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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.

Type
Diversified Pharma
Ticker
4502
Headquarters
Tokyo, Japan
Founded
1781
Website
takeda.com
45
Active Rare Disease Trials
13
Approved Rare Disease Drugs
47
Rare Diseases in Portfolio
245
Years Active

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.

Understand Takeda Pharmaceutical Company Limited's pipeline
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3
Early Phase / Phase 13 trials
Healthy Volunteers+1 more
Recruiting
Diffuse Large B Cell Lymphoma+4 more
Active Not Recruiting
10
Phase 210 trials
15
Phase 315 trials
1
Phase 4 / Post-Market1 trial
Recruiting
16
Other16 trials

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.

Ask about Takeda Pharmaceutical Company Limited's trials
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RECRUITINGPHASE3Recently updatedNCT06499285

A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions

Intervention: Elritercept, Placebo

The main aim of this study is to find out how well elritercept works in lowering the need for RBC transfusions. Other aims are to learn how well elritercept works in reducing the need for RBC transfusions over longer periods of time or in adults with high transfusion needs. The study will also check on how safe elritercept is and how well it is tolerated.

Ages 18 Years+182 locations
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RECRUITINGPHASE2Recently updatedNCT07319845

A Study of TAK-226 for Anemia in Japanese Patients With Lower-Risk Myelodysplastic Syndromes

Intervention: TAK-226

The main aim of the study is to evaluate how TAK-226 improves symptoms of transfusion-dependent anemia in Japanese patients with lower-risk myelodysplastic syndromes. The study consists of Screening Period (up to 6 weeks), Treatment Period, Safety Follow-Up Period (8 weeks), and Long-Term Follow-Up Period (5 years from the first dose of the study drug or 3 years after the last dose, whichever is longer). Participants of this study will be administered TAK-226 during Treatment Period. Subsequently, the participants will be monitored for side effects related to the study treatment during Safety Follow-Up Period and Long-Term Follow-Up Period. The approximate duration of participation for a participant is up to approximately 6 years. During the study period, participants will visit the study clinic/hospital multiple times as per the study schedule. During Treatment Period, the participants will come to the clinic/hospital approximately every two to four weeks.

Ages 18 Years+21 locations
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RECRUITINGPHASE2Recently updatedNCT07648030

A Study of Rusfertide in Japanese Adults With Polycythemia Vera

Intervention: Rusfertide

Polycythemia vera (PV) is a rare blood cancer in which the body makes too many red blood cells. This can make the blood thicker and may increase the risk of serious health problems such as blood clots. Many people with PV need regular phlebotomy, which is a procedure to remove blood, to help keep their hematocrit level under control. Hematocrit is the proportion of red blood cells in the blood. The main aim of this study is to evaluate whether rusfertide helps Japanese participants with PV keep their hematocrit under control and avoid the need for phlebotomy. All participants in this study will receive rusfertide. This study is open-label, which means both the participants and the study team will know what treatment is being given. Participants will be followed for up to about 244 weeks, including a screening period, a 52-week initial treatment period, a long-term extension period, and a 4-week safety follow-up period.

Ages 18 Years+9 locations
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RECRUITINGPHASE3Recently updatedNCT06722235

A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia

Intervention: Mezagitamab, Placebo

Primary immune thrombocytopenia (ITP) is a condition where the immune system mistakenly destroys platelets, which are cells that help stop bleeding. This leads to a low number of platelets, making it easier to bruise or bleed. The main aim of this study is to learn whether mezagitamab, when given just under the skin (subcutaneously \[SC\]), is effective in keeping the platelet count of adults with ITP stable when compared to a placebo. A placebo looks like medicine but doesn't have any active ingredients in it. The participants will be treated with mezagitamab for up to 6 months. During the study, participants will visit their study clinic several times. Participants who complete the TAK-079-3002 study or do not have any response to study treatment by week 16 (according to study criteria) will be given the opportunity to participate in a continuation study to receive open label mezagitamab (if they are eligible and the site is able to open the continuation study).

Ages 18 Years+124 locations
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RECRUITINGPHASE2, PHASE3Recently updatedNCT07646678

