exchange or plasma infusion (PE/PI).⁶ Moreover, 33-40
    percent of patients die or progress to end-stage renal disease with the
    first clinical manifestation of aHUS despite PE/PI.^6,7 The
    majority of patients with aHUS who receive a kidney transplant commonly
    experience subsequent systemic TMA, resulting in a 90 percent transplant
    failure rate in these TMA patients.⁸

    aHUS affects both children and adults. Complement-mediated TMA also
    causes reduction in platelet count (thrombocytopenia) and red blood cell
    destruction (hemolysis). While mutations have been identified in at
    least ten different complement regulatory genes, mutations are not
    identified in 30-50 percent of patients with a confirmed diagnosis of
    aHUS.⁸

    About Paroxysmal Nocturnal Hemoglobinuria (PNH)

    PNH is an ultra-rare blood disorder in which chronic, uncontrolled
    activation of complement, a component of the normal immune system,
    results in hemolysis (destruction of the patient’s red blood cells). PNH
    strikes people of all ages, with an average age of onset in the early
    30s.⁹ Approximately 10 percent of all patients first develop
    symptoms at 21 years of age or younger.¹⁰ PNH develops
    without warning and can occur in men and women of all races, backgrounds
    and ages. PNH often goes unrecognized, with delays in diagnosis ranging
    from one to more than 10 years.¹¹ In the period of time
    before treatment was available, it had been estimated that approximately
    one-third of patients with PNH did not survive more than 5 years from
    the time of diagnosis.⁹ PNH has been identified more commonly
    among patients with disorders of the bone marrow, including aplastic
    anemia (AA) and myelodysplastic syndromes (MDS).^12-14 In
    patients with thrombosis of unknown origin, PNH may be an underlying
    cause.⁹

    About Hypophosphatasia (HPP)

    HPP is a genetic, chronic, progressive, and life-threatening ultra-rare
    metabolic disease characterized by low alkaline phosphatase (ALP)
    activity and defective bone mineralization that can lead to destruction
    and deformity of bones and other skeletal abnormalities, as well as
    systemic complications such as profound muscle weakness, seizures, pain,
    and respiratory failure leading to premature death in infants.^15-19

    HPP is caused by mutations in the gene encoding an enzyme known as
    tissue non-specific alkaline phosphatase (TNSALP).^15,16 The
    genetic deficiency in HPP can affect people of all ages.¹⁵ HPP
    is traditionally classified by the age of the patient at the onset of
    symptoms of the disease. Symptoms can manifest in infants, children or
    adults.

    HPP can have devastating consequences for patients at any stage of life.¹⁵ In
    a natural history study, infants who had their first symptom of HPP
    within the first 6 months of life had high mortality, with an overall
    mortality rate of 73 percent at 5 years.²⁰ In these patients,
    mortality is primarily due to respiratory failure.^15,19,21 Regardless
    of age of symptom onset, long-term clinical consequences include impact
    on growth and development, difficulties with activities of everyday
    living (standing, climbing stairs, etc.), recurrent and non-healing
    fractures, profound muscle weakness, debilitating pain and the
    requirement for ambulatory assistive devices such as wheelchairs,
    wheeled walkers and canes.^15,18

    About Lysosomal Acid Lipase Deficiency (LAL-D)

    LAL-D is a genetic, chronic, and progressive ultra-rare metabolic
    disease associated with significant morbidity and premature mortality.²² In
    patients with LAL-D, genetic mutations result in a marked decrease or
    loss in activity of the vital LAL enzyme. This leads to marked
    accumulation of cholesteryl esters and triglycerides in vital organs,
    blood vessels, and other tissues, resulting in progressive and
    multi-organ damage including fibrosis, cirrhosis, liver failure,
    accelerated atherosclerosis, cardiovascular disease, and other
    devastating consequences.^22,23

    LAL-D affects patients of all ages with clinical manifestations from
    infancy through adulthood and may have sudden and unpredictable clinical
    complications. Infants experience profound growth failure, liver
    fibrosis, and cirrhosis, with a median age of death at 3.7 months.²⁴ In
    an observational study, approximately 50 percent of children and adults
    with LAL-D progressed to fibrosis, cirrhosis, or liver transplant in 3
    years.²⁵ The median age of onset of LAL-D is 5.8 years, and
    the disease can be diagnosed with a simple blood test.^26,27

    About Alexion

    Alexion is a global biopharmaceutical company focused on developing and
    delivering life-transforming therapies for patients with devastating and
    rare disorders. Alexion is the global leader in complement inhibition
    and has developed and commercializes the first and only approved
    complement inhibitor to treat patients with paroxysmal nocturnal
    hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two
    life-threatening ultra-rare disorders. In addition, Alexion’s metabolic
    franchise includes two highly innovative enzyme replacement therapies
    for patients with life-threatening and ultra-rare disorders,
    hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D).
    Alexion is advancing the most robust rare disease pipeline in the
    biotech industry with highly innovative product candidates in multiple
    therapeutic areas. This press release and further information about
    Alexion can be found at: www.alexion.com.

