Takeda Pharmaceutical Company Limited
Takeda Pharmaceutical Company Limited works on 47 rare diseases tracked on Trial Friend, including Alagille Syndrome, Alpha-1 Antitrypsin Deficiency, Chronic Graft-versus-Host Disease and 44 more, with 45 active clinical trials (31 recruiting) and 13 FDA-approved rare disease drugs.
Takeda became one of the world's largest rare disease companies through its 2019 acquisition of Shire, gaining a portfolio spanning hereditary angioedema (Takhzyro), Hunter syndrome (Elaprase), Gaucher disease (VPRIV), and hemophilia (ADVATE, ADYNOVATE). The company continues investing in rare hematology, immunology, and neuroscience programs.
Focus areas at Takeda Pharmaceutical Company Limited
Within its broader pharmaceutical portfolio, Takeda Pharmaceutical Company Limited has active clinical trial programs and drug development efforts across 47 rare diseases, including Alagille Syndrome, Alpha-1 Antitrypsin Deficiency, Chronic Graft-versus-Host Disease, Chronic Inflammatory Demyelinating Polyneuropathy, Common Variable Immunodeficiency, and 42 additional rare conditions. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows active and recruiting rare disease studies sponsored by Takeda Pharmaceutical Company Limited, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Takeda Pharmaceutical Company Limited is headquartered in Tokyo, Japan, founded in 1781, publicly traded under the ticker symbol 4502. The company maintains a dedicated rare disease division alongside its broader therapeutic portfolio, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Takeda Pharmaceutical Company Limited Drug Pipeline
Takeda Pharmaceutical Company Limited has 45 active clinical trials across 5 development stages, with 31 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
Takeda Pharmaceutical Company Limited Clinical Trials (45)
Active and recruiting clinical trials sponsored by Takeda Pharmaceutical Company Limited, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
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Takeda Pharmaceutical Company Limited FDA-Approved Drugs (13)
Medications developed or marketed by Takeda Pharmaceutical Company Limited that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.
| Drug Name | Brand Name | Rare Diseases | Approval Date |
|---|---|---|---|
| IDURSULFASE Hydrolytic Lysosomal Glycosaminoglycan-specific Enzyme [EPC] | ELAPRASE intravenous | Jul 24, 2006 | |
| BUDESONIDE Corticosteroid [EPC] | Eohilia oral | Feb 9, 2024 | |
| ICATIBANT ACETATE | Firazyr subcutaneous | Aug 25, 2011 | |
| TEDUGLUTIDE GLP-2 Analog [EPC] | Gattex subcutaneous | Dec 21, 2012 | |
| ECALLANTIDE Plasma Kallikrein Inhibitor [EPC] | Kalbitor subcutaneous | Dec 1, 2009 |
Takeda Pharmaceutical Company Limited Trial Locations
Takeda Pharmaceutical Company Limited clinical trials are running at 1,228 sites in 45 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (47)
Diseases targeted by Takeda Pharmaceutical Company Limited's clinical trial and drug development programs
Alagille syndrome is a rare autosomal dominant disorder affecting the liver, heart, skeleton, face, and eyes. The condition results from mutations in genes regulating the Notch signaling pathway, lead...
Alpha-1 antitrypsin deficiency is a genetic disorder affecting the lungs and liver, caused by insufficient production of the protective protein alpha-1 antitrypsin. Without adequate protection, neutro...
Chronic graft-versus-host disease is an immune-mediated complication after allogeneic stem cell or bone marrow transplant, in which donor immune cells attack the recipient's tissues. It can affect the...
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) is an autoimmune disorder affecting the peripheral nerves, causing progressive weakness and impaired function in the legs and arms. The immune ...
Common variable immunodeficiency is a primary immunodeficiency disorder characterized by low levels of immunoglobulins and impaired antibody responses. Patients experience recurrent infections affecti...
Cystic fibrosis is an autosomal recessive genetic disorder affecting the CFTR protein, which normally regulates chloride transport. Defective CFTR causes thick, sticky secretions in the lungs and dige...
Patient Resources
Organizations and resources related to Takeda Pharmaceutical Company Limited's rare disease focus areas
Frequently Asked Questions About Takeda Pharmaceutical Company Limited
Common questions about Takeda Pharmaceutical Company Limited's rare disease programs, clinical trials, and treatments.