Takeda Pharmaceutical Company Limited
Takeda Pharmaceutical Company Limited works on 44 rare diseases tracked on Trial Friend, including Alagille Syndrome, Alpha-1 Antitrypsin Deficiency, Chronic Graft-versus-Host Disease and 41 more, with 41 recruiting clinical trials and 20 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 44 rare diseases, including Alagille Syndrome, Alpha-1 Antitrypsin Deficiency, Chronic Graft-versus-Host Disease, Common Variable Immunodeficiency, Cystic Fibrosis, and 39 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 all active and recruiting 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 41 active clinical trials across 5 development stages, with 41 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 includes all of Takeda Pharmaceutical Company Limited's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.
Takeda Pharmaceutical Company Limited Clinical Trials (41)
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.
Takeda Pharmaceutical Company Limited FDA-Approved Drugs (20)
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 |
|---|---|---|---|
| PIOGLITAZONE AND METFORMIN HYDROCHLORIDE | Actoplus Met oral | Aug 29, 2005 | |
| PIOGLITAZONE | Actos oral | Jul 15, 1999 | |
| BRIGATINIB Kinase Inhibitor [EPC] | Alunbrig oral | Apr 28, 2017 | |
| CARBAMAZEPINE Mood Stabilizer [EPC] | Carbatrol oral | Sep 30, 1997 | |
| PIOGLITAZONE AND GLIMEPIRIDE Sulfonylurea [EPC] | Duetact oral | Jul 28, 2006 |
Takeda Pharmaceutical Company Limited Trial Locations
Takeda Pharmaceutical Company Limited clinical trials are running at 1,407 sites in 46 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 (44)
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 enzyme alpha-1 antitrypsin. Without adequate protection, neutrop...
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...
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...
Dravet Syndrome is a severe form of epilepsy that typically begins in infancy with prolonged seizures triggered by fever. Most cases are caused by mutations in the SCN1A gene encoding a sodium channel...
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.