    [ALXN-G]

    References

    1.			Engel PA, Bagal S, Broback M, Boice N. Physician and patient percepts regarding physician training in rare diseases: the need for stronger educational initiatives for physicians. Journal of Rare Diseases. 2013;1(2):1-15.
    2.			REGULATION (EU) No 536/2014 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 16 April 2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC. http://eur-lex.europa.eu/legal-content/ EN/TXT/PDF/?uri=CELEX:32000R0141&qid=1421232987002&from=EN.
    3.			Benz K, Amann K. Thrombotic microangiopathy: new insights. Curr Opin Nephrol Hypertens. 2010;19(3):242-7.
    4.			Ariceta G, Besbas N, Johnson S, et al. Guideline for the investigation and initial therapy of diarrhea-negative hemolytic uremic syndrome. Pediatr Nephrol. 2009;24:687-96.
    5.			Tsai HM. The molecular biology of thrombotic microangiopathy. Kidney Int. 2006;70(1):16-23.
    6.			Noris M, Remuzzi G. Atypical hemolytic-uremic syndrome. N Engl J Med. 2009;361:1676-87.
    7.			Noris M, Caprioli J, Bresin E, et al. Relative role of genetic complement abnormalities in sporadic and familial aHUS and their impact on clinical phenotype. Clin J Am Soc Nephrol. 2010;5:1844-59.
    8.			Bresin E, Daina E, Noris M, et al. Outcome of renal transplantation in patients with non-Shiga toxin-associated hemolytic uremic syndrome: prognostic significance of genetic background. Clin J Am Soc Nephrol. 2006;1:88-99.
    9.			Socie G, Mary JY, de Gramont A., Rio B, Leporrier M, Rose C, et al. Paroxysmal nocturnal haemoglobinuria: long-term follow-up and prognostic factors. French Society of Haematology. Lancet 1996 Aug 31;348(9027):573-7.
    10.			Parker C, Omine M, Richards S, et al. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood. 2005;106(12):3699-3709.
    11.			Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med. 1995;333(19):1253-1258.
    12.			Wang H, Chuhjo T, Yasue S, Omine M, Naka S. Clinical significance of a minor population of paroxysmal nocturnal hemoglobinuria-type cells in bone marrow failure syndrome. Blood. 2002;100 (12):3897-3902.
    13.			Iwanga M, Furukawa K, Amenomori T, et al. Paroxysmal nocturnal haemoglobinuria clones in patients with myelodysplastic syndromes. Br J Haematol. 1998;102(2):465-474.
    14.			Maciejewski JP, Rivera C, Kook H, Dunn D, Young NS. Relationship between bone marrow failure syndromes and the presence of glycophosphatidyl inositol-anchored protein-deficient clones. Br J Haematol. 2001;115:1015-1022.
    15.			Rockman-Greenberg C. Hypophosphatasia. Pediatr Endocrinol Rev. 2013; 10(2):380-388.
    16.			Whyte MP. Hypophosphatasia: nature’s window on alkaline phosphatase function in humans. In: Bilezikian JP, Raisz LG, Martin TJ, eds. Principles of Bone Biology. Vol 1. 3rd ed. San Diego, CA: Academic Press; 2008:1573-1598.
    17.			Whyte MP, Greenberg CR, Salman N, et al. Enzyme-replacement therapy in life-threatening hypophosphatasia. N Engl J Med.2012; 366(10):904-913.
    18.			Seshia SS, Derbyshire G, Haworth JC, Hoogstraten J. Myopathy with hypophosphatasia. Arch Dis Child. 1990; 65(1):130-131.
    19.			Baumgartner-Sigl S, Haberlandt E, Mumm S, et al. Pyridoxine-responsive seizures as the first symptom of infantile hypophosphatasia caused by two novel missense mutations (c.677T>C, p.M226T; c.1112C>T, p.T371I) of the tissue-nonspecific alkaline phosphatase gene. Bone. 2007; 40(6):1655-1661.
    20.			Whyte MP, Leung E, Wilcox W, et al. Hypophosphatasia: a retrospective natural history study of the severe perinatal and infantile forms. Poster presented at the 2014 Pediatric Academic Societies and Asian Society for Pediatric Research Joint Meeting, Vancouver, B.C., Canada, May 5, 2014. Abstract 752416.
    21.			Whyte MP, Rockman-Greenberg C, Hofmann C, et al. Improved survival with asfotase alfa treatment in pediatric patients with hypophosphatasia at high risk of death. Poster presented at the American Society for Bone and Mineral Research (ASBMR) 2014 Annual Meeting, Houston, September 14, 2014. Abstract 1097.
    22.			Bernstein DL, et al. Chloesteryl ester storage disease: review of the findings in 135 reported patients with an underdiagnosed disease. J Hepatol. 2013;58:1230-43.
    23.			Reiner Z, et al. Lysosomal acid lipase deficiency – an under-recognized cause of dyslipidemia and liver dysfunction. Atherosclerosis. 2014;235:21-30.
    24.			Jones SA et al. Rapid progression and mortality of lysosomal acid lipase deficiency presenting in infants. Genet Med. 27 August 2015. doi:10.1038/gim.2015.108.
    25.			Data on file, Alexion.
    26.			Burton et al. Clinical Features of Lysosomal Acid Lipase Deficiency – a Longitudinal Assessment of 48 Children and Adults. Journal of Pediatric Gastroenterology & Nutrition. 2015.
    27.			Hamilton J, et al. A new method for the measurement of lysosomal acid lipase in dried blood spots using the inhibitor Lalistat 2. Clin Chim Acta. 2012;413:1207-10. doi:10.1016/j.cca.2012.03.019.

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    Alexion Pharmaceuticals, Inc.
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    475-230-3777
    Senior Vice President, Corporate Communications
    or
    Emily
    Vlasak, 475-230-3782
    Associate Director, Corporate Communications
    or
    Investors
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    Ridloff, CFA, 475-230-3601
    Vice President, Investor Relations