Bristol Myers Squibb

Bristol Myers Squibb works on 37 rare diseases tracked on Trial Friend, including Adrenocortical Carcinoma, Aplastic Anemia, Cholangiocarcinoma and 34 more, with 46 recruiting clinical trials and 7 FDA-approved rare disease drugs.

Bristol Myers Squibb is a global biopharmaceutical company whose 2019 acquisition of Celgene significantly expanded its hematology and oncology portfolio. BMS develops treatments for myelofibrosis, thalassemia, and rare cancers, with Reblozyl (luspatercept) for beta thalassemia and active programs in rare immunologic conditions including myasthenia gravis.

Type
Diversified Pharma
Ticker
BMY
Headquarters
Princeton, United States
Founded
1989
Website
bms.com
46
Active Rare Disease Trials
7
Approved Rare Disease Drugs
37
Rare Diseases in Portfolio
37
Years Active
FDA decision ahead
The FDA is due to decide on Camzyos (mavacamten) for Obstructive hypertrophic cardiomyopathy, adolescents by September 30, 2026.
The FDA is due to decide on Mezigdomide for Relapsed/refractory multiple myeloma by May 13, 2027.
See all upcoming rare disease FDA decisions →

Focus areas at Bristol Myers Squibb

Within its broader pharmaceutical portfolio, Bristol Myers Squibb has active clinical trial programs and drug development efforts across 37 rare diseases, including Adrenocortical Carcinoma, Aplastic Anemia, Cholangiocarcinoma, Chronic Graft-versus-Host Disease, Common Variable Immunodeficiency, and 32 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 Bristol Myers Squibb, 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.

Bristol Myers Squibb is headquartered in Princeton, United States, founded in 1989, publicly traded under the ticker symbol BMY. 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.

Bristol Myers Squibb Drug Pipeline

Bristol Myers Squibb has 46 active clinical trials across 5 development stages, with 46 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 Bristol Myers Squibb'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.

Understand Bristol Myers Squibb's pipeline
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9
Early Phase / Phase 19 trials
B-Cell Lymphoma, Unspecified+4 more
Recruiting
Systemic Lupus Erythematosus+3 more
Recruiting
Recruiting
Advanced Malignant Tumors
Recruiting
Advanced Solid Tumor+2 more
Recruiting
18
Phase 218 trials
MDS+1 more
Recruiting
Locally Advanced Non-Small Cell Lung Cancer (NSCLC)+1 more
Recruiting
Anemia
Recruiting
Anemia, Sickle Cell+1 more
Recruiting
6
Phase 36 trials
Myeloproliferative Disorders+4 more
Recruiting
3
Phase 4 / Post-Market3 trials
Cardiomyopathy, Hypertrophic
Recruiting
10
Other10 trials
Recruiting
Cardiomyopathy
Recruiting
Non Small Cell Lung Cancer NSCLC
Recruiting

Bristol Myers Squibb Clinical Trials (46)

Active and recruiting clinical trials sponsored by Bristol Myers Squibb, 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 Bristol Myers Squibb's trials
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RECRUITINGPHASE1, PHASE2Recently updatedNCT05181735

Study Evaluating Combination of Luspatercept in LR-MDS Without RS Having Failed or Being Ineligible to ESA

Intervention: Luspatercept Injection [Reblozyl], Eprex

Study of the combination of luspatercept in low-risk myelodysplastic syndrom (LR-MDS) without ring sideroblasts (RS) having failed or being ineligible to ESA

Ages 18 Years+41 locations
View full study
ACTIVE NOT RECRUITINGPHASE1Recently updatedNCT02992522

Obinutuzumab, Venetoclax, and Lenalidomide in Treating Patients With Relapsed or Refractory B-cell Non-Hodgkin Lymphoma

Intervention: Lenalidomide, Obinutuzumab, Venetoclax

B-Cell Lymphoma, UnspecifiedGrade 1 Follicular LymphomaGrade 2 Follicular LymphomaGrade 3a Follicular LymphomaRecurrent Burkitt Lymphoma

This phase I study studies the side effects and best dose of venetoclax and lenalidomide when given together with obinutuzumab in treating patients with B-cell non-Hodgkin lymphoma that has returned after a period of improvement or not responding to treatment. Monoclonal antibodies, such as obinutuzumab, may interfere with the ability of cancer cells to grow and spread. Venetoclax may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as lenalidomide, 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 obinutuzumab, venetoclax, and lenalidomide may work better in treating patients with B-cell non-Hodgkin lymphoma.

