Transforming Rare Disease Care | Takeda Pharmaceuticals
Rare Diseases
We are activating meaningful change and empowering the rare disease community at every step of the journey.
With patients as our top priority, our rare portfolio of approved treatments and pipeline assets are designed to help address high unmet medical needs and improve patient outcomes. Our core rare disease focus areas span Hereditary Angioedema (HAE), Hematology, Lysosomal Storage Disorders (LSD), Transplant, Neuroscience, Immune-mediated and Primary Immune Deficiency Diseases, Protein Deficiencies, Immunology and Gastrointestinal and Inflammation.
Together with the community, we are optimistic about the progress we can achieve in rare diseases—because patients and their caregivers deserve answers and a path forward to a brighter future.
Rare Hematology and Hemostasis
Guided by our longstanding heritage, we are dedicated to pioneering innovative therapies and solutions that address patients’ unmet needs for the blood and bleeding disorder community. We are continuing to invest in our broad portfolio of innovative medicines, including in the hematology space, to optimize and expand the use of existing treatments so patients can live life with fewer limitations.
Disease areas
- Hemophilia
- Von Willebrand Disease
- Thrombotic Thrombocytopenic Purpura
- Immune Thrombocytopenia
Rare Immunology
Underscored by decades of scientific research and innovation in advancing treatment options in allergy and immunology, we’re actively partnering with the community and prioritizing the evolving needs of patients. We seek to personalize care for individuals living with various rare angioedema, immune-mediated and primary immune deficiency diseases by addressing unmet needs across generations, reducing the time to diagnosis and ensuring equitable access to care.
Disease areas
- Hereditary Angioedema (HAE)
- Immune Deficiency Diseases
- Rare Autoimmune Disorders
- Hypoalbuminemia and Hypovolemia
- Severe Congenital Protein C Deficiency
- Prothrombin Deficiency
- Alpha-1 Antitrypsin Deficiency (AATD)
- Eosinophilic Esophagitis (EoE)
- Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
- Multifocal Motor Neuropathy (MMN)
Lysosomal Storage Disorders (LSD)
Our strong legacy in LSD is guided by our patient-first approach, ensuring each patient we serve is valued and supported along their journey. We work closely with the medical community and advocacy organizations to advance understanding of treatment strategies, enhance disease management, and drive scientific knowledge exchange—with the shared goal of improving the diagnostic journey.
Disease areas
- Hunter Syndrome
- Fabry Disease
- Gaucher Disease
Post-Transplant Cytomegalovirus (CMV)
Our commitment to the transplant community is backed by our deep understanding of the complexities of managing post-transplant CMV infection. We are dedicated to supporting endeavors in post-transplant care and seek to translate the community’s needs into science that helps CMV patients preserve and improve life after transplant.
