Bristol Myers Squibb
Bristol Myers Squibb works on 38 rare diseases tracked on Trial Friend, including Adrenocortical Carcinoma, Aplastic Anemia, Cholangiocarcinoma and 35 more, with 63 active clinical trials (23 recruiting) and 10 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.
Focus areas at Bristol Myers Squibb
Within its broader pharmaceutical portfolio, Bristol Myers Squibb has active clinical trial programs and drug development efforts across 38 rare diseases, including Adrenocortical Carcinoma, Aplastic Anemia, Cholangiocarcinoma, Chronic Graft-versus-Host Disease, Common Variable Immunodeficiency, and 33 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 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 63 active clinical trials across 5 development stages, with 23 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.
Bristol Myers Squibb Clinical Trials (63)
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.
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Bristol Myers Squibb FDA-Approved Drugs (10)
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 Name | Brand Name | Rare Diseases | Approval Date |
|---|---|---|---|
| REPOTRECTINIB | AUGTYRO | Nov 15, 2023 | |
| MAVACAMTEN | CAMZYOS | Apr 28, 2022 | |
| FEDRATINIB HYDROCHLORIDE | Inrebic oral | Aug 16, 2019 | |
| POMALIDOMIDE Thalidomide Analog [EPC] | Pomalyst oral | Feb 8, 2013 | |
| LUSPATERCEPT Erythroid Maturation Agent [EPC] | Reblozyl subcutaneous | Nov 8, 2019 |
Bristol Myers Squibb Trial Locations
Bristol Myers Squibb clinical trials are running at 2,390 sites in 49 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 (38)
Diseases targeted by Bristol Myers Squibb's clinical trial and drug development programs
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...
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...
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 (...
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...
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...
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.