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Alexion Pharmaceuticals

Rare disease drugmaker · part of AstraZeneca · based in Boston, United States · founded 1992 · alexion.com

Alexion Pharmaceuticals works on 35 rare diseases we track, including Acromegaly, Amyotrophic Lateral Sclerosis and 33 more. It has 37 active clinical trials, and 18 are looking for volunteers now. It also has 5 FDA-approved rare disease drugs.

Alexion, now AstraZeneca's Rare Disease division following a $39 billion acquisition in 2021, is the pioneer of complement-targeted therapies. Key products include Soliris (eculizumab) and its successor Ultomiris (ravulizumab) for paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome, generalized myasthenia gravis, and neuromyelitis optica spectrum disorder.

Alexion Pharmaceuticals pipeline

Each row lists the diseases Alexion Pharmaceuticals 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.

Myasthenia Gravis 4 trials recruiting · Neuromyelitis Optica 3 trials recruiting · IgA Nephropathy 2 trials recruiting · Paroxysmal Nocturnal Hemoglobinuria 2 trials recruiting · Hypophosphatasia 3 trials · Light Chain Amyloidosis 2 trials · Wild-Type ATTR Cardiac Amyloidosis · Hypoparathyroidism

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

Dilated Cardiomyopathy recruiting · Light Chain Amyloidosis recruiting · Membranous Nephropathy recruiting · Amyloid Transthyretin Cardiomyopathy

After approval
Studies that follow people already taking an approved drug.

Atypical Hemolytic Uremic Syndrome recruiting · Myasthenia Gravis recruiting · Transthyretin-type Cardiac Amyloidosis recruiting

Other studies
Studies with no phase listed, such as registries and natural history studies.

Alexion Pharmaceuticals clinical trials

These are Alexion Pharmaceuticals 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.

18 trials looking for volunteers now

Looking for volunteersPhase 3Recently updatedNCT07596784

Efficacy and Safety of Ravulizumab in Chinese Adults Participants With Generalized Myasthenia Gravis (gMG)

Tests Ravulizumab

For Generalized Myasthenia Gravis and gMG

The primary purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of ravulizumab in Chinese adult participants with Acetylcholine receptor (AChR) + Generalized Myasthenia Gravis (gMG).

Ages 18 Years - 130 Years10 locations
View full study
Looking for volunteersRecently updatedNCT06398158

Study of the Clinical and Radiological Impact of Ravulizumab in People With Neuromyelitis Optica Spectrum Disorder

Tests Ravulizumab

For Neuromyelitis Optica

This is an observational study to: * evaluate the on-treatment clinical performance of ravulizumab in relation to the pre-treatment time period (time period prior to exposure), * enhance knowledge regarding conventional MRI outcomes in people with NMOSD treated with ravulizumab, * identify factors suggestive of subclinical disease progression through conventional MRI sequences, * determine if treatment with ravulizumab, impacts longitudinal 3D conformational MRI measures at the dorsal medulla and other regions of the CNS, and * identify biomarkers (e.g., serum neurofilament light chain (sNfL), conventional and novel MRI markers, etc.) related to disease activity.

Ages 18 Years+1 location
View full study
Looking for volunteersRecently updatedNCT05966467

Registry of Patients With AQP4+ NMOSD Treated With Alexion C5 Inhibitor Therapies

For Neuromyelitis Optica Spectrum Disorder, NMOSD and AQP4+ NMOSD

Long-term, multicenter, multinational, observational, registry of patients with AQP4+ NMOSD that is designed to collect data on clinical outcomes and safety in patients prescribed Alexion C5 inhibitor therapies (C5IT). The registry will also collect data on patient reported outcomes (PROs), quality of life (QoL), and targeted AQP4+ NMOSD therapies used to provide evidence on the real-world impact of ALXN-C5IT on patients with AQP4+ NMOSD.

Ages 18 Years+33 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT07557420

Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity Study of Ravulizumab in Chinese Adults With Neuromyelitis Optica Spectrum Disorder (NMOSD)

Tests Ravulizumab

For NMOSD and Neuromyelitis Optica Spectrum Disorder

The primary objective of this study is to confirm the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of ravulizumab in the treatment of Chinese adults with anti-aquaporin-4 (AQP4) antibody (Ab) + neuromyelitis optica spectrum disorder (NMOSD).

Ages 18 Years - 130 Years10 locations
View full study
Looking for volunteersPhase 1/2Recently updatedNCT07218887

ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy

Tests ALXN2350

For BAG3 Mutation Associated Dilated Cardiomyopathy

This Phase 1/2 study is an open-label, dose finding and dose expansion study investigating the safety, tolerability, and efficacy of a single IV infusion of ALXN2350 in adult participants with BAG3 associated DCM.

Ages 18 Years - 70 Years11 locations
View full study
Looking for volunteersPhase 3Updated a few months agoNCT07024563

Study of Ravulizumab in Pediatric Participants With Primary IgAN

Tests Ravulizumab

For IgAN, IgAVN, Immunoglobulin A Nephropathy, Immunoglobulin A Vasculitis Associated Nephritis and Henoch-schonlein Purpura Nephritis

The primary objectives of this study are to characterize ravulizumab pharmacokinetics (PK) and pharmacodynamics (PD), and to evaluate safety and efficacy following ravulizumab IV dosing in pediatric participants with IgAN or IgAVN.

