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At Shire, we are champions for people around the world who are struggling with rare diseases – they are at the center of everything we do.

Focusing on a Significant Unmet Medical Need

Rare diseases, most of which are genetic and are present throughout a person's entire life, pose a significant medical and economic burden for patients, communities and healthcare systems. There are more than 7,000 known rare diseases, including Hemophilia, Hereditary Angioedema (HAE), Gaucher disease, Hunter Syndrome, and many forms of rare cancer, and an estimated 350 million people worldwide are living with one.1

These figures do not reflect the untold number of family members and friends who watch a loved one struggle with health challenges that, in many cases, cannot be adequately addressed today, especially considering nearly half of the time these loved ones are children. What's more, patients with rare diseases commonly experience delays to diagnosis, which can lead to serious consequences for their health and the wider healthcare system.

Innovation and Collaboration Will Lead to Breakthrough Progress

This unmet need drives our unique model for innovation, which is a mix of internal knowledge, capabilities and research, combined with collaborations with external partners, patients, doctors, caregivers and advocacy groups, and supplemented by M&A and licensing.

As we look to the future, we are sharply focused on developing new medicines in our chosen therapeutic areas that deliver value and progress for all impacted. We are also committed to working alongside governments and payers to deliver meaningful outcomes that help ease the long-term burden of these diseases for patients and all who support them on their journey.


  1. Global Genes Factsheet on Rare Diseases:
Type Public / Private


Today, Shire is the leading global biotechnology company focused on serving people with rare diseases.

We trace our origins back to 1986, when a small team of entrepreneurs founded the company to seek out solutions to address a number of unmet medical needs. Within the first two years of operation, we launched a range of supplemental calcium products for patients seeking to treat or prevent osteoporosis. Soon after, the company had innovative drug development programs under way on behalf of patients facing challenging conditions such as Alzheimer's disease and end-stage renal failure.

By the mid-90s, we had embarked on the first of what would be a series of strategic acquisitions to expand into new areas, develop our clinical pipeline and bring new products to market. Since those early years, we have continued to grow dramatically through the completion of more than 20 combinations over the past two-plus decades.

While Shire continued to evolve in the specialty pharma field and expand its portfolio, the transformative acquisition of TKT in 2005 for lysosomal storage diseases opened the door to biologic therapies to begin our focus on rare diseases.

Shire continued to sharpen its rare disease focus through acquisitions of not only marketed products, but also compounds that were in clinical development, getting more involved in ADHD via the acquisition of New River Pharmaceuticals in 2007, and Hereditary Angioedema (HAE) via Jerini in 2008.

From 2013 to 2016, we sustained our significant strategic transformation to be recognized in our industry as a leading global biotech company by increasing scale and momentum. We acquired SARcode to enter ophthalmics, we expanded our HAE business with the acquisitions of ViroPharma and Dyax, and we strengthened our GI business with the acquisition of NPS.

Our leadership was further solidified with the completion of the acquisition of Baxalta in 2016, creating the world's leading global biotechnology company focused on serving patients with rare diseases.

The Baxalta acquisition is by far the largest we have ever completed. Through this integration, we acquired three new therapeutic areas (hematology, immunology, and oncology), more than tripled our number of employees, gained several market-leading drugs, increased our clinical development programs and expanded the clinical pipeline.

Now, with medicines available in more than 100 countries, we are in a leading position to provide solutions globally that enable more patients and families around the world to live their lives to the fullest.


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