Therapeutic Focus - Rare Diseases | Takeda Pharmaceuticals

Rare Diseases

Children running on a field

We aspire to transform the treatment of rare diseases in hematology and hemostasis, immunology, metabolic and lysosomal storage disorders, and cytomegalovirus (CMV) infection in transplant.

These rare diseases can have symptoms that vary widely and progress very differently from person to person, which means that people affected by these diseases are frequently misdiagnosed, leading to significant diagnostic delay.

Rare Hematology and Hemostasis

We are a leader with more than 70 years of experience in rare hematology. Our experience in hematology means we are well-prepared to meet today’s needs as we pursue future developments in the treatment of bleeding disorders. Together with the hematology community, we are raising expectations for the future, including earlier diagnosis, earlier and protection against bleeds, and more personalized patient care.

Disease areas 

  • Hemophilia

  • Von Willebrand Disease

  • Thrombotic thrombocytopenic purpura

Rare Immunology

Within Rare Immunology we focus on driving continuous innovation and personalized care through our portfolio of plasma products and innovative targeted treatments, devices, diagnostics and other technological services, for the benefit of patients with rare immunological disorders. Our core therapeutic areas include Hereditary Angioedema, immuno-deficiencies, rare autoimmune disorders, post-transplant complications and rare specialty care.

Disease areas

  • Hereditary Angioedema

  • Immune Deficiency Diseases

  • Rare Autoimmune Disorders

  • Hypoalbuminemia and Hypovolemia

  • Severe Congenital Protein C Deficiency

  • Prothrombin Deficiency

  • Alpha-1 Antitrypsin Deficiency

Rare Metabolic

We have a strong legacy in developing treatments for lysosomal storage disorders (LSDs), with a portfolio that includes commercial products, late-stage investigational therapies, and pipeline candidates. Because rare genetic and metabolic diseases can have symptoms that vary widely and progress differently from person to person, we empower global education and awareness, and partner with medical and research organizations. We are committed to helping reduce the amount of time between the onset of symptoms and diagnosis and to accelerating the development of innovative new treatments.

Select disease areas

  • Hunter Syndrome

  • Fabry Disease

  • Gaucher Disease

  • CMV and Transplant