A Study of TAK-360 in People With Narcolepsy or Idiopathic Hypersomnia

Intervention: TAK-360, Placebo

Idiopathic HypersomniaNarcolepsy Type 1Narcolepsy Type 2

Central hypersomnia conditions are a group of sleeping disorders where the brain has trouble keeping a person awake during the day (called excessive daytime sleepiness or EDS). These conditions usually include narcolepsy (type 1 and 2) and idiopathic hypersomnia (IH). Narcolepsy type 1 (NT1) includes sudden muscle weakness while you stay awake, called cataplexy, often triggered by strong emotions. Narcolepsy type 2 (NT2) does not include cataplexy. People with narcolepsy typically feel refreshed by short naps. People with IH feel extremely sleepy during the day, and do not feel refreshed by sleep. Waking up from sleep is difficult. This is common in the morning and also when waking up from long naps. The study wants to learn about TAK-360 when taken over a long time period; this is called a long-term extension or LTE study. The main aim of this LTE study is to find out how well participants with NT1, NT2, and IH tolerate TAK-360 over a longer period (long-term tolerability) and to learn how safe TAK-360 is when given over a longer period of time (long-term safety). Participants who completed one of the TAK-360 parent studies can join this study if they meet the study rules. Parent studies include TAK-360-2001(NCT06952699), TAK-360-2002 (NCT06812078), or other TAK-360 studies that evaluate the TAK-360 medicine. All participants will receive TAK-360 in this study. They will either receive the same dose as they did in the parent study, or the closest dose available in this LTE study. Participants who received placebo (the placebo looks just like TAK-360 but does not have any medicine in it) in their parent study will receive one of the TAK-360 doses available in this study. Placebo will only be used to not reveal the dose of TAK-360 from parent studies to investigator, participants, and sponsor. Sponsor, investigators and participants will not know which TAK-360 dose was used in the LTE study as long as the parent study is ongoing. The participants will have to visit the clinic multiple times during this study.

Ages 18 Years - 71 Years24 locations
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RECRUITINGRecently updatedNCT05001087

A "Physician & Patient-powered" Cohort Registry (MY MYELOMA)

Intervention: Observation

The aim of this observational study is the creation of a national multiple myeloma registry to monitor the current routine clinical practice in Italy and describe the standard of care adopted for the diagnosis and treatment of patients with multiple myeloma in the different Italian hematology centers.

Ages 18 Years+27 locations
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RECRUITINGPHASE3Recently updatedNCT06948318

A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia

Intervention: Mezagitamab

Primary immune thrombocytopenia (ITP) is a condition where the immune system mistakenly destroys platelets, which are cells that help stop bleeding. This leads to a lower number of platelets, making it easier to bruise or bleed. The main aim of this study is to check how safe mezagitamab is and how well it is tolerated by adults with chronic primary ITP, if given over a longer time. Other aims are to learn how effective treatment with mezagitamab is and how the body processes it (called pharmacokinetics or PK) over a longer time. Participants of the following previous mezagitamab studies will be invited to join this continuation study: TAK-079-3002 and TAK-079-1004. In this continuation study, participants will receive mezagitamab when certain protocol criteria are met. During the study, participants will visit their study clinic several times.

Ages 18 Years+118 locations
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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 NameBrand NameRare DiseasesApproval Date
IDURSULFASE
Hydrolytic Lysosomal Glycosaminoglycan-specific Enzyme [EPC]
ELAPRASE
intravenous
Jul 24, 2006
BUDESONIDE
Corticosteroid [EPC]
Eohilia
oral
Feb 9, 2024
ICATIBANT ACETATEFirazyr
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.

United States
290▼
United Kingdom
166▼
Italy
92▼
Japan
86▼
Spain
64▼
Australia
56▼
China
54▼
Germany
48▼
France
40▼
Poland
34▼
South Korea
23▼
Canada
22▼

Rare Disease Focus Areas (47)

Diseases targeted by Takeda Pharmaceutical Company Limited's clinical trial and drug development programs

Alagille SyndromeLiver & Hepatic

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...

Prevalence: 1 in 30,000 to 43,000 people
Alpha-1 Antitrypsin DeficiencyPulmonary & Respiratory

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...

Prevalence: 1 in 1,500 to 3,500 people of European ancestry; about 100,000 people in the U.S.
Chronic Graft-versus-Host DiseaseBlood & Immune

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...

Prevalence: Affects 30 to 70% of patients who receive allogeneic hematopoietic stem cell transplant; approximately 14,000 new cases annually in the U.S.
Chronic Inflammatory Demyelinating PolyneuropathyNeurological & Neuromuscular

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 ...

Prevalence: About 2.8 per 100,000 people in a 2019 pooled analysis of 9 studies (Broers et al., Neuroepidemiology); NORD estimates 5 to 7 per 100,000, and NIH's GARD estimates about 50,000 people in the U.S.
Common Variable ImmunodeficiencyBlood & Immune

Common variable immunodeficiency is a primary immunodeficiency disorder characterized by low levels of immunoglobulins and impaired antibody responses. Patients experience recurrent infections affecti...

Prevalence: 1 in 25,000 to 50,000 people
Cystic FibrosisPulmonary & Respiratory

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...

Prevalence: Nearly 40,000 children and adults in the U.S.; 1 in 2,500 to 3,500 births among Caucasians

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.