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Amgen Inc.

Drugmaker with rare disease work · based in Thousand Oaks, United States · founded 1980 · ticker AMGN · amgen.com

Amgen Inc. works on 24 rare diseases we track, including Acute Myeloid Leukemia, Atypical Hemolytic Uremic Syndrome and 22 more. It has 33 active clinical trials, and 16 are looking for volunteers now. It also has 12 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.

Amgen Inc. pipeline

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

Approved
Drugs the FDA has approved.

Actimmune (interferon gamma-1b) · Bkemv (eculizumab-aeeb) · Corlanor (ivabradine) · Kyprolis (carfilzomib) · Nplate (romiplostim) · Otezla (apremilast) · Procysbi (cysteamine bitartrate delayed-release) · Repatha (evolocumab) · Riabni (rituximab-arrx) · Tepezza (teprotumumab) · 2 more

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

Behçet Disease 2 trials recruiting · Osteogenesis Imperfecta 2 trials recruiting · Sjögren's Syndrome 2 trials recruiting · Neovascular Age-related Macular Degeneration recruiting · Autoimmune Hepatitis recruiting · Immune Thrombocytopenia recruiting · Granulomatosis with Polyangiitis recruiting · Obstructive Sleep Apnea · Multiple Sclerosis · Multiple Myeloma · and 3 more

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

Multiple Myeloma 7 trials recruiting · Thyroid Eye Disease 2 trials recruiting · Medullary Thyroid Cancer recruiting · Acute Myeloid Leukemia recruiting · Waldenström Macroglobulinemia

Phase 1
First studies in people, mostly checking safety.
After approval
Studies that follow people already taking an approved drug.
Other studies
Studies with no phase listed, such as registries and natural history studies.

Neuromyelitis Optica recruiting · Immune Thrombocytopenia recruiting · Ankylosing Spondylitis (AS) / Radiographic Axial SpA (r-axSpA) recruiting

Amgen Inc. clinical trials

These are Amgen Inc. 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.

16 trials looking for volunteers now

Looking for volunteersPhase 3Recently updatedNCT07614776

A Study to Evaluate Efficacy, Safety, and Immunogenicity With ABP 938 8 mg Versus EYLEA® HD (Aflibercept) in Participants With Neovascular Age-related Macular Degeneration

Tests ABP 938 8 mg, Aflibercept (US) 8 mg

For Neovascular Age-related Macular Degeneration and nAMD

The aim of this trial is to demonstrate similarity in efficacy between ABP 938 8 mg and aflibercept (US) 8 mg by evaluating the change in best corrected visual acuity (BCVA) in participants with neovascular age-related macular degeneration (nAMD)

Ages 50 Years+101 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT05767047

A Study of Apremilast in Children With Oral Ulcers Associated With Behçet's Disease or Juvenile Psoriatic Arthritis

Tests Apremilast

For Behçet's Disease and Juvenile Psoriatic Arthritis

The primary objective of this study is to evaluate the long-term safety of apremilast in subjects 2 years of age or older with oral ulcers associated with Behçets disease or 5 years of age or older with active juvenile psoriatic arthritis that have completed Study 20190530 or Study 20190529.

Ages 5 Years - 18 Years7 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT07598825

A Trial of Inebilizumab in Participants With Autoimmune Hepatitis

Tests Inebilizumab, Placebo, Standard of Care

For Autoimmune Hepatitis and AIH

The main objectives of this trial are to evaluate the safety and tolerability of inebilizumab in participants with autoimmune hepatitis (AIH) (Part 1) and to evaluate the efficacy of inebilizumab on AIH disease activity and glucocorticoid (GC) use in the management of AIH (Part 2).

Ages 18 Years - 74 Years36 locations
View full study
Looking for volunteersRecently updatedNCT05909761

Observational Safety Study in Women With Neuromyelitis Optica Spectrum Disorder (NMOSD) Exposed to UPLIZNA® During Pregnancy

Tests UPLIZNA

For Neuromyelitis Optica Spectrum Disorder and Pregnancy Related

This is an observational study to monitor female participants exposed to UPLIZNA during pregnancy. This study requires voluntary reporting of pregnancies in female participants with NMOSD exposed to UPLIZNA during pregnancy or within 6 months preceding conception. Pregnancy-related data, potential confounding factors and information related to pregnancy outcome will be collected. The schedule of office visits and all treatment regimens will be determined by the treating healthcare provider. Duration of the study is 10 years, at minimum.

