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Daiichi Sankyo

Drugmaker with rare disease work · based in Tokyo, Japan · founded 2005 · ticker 4568 · daiichisankyo.com

Daiichi Sankyo works on 7 rare diseases we track, including Acute Myeloid Leukemia, Adrenocortical Carcinoma and 5 more. It has 8 active clinical trials, and 3 are looking for volunteers now. It also has 2 FDA-approved rare disease drugs.

Daiichi Sankyo is a Japanese pharmaceutical company with roots dating back to 1899 (formed through the 2005 merger of Daiichi Pharmaceutical and Sankyo). It is best known for cancer drugs called antibody-drug conjugates.

Daiichi Sankyo pipeline

Each row lists the diseases Daiichi Sankyo is studying at that stage, from the ones closest to approval down to the earliest work.

Approved
Drugs the FDA has approved.
Phase 3
Large studies, usually the last step before a company asks the FDA for approval.

Tenosynovial Giant Cell Tumor · HER2-Positive Primary Breast Cancer

Phase 2
Mid-size studies that check whether a drug works and at what dose.

Acute Myeloid Leukemia 3 trials recruiting · Hematological Malignancies recruiting · Duchenne Muscular Dystrophy

Phase 1
First studies in people, mostly checking safety.

Daiichi Sankyo clinical trials

These are Daiichi Sankyo trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.

3 trials looking for volunteers now

Looking for volunteersPhase 2Recently updatedNCT06824168

Evaluation of Two Dose Levels of Quizartinib as Maintenance in FLT3-ITD (+) Acute Myeloid Leukemia Patients in Complete Remission

Tests Quizartinib High Dose, Quizartinib Low Dose

For Acute Myeloid Leukemia and Leukemia

This clinical two-arm trial is designed to evaluate two doses of quizartinib as maintenance therapy after induction/consolidation in participants with FMS-like tyrosine kinase 3 (FLT3)-internal tandem duplication (ITD) (+) acute myeloid leukemia (AML) in first complete remission (CR) who have not received allogeneic hematopoietic stem cell transplantation (allo-HSCT).

Ages 18 Years+64 locations
View full study
Looking for volunteersPhase 1/2Recently updatedNCT07220616

A First-in-Human Trial of DS3790a in Participants With Hematological Malignancies

Tests DS3790a, Combination drug, Combination drug

For Hematological Malignancies and B-cell Non-Hodgkin Lymphoma

This clinical trial is designed to assess the safety, preliminary efficacy, and pharmacokinetics (PK) of DS3790a monotherapy and combination regimens in participants with hematological malignancies.

Ages 18 Years+8 locations
View full study
Looking for volunteersPhase 1Updated a few months agoNCT07189403

A Study of DS9051b in Participants With Advanced or Metastatic Adrenocortical Carcinoma and Metastatic Castration-resistant Prostate Cancer

Tests DS9051b

For Advanced or Metastatic Adrenocortical Carcinoma and Metastatic Castration-resistant Prostate Cancer

This FIH study is designed to assess the safety, tolerability, and preliminary efficacy signals of DS9051b in participants with ACC and mCRPC.

Ages 18 Years+6 locations
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5 trials underway, not taking new volunteers

Underway, not taking new volunteersPhase 1/2Recently updatedNCT03661307

Quizartinib, Decitabine, and Venetoclax in Treating Participants With Untreated or Relapsed Acute Myeloid Leukemia or High Risk Myelodysplastic Syndrome

Tests Decitabine, Quizartinib, Venetoclax

For Acute Myeloid Leukemia, Myelodysplastic Syndrome, Recurrent Acute Myeloid Leukemia, Recurrent Myelodysplastic Syndrome and Refractory Acute Myeloid Leukemia

This phase I/II trial studies how well quizartinib, decitabine, and venetoclax work in treating participants with acute myeloid leukemia or high risk myelodysplastic syndrome that is untreated or has come back (relapsed). Quizartinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as decitabine and venetoclax, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving quizartinib and decitabine may work better at treating acute myeloid leukemia and myelodysplastic syndrome.

Ages 18 Years+1 location
View full study
Underway, not taking new volunteersPhase 2Recently updatedNCT04433234

Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

Tests DS-5141b

For Duchenne Muscular Dystrophy

This is a multicenter, open-label, long-term, extension, phase 2 study to evaluate the safety and efficacy of long-term treatment with DS-5141b in patients with DMD who have completed DS5141-A-J101.

Ages 5 Years+2 locations
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Underway, not taking new volunteersPhase 3Updated a few months agoNCT04488822

A Study of the Efficacy and Safety of Pexidartinib in Adult Subjects With TGCT

Tests Pexidartinib

For Tenosynovial Giant Cell Tumor

This study will assess pexidartinib in adult participants with symptomatic TGCT that is associated with severe morbidity or functional limitations and not amendable to improvement with surgery.