Ages 18 Years+2 locations
View full study
RECRUITINGPHASE2Recently updatedNCT07680764

A Study To Assess the Safety, Tolerability, and Efficacy of Imzokitug in Combination With Pumitamig and Platinum-Doublet Chemotherapy (PDCT) Versus Pumitamig and PDCT as First-line Treatment for Participants With Locally Advanced or Metastatic Non-Small Cell Lung Cancer (NSCLC)

Intervention: Pumitamig, Imzokitug, Pemetrexed, Cisplatin, Carboplatin, Paclitaxel, Infliximab, Vedolizumab

Locally Advanced Non-Small Cell Lung Cancer (NSCLC)Metastatic Non-Small Cell Lung Cancer (NSCLC)

The purpose of this study is to assess the safety, tolerability, and efficacy of Imzokitug in combination with Pumitamig and Platinum-Doublet Chemotherapy (PDCT) versus Pumitamig and PDCT as first-line treatment for participants with Locally Advanced or Metastatic Non-Small Cell Lung Cancer (NSCLC)

Ages 18 Years+71 locations
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ACTIVE NOT RECRUITINGPHASE4Recently updatedNCT06112743

A Study to Evaluate Mavacamten Impact on Myocardial Structure in Participants With Symptomatic Obstructive Hypertrophic Cardiomyopathy

Intervention: Mavacamten

Cardiomyopathy, Hypertrophic

The purpose of this study is to evaluate the mavacamten impact on myocardial structure with cardiac magnetic resonance imaging (CMR) in adult participants with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) \[New York Heart Association (NYHA) Functional Class II or III\].

Ages 18 Years+23 locations
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ACTIVE NOT RECRUITINGPHASE2Recently updatedNCT05664737

A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia

Intervention: Luspatercept, Placebo

Anemia

The purpose of the study is to evaluate the efficacy and safety of luspatercept plus best supportive care (BSC) vs placebo plus BSC on anemia in adult participants with α-thalassemia hemoglobin H (HbH) disease and determine the safety and drug levels in adolescent participants.

Ages 12 Years+31 locations
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RECRUITINGPHASE2Recently updatedNCT06782490

A Study to Evaluate the Efficacy, Safety and Tolerability of BMS-986368 in Participants With Multiple Sclerosis Spasticity

Intervention: BMS-986368, Placebo

The purpose of this study is to evaluate the efficacy, safety, and tolerability of BMS-986368 in participants with Multiple Sclerosis Spasticity

Ages 18 Years - 70 Years55 locations
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RECRUITINGPHASE1, PHASE2Recently updatedNCT06481306

A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease

Intervention: BMS-986470, Placebo, Famotidine, Pantoprazole

Anemia, Sickle CellHealthy Volunteers

The purpose of this study is to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of BMS-986470 in healthy volunteers and participants with sickle cell disease.

Ages 18 Years+32 locations
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Bristol Myers Squibb FDA-Approved Drugs (7)

Medications developed or marketed by Bristol Myers Squibb 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
ENASIDENIB MESYLATEIdhifa
oral
Aug 1, 2017
FEDRATINIB HYDROCHLORIDEInrebic
oral
Aug 16, 2019
LUSPATERCEPT
Erythroid Maturation Agent [EPC]
Reblozyl
subcutaneous
Nov 8, 2019
LENALIDOMIDE
Thalidomide Analog [EPC]
Revlimid
oral
Dec 27, 2005
THALIDOMIDEThalomid
oral
Jul 16, 1998

Bristol Myers Squibb Trial Locations

Bristol Myers Squibb clinical trials are running at 1,729 sites in 47 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
335▼
China
163▼
France
128▼
Japan
127▼
Germany
118▼
Italy
101▼
Spain
91▼
Canada
60▼
Australia
59▼
India
48▼
United Kingdom
45▼
South Korea
41▼

Rare Disease Focus Areas (37)

Diseases targeted by Bristol Myers Squibb's clinical trial and drug development programs

Adrenocortical CarcinomaRare Cancers

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. These glands produce essential hormones including cor...

Prevalence: About 1-2 per million people per year; approximately 600 new cases per year in the U.S.
Aplastic AnemiaBlood & Immune

Aplastic anemia is a rare bone marrow failure syndrome characterized by pancytopenia resulting from absent or severely reduced hematopoiesis. Approximately half are immune-mediated, while others resul...

Prevalence: 1-2 cases per 1 million people per year
CholangiocarcinomaRare Cancers

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. It can occur inside the liver (...

Prevalence: About 8,000 new cases per year in the U.S.; rising incidence worldwide
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.
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
DermatomyositisAutoimmune & Inflammatory

Dermatomyositis is a rare autoimmune inflammatory disease causing muscle weakness and distinctive skin rashes, particularly over joints. It involves inflammation of muscles and skin blood vessels. Abo...

Prevalence: Approximately 1-10 cases per million people; juvenile-onset incidence about 0.4 cases per 100,000 children

Patient Resources

Organizations and resources related to Bristol Myers Squibb's rare disease focus areas

Frequently Asked Questions About Bristol Myers Squibb

Common questions about Bristol Myers Squibb's rare disease programs, clinical trials, and treatments.