Ages 15 Years+1 location
View full study
Looking for volunteersPhase 1/2Recently updatedNCT06814496

Radiation Combined With BIspecific T-Cell Engager in DLL3 Expressing Tumors

Tests Tarlatamab, Concurrent Radiation Therapy, Sequential Radiation therapy

For Melanoma, Medullary Thyroid Cancer, Sinonasal Undifferentiated Carcinoma, Esthesioneuroblastoma and Bladder Cancer

Phase I study to examine safety of the addition of concurrent tarlatamab with standard palliative and consolidative RT regimens , with a main cohort of N=20-24 patients with extracranial anatomic radiation sites. I) After lead in of 10 patients demonstrating safety of treatment, allow for expansion to cranial sites of disease (N=6-10) with continued enrollment in main cohort II) If toxicity criteria is not met in concurrent RT tarlatamab cohort, we will continue with sequential RT, either A) delivered within 7 days prior to cycle 1 day 1, or B) delivered during cycle 1 -2 but with pre- and post-RT washout of 7 days with no drug during RT, to examine safety in a temporally spaced setting. III) If sequential tarlatamab and radiation is not deemed safe, we would allow for continued enrollment to assess efficacy of drug sans radiation treatment, enriching for tumors not of small cell lung cancer histology and allowing for patients without sites amenable to RT. A nested phase II study will attempt to assess for ORR and safety of study intervention amongst tumors not of small cell lung cancer histology.

Ages 18 Years - 99 Years3 locations
View full study
Looking for volunteersPhase 2Recently updatedNCT05789303

Study of Belantamab Mafodotin With Carfilzomib, Pomalidomide, and Dexamethasone in Relapsed Multiple Myeloma

Tests Belantamab mafodotin, Carfilzomib, Pomalidomide, Dexamethasone

For Multiple Myeloma and Relapse Multiple Myeloma

Doctors leading this study hope to learn if the combination of belantamab mafodotin, carfilzomib, pomalidomide, and dexamethasone is effective and safe when given to people who have multiple myeloma that has gotten worse and is not responding to standard drugs that are used for treating multiple myeloma, including chimeric antigen receptor T-cell therapy. Participation in this research will last about 6 -24 months, but it may be less or more depending on your response to treatment.

Ages 18 Years+2 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT07700238

A Study of Romiplostim Plus Predniso(lo)ne vs. Predniso(lo)ne Alone for the Treatment of Previously Untreated Primary Immune Thrombocytopenia (ITP)

Tests Romiplostim, Predniso(lo)ne

For Primary Immune Thrombocytopenia

This Phase 3 study is designed to evaluate the efficacy and safety of romiplostim in combination with predniso(lo)ne compared with predniso(lo)ne alone in adults with previously untreated Primary Immune Thrombocytopenia (ITP).

Ages 18 Years - 100 Years3 locations
View full study
Looking for volunteersPhase 2/3Recently updatedNCT07176546

TAVNEOS for Otolaryngologic Manifestations of Granulomatosis With Polyangiitis

Tests Avacopan, Placebo

For Granulomatosis With Polyangiitis, Wegener's and GPA

This is a single center double-blind placebo-controlled study. Patients with GPA and active ears, nose, and throat (ENT) disease in at least two ENT domains, as defined after endoscopic visualization of the upper airway and audiometric evaluation, if applicable, by a single otolaryngologist using a validated GPA ENT disease activity score, will be eligible for inclusion. Patients will be treated with standard of care (SOC) treatment as determined by their treating rheumatologist. In addition to SOC, patients will be randomized to receive TAVNEOS 30mg BID or placebo. Patients will be followed for 52 weeks with standardized ENT assessment along with rheumatologic evaluation of overall disease activity with BVAS.

Ages 18 Years+2 locations
View full study
Ask about Amgen Inc.'s trials
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FDA-approved drugs from Amgen Inc.

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

Actimmune interferon gamma-1b

For Chronic Granulomatous Disease. Approved Feb 25, 1999.

Bkemv eculizumab-aeeb

For Myasthenia Gravis, Paroxysmal Nocturnal Hemoglobinuria, Atypical Hemolytic Uremic Syndrome and Neuromyelitis Optica. Approved May 28, 2024. Taken by intravenous route.

