Every Day Is An Inspiration 2018 ANNUAL REPORT RACHEL LIVING WITH gMG AIRA LIVING WITH HPP RUTHIE AND HER SON LIVING WITH PNH MAUREEN (FAR LEFT) AND LAUREN (FAR RIGHT) WITH THEIR PARENTS AND BROTHER ETHAN LIVING WITH LAL-D LIVING WITH aHUS Every Day Builds A Tomorrow People with rare and devastating diseases are our inspiration and our Guiding Star. While their diseases are rare, their special life moments should not be. Together, we help patients and their families live their lives fully so they can celebrate the moments that matter most. p.1 p.2 With your support, we are achieving our mission to transform the lives of people affected by rare TO OUR SHAREHOLDERS and devastating diseases by developing and delivering valued innovative therapies. t’s a privilege to report on a year of tremendous progress for Alexion and, • Double-digit underlying volume growth in our base business, serving most importantly, for the patients we serve through our unwavering more patients than ever before with paroxysmal nocturnal hemoglobinuria mission to transform the lives of people living with rare and devastating (PNH), atypical hemolytic uremic syndrome (aHUS), gMG, Idiseases by developing and delivering valued innovative therapies. hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D), exceeding our financial ambitions; and 2018 was a year of transformation for Alexion. When I joined Alexion in 2017, I saw a remarkable opportunity to create more value for patients and • A new Boston headquarters with a strong talent base and a culture where shareholders. We outlined ambitious goals for 2018, which we exceeded, and employees feel empowered, valued and safe to speak up, and where new in the majority of cases far surpassed, through the hard work of our more and diverse ideas are welcome. than 3,000 employees across the globe. In doing so, we built a foundation, We know we still have work to do, as only a small fraction of the more than with integrity and compliance at the center, that positions us to continue 7,000 identified rare diseases have approved therapies. With 30 million our momentum in 2019 and beyond. patients diagnosed with rare diseases in the U.S. alone, there is a clear unmet As I reflect upon the past year, I am proud of the dedication and focus need. With a growing portfolio and product pipeline, we are fully committed of our teams, which helped deliver: to advancing our mission of serving patients who suffer from rare and devastating diseases. • Extension of our complement leadership with ULTOMIRIS® (ravulizumab-cwvz) following positive Phase 3 results comprising the Our strong foundation positions us well to build four durable, potential largest-ever PNH clinical trial program, regulatory filings, and approval blockbuster franchises in hematology and nephrology, metabolics, for the treatment of patients with PNH in the United States; neurology and FcRn. We will achieve these goals by establishing ULTOMIRIS as the new standard of care for PNH, growing our neurology and metabolic • The best Alexion launch to date with SOLIRIS® (eculizumab) in generalized portfolios and continuing to expand our pipeline to drive long-term, myasthenia gravis (gMG), a chronic and debilitating neuromuscular sustainable shareholder value. disorder, bringing a much-needed therapy to patients who had not seen a new treatment option in more than 60 years; Every day we have a clear purpose because people living with rare diseases are waiting. The dedication of our employees enables us to stay focused on • A robust and diverse pipeline, resulting from disciplined and successful what matters most – delivering for patients. We are motivated and energized business development that is helping to drive the change in Alexion’s because we know lives are at stake. With a strong foundation in place, focus from ultra-rare to rare diseases with the addition of two clinical we will continue to push ourselves to redefine what it means to live with stage assets, including one for Wilson disease and one for the neonatal a rare disease. Fc receptor (FcRn), as well as two pre-clinical collaborations; Thank you to the patients and their families who inspire us, to our • Remarkable results from our Phase 3 study for SOLIRIS in neuromyelitis employees for their passion and drive and to you, our shareholders, for optica spectrum disorder (NMOSD), a rare, devastating, complement- supporting our mission. mediated disorder of the central nervous system characterized by relapses. Each relapse results in stepwise accumulation of disability, including Sincerely, blindness and paralysis, and sometimes premature death. In this pivotal trial, 98% of patients receiving SOLIRIS were relapse free at 48 weeks. In late 2018, we filed for regulatory approval in the U.S. and subsequently received priority review. We are hopeful for a positive decision later this year, bringing the Ludwig Hantson, Ph.D. first-approved therapy to patients suffering from this debilitating