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If you had hemophilia in the 1940s, your life would have been very different from the one a person with hemophilia lives today. Then, there were no factor replacement therapies, bypassing agents, or prophylaxis regimens. For a person living with hemophilia, there was joint pain, bleeds treated with whole-blood infusions, and life spans that often didn’t reach adulthood.1-3 Clearly, a new approach to hemophilia treatment was needed. Fortunately, that’s when Baxter’s hemophilia division stepped in, which is now part of Shire, as of 2016.4
A revolution begins
It began with an aggressive and passionate effort to provide people with hemophilia a bleed-free life more than 70 years ago.5 Then in 1966, one of our scientists, a hemophilia patient himself, had a significant breakthrough. Dr. Murray Thelin helped create one of the first factor VIII replacement therapies and ushered in the modern age of hemophilia treatment.3,6
Although the first factor VIII replacement therapies were a life-changing improvement for tens of thousands of people with hemophilia, the innovation didn’t stop there.
In the years that followed, we advanced the treatment of hemophilia A, hemophilia B, hemophilia A or B with inhibitors, von Willebrand disease, and acquired hemophilia A with significant developments. Many of these were firsts: the first recombinant factor VIII treatment, the first needleless transfer device, the first recombinant factor VIII treatment free of blood-based additives, the first recombinant treatment for people with von Willebrand disease, and the first recombinant porcine factor VIII for acquired hemophilia.6-9
The breakthroughs continue
Thanks to the many contributions that have been made in the past, and which Shire continues to make, to the treatment of bleeding disorders, Shire’s vision for patients with a bleeding disorder is closer to realization than ever before.
Today, the focus is on using individualized prophylactic dosing for preventing bleeds.10 With a recent Shire development, the first FDA-cleared PK dosing software for use in hemophilia, people with hemophilia can conveniently estimate their PK profile and the factor levels in their body at any given time. This may allow patients to tailor their prophylaxis to their activities while ensuring adequate factor coverage.11-13
And the innovation continues. Research and development is going strong with 20 ongoing clinical trials in bleeding disorders, including one in gene therapy, as well as advancements in other novel therapies. Shire has engaged hundreds of the world’s leading scientists, researchers, and patient support specialists to help them.5
Most fundamentally, Shire is collaborating with the bleeding disorders community, including patient associations that have enabled the diagnosis of more than 30,000 hemophilia patients around the world.5 Shire has listened to, learned from, and championed their needs. This bleeding disorders community is our community. It’s why Shire is always pushing ahead, proactively shaping the future of bleeding disorders and continually elevating care for patients.
- Philipp C. The aging patient with hemophilia: complications, comorbidities, and management issues. Hematology Am Soc Hematol Educ Program. 2010;2010:191-196.
- History of bleeding disorders. National Hemophilia Foundation. https://www.hemophilia.org/Bleeding-Disorders/History-of-Bleeding-Disorders. Accessed August 30, 2018.
- Dateline Federation: Honoring Our Past, Building Our Future. 41st ed. Washington, DC: Hemophilia Federation of America; 2014. http://www.hemophiliafed.org/dateline/HFA_Dateline_2014_Q2_Summer/HFA_Dateline_2014_Q2_Summer.pdf. Accessed August 30, 2018.
- Shire to combine with Baxalta, creating the global leader in rare diseases [news release]. January 11, 2016. https://www.shire.com/-/media/shire/shireglobal/shirecom/pdffiles/newsroom/2016/shire-to-combine-with-baxalta-pr-1-11-16-final.pdf?la=en&hash=A7A87F964B4026EDB959A608A5D57357795DCC44. Accessed September 14, 2018.
- Shire’s 70+ year commitment to the hemophilia community [news release]. June 6, 2018. https://www.shire.com/newsroom/2018/january/7sossj. Accessed August 30, 2018.
- Kingdon HS, Lundblad RL. An adventure in biotechnology: the development of haemophilia A therapeutics – from whole-blood transfusion to recombinant DNA to gene therapy. Biotechnol Appl Biochem. 2002;35:141-148.
- Grillberger L, Kreil TR, Nasr S, Reiter M. Emerging trends in plasma-free manufacturing of recombinant protein therapeutics expressed in mammalian cells. Biotechnol J. 2009;4:186-201.
- Janbain M, Leissinger C, Kruse-Jarres R. Acquired hemophilia A: emerging treatment options. J Blood Med. 2015;6:143-150.
- Shire, Inc. The first and only recombinant treatment for adults affected by von Willebrand disease, launches in the US. [press release]. https://www.shire.com/newsroom/2016/august/nbtjyp. Accessed September 28, 2018.
- Berntorp E, Spotts G, Patrone L, Ewenstein BM. Advancing personalized care in hemophilia A: ten years’ experience with an advanced category antihemophilic factor prepared using a plasma/albumin-free method. Biologics. 104:8;115-127.
- Collins PW. Personalized prophylaxis. Haemophilia. 2012;18(suppl 4):131-135.
- Hazendonk HCAM, van Moort I, Mathôt RAA, et al. Setting the stage for individualized therapy in hemophilia: what role can pharmacokinetics play? Blood Rev. 2018;32(4):265-271.
- US Food & Drug Administration. Substantially Equivalent 510(k) Device Information: BK170028. https://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/SubstantiallyEquivalent510kDeviceInformation/UCM592876.pdf. Accessed September 28, 2018.