Corlanor ivabradine

For Dilated Cardiomyopathy. Approved Apr 15, 2015. Taken by mouth.

Kyprolis carfilzomib

For Multiple Myeloma. Approved Jul 20, 2012.

Nplate romiplostim

For Immune Thrombocytopenia. Approved Aug 22, 2008. Taken by subcutaneous route.

Otezla apremilast

For Behçet Disease. Approved Mar 21, 2014. Taken by mouth.

Procysbi cysteamine bitartrate delayed-release

For Cystinosis. Approved Apr 30, 2013.

Repatha evolocumab

For Homozygous Familial Hypercholesterolemia. Approved Feb 26, 2021. Taken by subcutaneous route.

Riabni rituximab-arrx

For Granulomatosis with Polyangiitis and Pemphigus Vulgaris. Approved Dec 17, 2020. Taken by intravenous route.

Tepezza teprotumumab

For Thyroid Eye Disease. Approved Jan 21, 2020.

Uplizna inebilizumab

For Myasthenia Gravis. Approved Jun 11, 2020.

Xgeva denosumab

For Multiple Myeloma. Approved Jun 1, 2010. Taken by subcutaneous route.

More about Amgen Inc.

Within its broader pharmaceutical portfolio, Amgen Inc. has active clinical trial programs and drug development efforts across 24 rare diseases, including Acute Myeloid Leukemia, Atypical Hemolytic Uremic Syndrome, Autoimmune Hepatitis, Behçet Disease, Chronic Granulomatous Disease, and 19 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.

Where Amgen Inc. trials take place

The trials above run at 1,233 study sites in 51 countries. Tap a country to see its states, cities and hospitals.

United States
282▼
Germany
123▼
Japan
98▼
Poland
87▼
Spain
77▼
Italy
57▼
China
46▼
Argentina
38▼
United Kingdom
36▼
France
32▼
Canada
25▼
Turkey (Türkiye)
22▼

Rare diseases Amgen Inc. works on

Each disease page lists every open trial, not only Amgen Inc.'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

Atypical Hemolytic Uremic Syndrome

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.

How common: Approximately 1-2 cases per million people per year; rare form of HUS accounting for 5-10% of all HUS cases

Autoimmune Hepatitis

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.

How common: About 16 per 100,000 people worldwide in a 2023 pooled analysis of 37 studies, with wide differences by region and a rise over time

Behçet Disease

Behçet Disease is a rare, chronic, recurrent vasculitis causing painful oral and genital ulcers, eye inflammation, and skin lesions.

How common: Estimated 3-300 cases per 100,000 depending on geography; highest in Turkey (80-370 per 100,000, according to NORD), common in Middle East and East Asia, rare in North America and Northern Europe

Chronic Granulomatous Disease

Chronic granulomatous disease is a rare inherited immunodeficiency affecting the ability of neutrophils to produce reactive oxygen species.

How common: 1 in 250,000 people; X-linked form (most common) and autosomal recessive variants

Cystinosis

Cystinosis is an inherited condition in which the amino acid cystine builds up inside cells and forms crystals that damage organs, especially the kidneys and eyes.

How common: About 1 in 100,000 to 200,000 newborns worldwide (MedlinePlus Genetics), and about 1 in 26,000 in Brittany, France

Dermatomyositis

Dermatomyositis is a rare autoimmune inflammatory disease causing muscle weakness and distinctive skin rashes, particularly over joints.

How common: About 9.6 new cases per million people a year, according to NORD; juvenile-onset incidence about 0.4 cases per 100,000 children

Dilated Cardiomyopathy

Dilated cardiomyopathy is a condition where the heart's main pumping chamber, the left ventricle, becomes enlarged and weakened, reducing the heart's ability to pump blood efficiently.

How common: Approximately 1 in 250 to 1 in 500 people; accounts for about 30-40% of heart failure cases

Eosinophilic Esophagitis

Eosinophilic Esophagitis is a rare allergic/immune-mediated inflammatory disease of the esophagus characterized by eosinophil infiltration causing progressive narrowing and dysfunction.

How common: About 142.5 per 100,000 people in the United States, about 1 in 700 or an estimated 472,380 people, in a 2025 insurance claims study (Thel et al., Clin Gastroenterol Hepatol 2025)

Granulomatosis with Polyangiitis

Granulomatosis with Polyangiitis is a rare autoimmune disease causing inflammation of small and medium blood vessels in the lungs, kidneys, and upper respiratory tract.

