Amgen Inc.
Amgen Inc. works on 42 rare diseases tracked on Trial Friend, including Aplastic Anemia, Atypical Hemolytic Uremic Syndrome, Autoimmune Hepatitis and 39 more, with 2 active clinical trials (1 recruiting) and 10 FDA-approved rare disease drugs.
Amgen is a multinational biopharmaceutical company with growing focus on rare diseases, strengthened by the 2023 acquisition of Horizon Therapeutics. The company develops therapies for rare inflammatory diseases, genetic disorders, and severe unmet medical conditions.
Focus areas at Amgen Inc.
Within its broader pharmaceutical portfolio, Amgen Inc. has active clinical trial programs and drug development efforts across 42 rare diseases, including Aplastic Anemia, Atypical Hemolytic Uremic Syndrome, Autoimmune Hepatitis, Behçet Disease, C3 Glomerulopathy, and 37 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 Amgen Inc., 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.
Amgen Inc. is headquartered in Thousand Oaks, United States, founded in 1980, publicly traded under the ticker symbol AMGN. 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.
Amgen Inc. Drug Pipeline
Amgen Inc. has 2 active clinical trials across 2 development stages, with 1 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.
Amgen Inc. Clinical Trials (2)
Active and recruiting clinical trials sponsored by Amgen Inc., 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.
Pick a condition. One email when a new trial opens, a trial changes or the FDA decides, from any company, not only Amgen Inc.. Unsubscribe anytime.
You'll get new recruiting trials, changes to trials, FDA decisions and condition news for Aplastic Anemia. Turn any of these off from the link in every email.
Amgen Inc. FDA-Approved Drugs (10)
Medications developed or marketed by Amgen Inc. 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 |
|---|---|---|---|
| INTERFERON GAMMA-1B | ACTIMMUNE | Feb 25, 1999 | |
| ECULIZUMAB-AEEB Complement Inhibitor [EPC] | BKEMV intravenous | May 28, 2024 | |
| IVABRADINE | Corlanor oral | Apr 15, 2015 | |
| ROMIPLOSTIM Thrombopoietin Receptor Agonist [EPC] | Nplate subcutaneous | Aug 22, 2008 | |
| APREMILAST Phosphodiesterase 4 Inhibitor [EPC] | Otezla oral | Mar 21, 2014 |
Amgen Inc. Trial Locations
Amgen Inc. clinical trials are running at 11 sites in 2 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 (42)
Diseases targeted by Amgen Inc.'s clinical trial and drug development programs
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...
Atypical Hemolytic Uremic Syndrome (aHUS) is a rare kidney disease caused by uncontrolled activation of the complement system, a part of the immune system that normally helps fight infection. This act...
Autoimmune Hepatitis (AIH) is a chronic liver disease characterized by persistent inflammation and progressive fibrosis of the liver due to loss of immune tolerance to hepatocyte antigens. The immune ...
Behçet Disease is a rare, chronic, recurrent vasculitis causing painful oral and genital ulcers, eye inflammation, and skin lesions. It can affect multiple organ systems including the brain, heart, an...
C3 glomerulopathy is a group of rare kidney diseases caused by dysregulation of the alternative complement pathway, leading to uncontrolled C3 deposition in the glomeruli. The two subtypes, C3 glomeru...
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...
Patient Resources
Organizations and resources related to Amgen Inc.'s rare disease focus areas
Frequently Asked Questions About Amgen Inc.
Common questions about Amgen Inc.'s rare disease programs, clinical trials, and treatments.