Ages 18 Years+9 locations
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Underway, not taking new volunteersPhase 3No updates in a whileNCT04622319

A Study of Trastuzumab Deruxtecan (T-DXd) Versus Trastuzumab Emtansine (T-DM1) in High-risk HER2-positive Participants With Residual Invasive Breast Cancer Following Neoadjuvant Therapy (DESTINY-Breast05)

Tests DS-8201a, T-DM1

For HER2-Positive Primary Breast Cancer and Residual Invasive Breast Cancer

Patients with HER2-positive primary breast cancer (BC) who do not achieve complete response after appropriate neoadjuvant therapy are at higher risk of disease recurrence. More effective treatment options are needed for this patient population. This study will examine the efficacy and safety of trastuzumab deruxtecan (T-DXd) compared with trastuzumab emtansine (T-DM1) in high-risk patients with residual invasive breast cancer following neoadjuvant therapy.

Ages 18 Years+482 locations
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Underway, not taking new volunteersPhase 1/2No updates in a whileNCT03793478

Safety and Efficacy of Quizartinib in Children and Young Adults With Acute Myeloid Leukemia (AML), a Cancer of the Blood

Tests Quizartinib, Fludarabine, Cytarabine, Intrathecal (IT) triple chemotherapy prophylaxis, Etoposide

For Acute Myeloid Leukemia

Quizartinib is an experimental drug. It is not approved for regular use. It can only be used in medical research. Children or young adults with a certain kind of blood cancer (FLT3-ITD AML) might be able to join this study if it has come back after remission or is not responding to treatment.

Ages 1 Month - 21 Years27 locations
View full study
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FDA-approved drugs from Daiichi Sankyo

From the openFDA database. Tap a drug for side effects, dosing and help paying.

Turalio pexidartinib hydrochloride

For Tenosynovial Giant Cell Tumor. Approved Aug 2, 2019. Taken by mouth.

Vanflyta quizartinib

For Acute Myeloid Leukemia. Approved Jul 20, 2023. Taken by mouth.

More about Daiichi Sankyo

Its FDA-approved rare disease medicines include Vanflyta (quizartinib), approved in July 2023 for newly diagnosed FLT3-ITD acute myeloid leukemia, and Turalio (pexidartinib), approved in August 2019 for adults with tenosynovial giant cell tumor that causes severe symptoms and cannot be helped by surgery. It is also running a first-in-human trial of DS9051b, a drug taken by mouth, in adrenocortical carcinoma (NCT07189403).

Where Daiichi Sankyo trials take place

The trials above run at 599 study sites in 32 countries. Tap a country to see its states, cities and hospitals.

United States
157▼
Germany
81▼
Spain
52▼
China
43▼
France
29▼
Japan
29▼
South Korea
26▼
Italy
24▼
Brazil
17▼
United Kingdom
17▼
Australia
16▼
Taiwan
15▼

Rare diseases Daiichi Sankyo works on

Each disease page lists every open trial, not only Daiichi Sankyo's, plus approved treatments.

Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a fast-growing cancer of the bone marrow in which immature white blood cells called blasts crowd out normal blood cells, causing fatigue, infections and bleeding...

How common: The National Cancer Institute's SEER program estimates 22,720 new US cases and 11,500 deaths in 2026, about 1.1% of new cancers, and 83,311 people were living with AML in 2023

Adrenocortical Carcinoma

Adrenocortical carcinoma is a rare, aggressive cancer of the adrenal cortex, the outer layer of the adrenal glands that sit on top of each kidney.

How common: About 1-2 per million people per year; approximately 600 new cases per year in the U.S

Cholangiocarcinoma

Cholangiocarcinoma is a rare and aggressive cancer that forms in the bile ducts, the thin tubes that carry digestive fluid (bile) from the liver to the small intestine.

How common: About 8,000 new cases per year in the U.S.; rising incidence worldwide

Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is an X-linked genetic disorder causing progressive muscle weakness and degeneration, beginning in early childhood.

How common: 1 in 3,500 to 5,000 male births

Fibrodysplasia Ossificans Progressiva

Fibrodysplasia Ossificans Progressiva (FOP) is an extremely rare genetic disorder characterized by progressive heterotopic ossification, where soft tissues (muscles, tendons, ligaments) gradually...

How common: Extremely rare, about 1 in 1 million to 1 in 2 million people worldwide

Myelodysplastic Syndromes

Myelodysplastic syndromes are a group of blood cancers in which the bone marrow fails to produce enough healthy blood cells.

How common: 10,000 to 15,000 new cases annually in the U.S.; estimated 60,000 to 170,000 Americans living with MDS

Tenosynovial Giant Cell Tumor

Tenosynovial giant cell tumor (TGCT) is a rare tumor that grows in the lining of a joint, tendon sheath or bursa.

How common: A Dutch nationwide study found about 43 new cases per million people each year, 39 localized and 4 diffuse (Mastboom et al., 2017)

Where to get support

Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Daiichi Sankyo's own site is daiichisankyo.com, and our help paying for treatment page lists drugmaker programs and charity funds.

Questions about Daiichi Sankyo