How common: An estimated 3 in 100,000 people in the United States, according to MedlinePlus Genetics

Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia is a severe genetic disorder causing extremely high cholesterol levels from birth, leading to early heart disease and cholesterol deposits in tendons and...

How common: Approximately 1 in 250,000 to 1 in 360,000 people globally

IgG4-Related Disease

IgG4-related disease is a chronic fibroinflammatory condition characterized by tumor-like swelling in affected organs, dense lymphoplasmacytic infiltration rich in IgG4-positive plasma cells...

How common: Approximately 5 in 100,000 people; an estimated 57,000 diagnosed individuals in the U.S. as of 2024

Immune Thrombocytopenia

Immune thrombocytopenia (ITP) is a rare autoimmune disorder in which the immune system destroys platelets, the blood cells that stop bleeding, and slows their production.

How common: Estimated 9 to 20 per 100,000 people; children often have a temporary form that resolves within months

Lupus Nephritis

Lupus nephritis is kidney inflammation caused by systemic lupus erythematosus (SLE), in which the immune system attacks the kidneys' filters and, without treatment, can lead to kidney failure.

How common: About 21 per 100,000 people in a Minnesota population study in 2015 (Lupus Midwest Network, 2022)

Membranous Nephropathy

Membranous nephropathy is a rare autoimmune kidney disease where the body's own antibodies attack proteins in the kidney's filtering units (glomeruli).

How common: About 12 new cases per million people each year in the U.S.; roughly 40,000-50,000 Americans living with the disease

Multiple Myeloma

Multiple myeloma is a cancer of plasma cells, the white blood cells that make antibodies.

How common: About 36,000 new US diagnoses expected in 2026 and roughly 203,000 people living with it, which puts myeloma right at the edge of the US rare disease threshold; it is 1.7% of all new cancers (NCI SEER)

Multiple Sclerosis

Multiple sclerosis is a chronic autoimmune disease where the immune system attacks the protective myelin sheath surrounding nerve fibers in the brain and spinal cord.

How common: Approximately 1 million people in the U.S.; about 1 in 300

Myasthenia Gravis

Myasthenia gravis is a chronic autoimmune neuromuscular disorder causing muscle weakness and rapid fatigue.

How common: 1 in 5,000 people

Neuromyelitis Optica

Neuromyelitis Optica (NMO) is a rare inflammatory autoimmune disorder of the central nervous system characterized by recurrent attacks of optic neuritis (vision loss) and acute myelitis (spinal cord...

How common: About 1 to 2 per 100,000 people worldwide, according to MedlinePlus Genetics

Osteogenesis Imperfecta

Osteogenesis imperfecta is a genetic disorder affecting type I collagen synthesis or structure, causing bones to break easily with little or no apparent trauma.

How common: 1 in 10,000 to 1 in 20,000 people

Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal Nocturnal Hemoglobinuria is a rare, life-threatening blood disorder where complement-mediated destruction of red blood cells occurs due to acquired mutations in the PIGA gene.

How common: Estimated at 1 to 5 per million people (MedlinePlus Genetics); NORD gives 0.5 to 1.5 per million

Sjögren's Syndrome

Sjögren's syndrome is a chronic autoimmune disorder in which the immune system attacks the moisture-producing exocrine glands, primarily the salivary and lacrimal (tear) glands.

How common: Estimated 1 to 4 million Americans (including undiagnosed); approximately 1 in 1,000 adults based on physician-diagnosed prevalence

Thyroid Eye Disease

Thyroid eye disease (TED) is a rare autoimmune condition in which the immune system attacks the muscles and fat behind the eyes, causing bulging eyes, double vision, pain, and in severe cases vision...

How common: About 16 in 100,000 women and 3 in 100,000 men diagnosed each year; up to half of people with Graves' disease develop some degree of TED

Urea Cycle Disorders

Urea Cycle Disorders (UCDs) are a group of genetic conditions affecting the enzymes that convert toxic ammonia to urea for excretion.

How common: About 1 in 35,000 births in the United States, based on newborn screening and registry data (Summar et al., 2013); Ornithine Transcarbamylase (OTC) deficiency is most common form

Where to get support

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

Questions about Amgen Inc.