disease; Chief Executive Officer p.3 p.4 Every Day A commitment to compliance... Quality, ethics, integrity and compliance play a central role in our success. We all Strengthens Our share responsibility for building trust with patients, customers, shareholders and Foundation the communities where we live and work. QUALITY IS FOR EVERYONE We are committed to: • Operating a Quality Manage- ment System and improving • Delivering safe and effective our systems and processes therapies that meet or exceed the requirements of our • Ensuring the integrity of patients and our customers our data • Complying with all applicable • Upholding our individual regulatory requirements and collective accountability for quality Serve Patients Act with Integrity We put patients at the heart We embrace compliance and of decisions we make quality as essential to every- thing we do We seek to understand …while living our values. patients’ journeys and with We build trust and earn our passion and persistence, credibility by doing things help each patient find answers the right way and deliver transformative therapies that change lives We are all leaders and accountable to each other, We collaborate and partner patients, customers, stakehold- with the world around us to ers and shareholders create value for patients Empower People Innovate for Solutions We trust, value, respect and We innovate with discipline and empower team members focus to seek creative solutions in all aspects of our work We unlock value through diversity of people, ideas, We take balanced risks and cross-functional collaboration encourage experimentation and continuous learning We actively listen with humility We celebrate success and to diverse internal and external embrace failure as an opportunity inputs, and harness new ideas to learn and improve p.5 p.6 Dan LIVING WITH PNH Dan recalls experiencing symptoms of PNH as early as when he was a young teenager. He would bruise easily and fatigue was something he seemingly always lived with. After a period of rapid weight loss when he was 19, and at the insistence of a cousin who was a doctor, Dan consulted a hematologist. He was told based on his blood test results that he likely only had six months to a year to live. Dan remembers thinking he was young, invulnerable, invincible; how could he be that sick? Fortunately, Dan surpassed those odds, persevering despite years of countless blood tests, bone marrow biopsies, blood transfusions and misdiagnoses. Finally, at age 30, Dan was diagnosed with PNH and was able to receive treatment with SOLIRIS and get his disease under control. Once I finally received a diagnosis and worked with my doctor, it was the first time that I felt my health and life could become stable. DAN I appreciate all of the little things like taking Justice to the store, or taking him to the park, or going to parades. Every day is a very big blessing for us. LIVING WITH PNH DANIELLE, JUSTICE’S MOM When Justice was nine-months old, he woke one morning with facial swelling. His parents brought him to Urgent Care where he was treated for pink eye and sent home. Later that day, his body became very limp and his eyes could not focus. His parents rushed him to the local hospital, by which point Justice had begun to have a seizure. Doctors at the hospital were unable to stop his seizure and he was transferred to a specialist children’s hospital. Once there, the medical team discovered that Justice was experiencing kidney failure. He was intubated and started on dialysis, followed by plasmapheresis, while doctors tried to determine a diagnosis. Several days later, Justice was diagnosed with aHUS. His parents were devastated at first, but Justice’s doctor explained that his aHUS could be managed with SOLIRIS. Today, Justice is an energetic six-year old with a vibrant personality, bringing joy to everyone around him. Justice LIVING WITH a HUS p.9 p.10 2018 was a year focused on strengthening our foundation, establishing a culture of Every Day high performance, with integrity and compliance at the center, and refocusing our We Deliver On strategy to drive Alexion into its next chapter. The foundation for 2019 is solid. We have a strong culture, clear business objectives, strong leadership, amazing teams Our Mission and a promising pipeline. We have a clear purpose. People living with rare diseases are waiting. We are inspired and energized by our work together because we know there still is so much yet to do. Grew EXTENDED 4 IN-LINE BUSINESS TRANSFORMATIVE THERAPIES ACROSS COMPLEMENT 5 LEADERSHIP RARE DISEASES WITH IN OUR GROWING ® PORTFOLIO ULTOMIRIS DELIVERED ON FINANCIAL AMBITIONS TRISTAN AND HIS MOM LIVING WITH LAL-D SOLIRIS® IN gMG MOST SUCCESSFUL U.S. LAUNCH IN ADVANCED ALEXION HISTORY AND REBUILT ~800 OUR PIPELINE PATIENTS ON THERAPY ROBERTA AND HER PARTNER LIVING WITH gMG p.11 p.12 Jesse LIVING WITH gMG I feel like tomorrow I can do something, whereas before, I didn’t feel like tomorrow was possible.