Ages 2 Years - 18 Years15 locations
View full study
Looking for volunteersPhase 1/2Updated a few months agoNCT07081646

A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis.

Tests AZD0120

For Relapsed AL Amyloidosis, Refractory AL Amyloidosis, Light Chain Amyloidosis and Amyloidosis

Open-label Phase 1b/2 study with primary objective of this study is to evaluate the safety, tolerability and efficacy of AZD0120 in participants with light chain (AL) amyloidosis.

Ages 18 Years+18 locations
View full study
Looking for volunteersPhase 2Updated a few months agoNCT07157787

Study of ALXN1920 in Adult Participants With Primary Membranous Nephropathy (PMN)

Tests ALXN1920, Placebo

For Primary Membranous Nephropathy

The primary objective of this study is to evaluate the efficacy of ALXN1920 compared with placebo in participants with PMN who are at a high risk for disease progression using 24-hour urine protein creatinine ratio (UPCR).

Ages 18 Years - 75 Years40 locations
View full study
Ask about Alexion Pharmaceuticals's trials
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FDA-approved drugs from Alexion Pharmaceuticals

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

Koselugo selumetinib

For Neurofibromatosis Type 1. Approved Apr 10, 2020.

Soliris eculizumab

For Myasthenia Gravis, Paroxysmal Nocturnal Hemoglobinuria, Atypical Hemolytic Uremic Syndrome and Neuromyelitis Optica. Approved Mar 16, 2007. Taken by intravenous route.

Strensiq asfotase alfa

For Hypophosphatasia. Approved Oct 23, 2015. Taken by subcutaneous route.

Voydeya danicopan

For Paroxysmal Nocturnal Hemoglobinuria. Approved Mar 29, 2024. Taken by mouth.

More about Alexion Pharmaceuticals

Voydeya (danicopan), the first oral Factor D inhibitor, was FDA-approved in March 2024 as add-on therapy to ravulizumab or eculizumab for clinically significant extravascular hemolysis in adults with PNH. The portfolio also includes Strensiq (asfotase alfa) for hypophosphatasia and Kanuma (sebelipase alfa) for lysosomal acid lipase deficiency.

Where Alexion Pharmaceuticals trials take place

The trials above run at 1,499 study sites in 40 countries. Tap a country to see its states, cities and hospitals.

United States
308▼
China
143▼
France
114▼
Italy
109▼
Japan
106▼
Spain
86▼
South Korea
64▼
Germany
61▼
Brazil
59▼
United Kingdom
53▼
Canada
49▼
Australia
44▼

Rare diseases Alexion Pharmaceuticals works on

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

Acromegaly

Acromegaly is a rare hormonal disorder caused by excessive growth hormone production, typically from a pituitary adenoma, resulting in abnormal growth of hands, feet, and facial features.

How common: About 50 to 70 people per million, according to NORD, with 3 to 11 new cases per million people each year

Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis is a progressive neurodegenerative disease destroying motor neurons in the brain and spinal cord.

How common: About 5,000 new cases per year in the U.S.; CDC's National ALS Registry estimates about 33,000 Americans were living with ALS in 2022, a number projected to pass 36,000 by 2030

ATTR Amyloidosis (Transthyretin Amyloidosis)

ATTR amyloidosis is a rare progressive disease where abnormal transthyretin protein accumulates as amyloid deposits in the heart, nerves, and other organs.

How common: Approximately 5,000 to 7,000 new cases diagnosed annually in the U.S

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

Bullous Pemphigoid

Bullous pemphigoid is an autoimmune blistering disorder caused by autoantibodies against basement membrane proteins, particularly BP180 and BP230.

How common: Approximately 2-23 per million people per year; incidence increases sharply with age

C3 Glomerulopathy

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.

How common: 1 to 2 new cases per million per year; approximately 5,000 to 10,000 affected individuals in the U.S

Cold Agglutinin Disease

Cold agglutinin disease is a rare autoimmune hemolytic anemia caused by IgM antibodies that stick to red blood cells in the cold.

How common: About 16 per million people for primary CAD in a Norwegian population study, roughly 1 in 60,000, with about 1 new case per million people each year

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

Factor V Deficiency

Factor V deficiency is a rare inherited bleeding disorder caused by deficiency or dysfunction of coagulation factor V. The condition leads to prolonged bleeding from minor injuries and spontaneous...

How common: Fewer than 1 in 1,000,000 people

Geographic Atrophy

Geographic atrophy is the advanced form of dry age-related macular degeneration, characterized by progressive loss of retinal pigment epithelium, photoreceptors, and choriocapillaris in well-defined...

How common: About 1 million people in the U.S. are estimated to have geographic atrophy, the advanced dry form of age-related macular degeneration

Guillain-Barré Syndrome

Guillain-Barré syndrome is a rare autoimmune neurological disorder in which the body's immune system attacks the peripheral nerves, causing rapidly progressive muscle weakness that typically starts...

How common: 1 in 60,000 people per year in the U.S. (3,000 to 6,000 new cases annually)

Hodgkin Lymphoma

Hodgkin lymphoma is a cancer of the lymphatic system characterized by the presence of abnormal Reed-Sternberg cells.

How common: About 8,920 new cases expected in the U.S. in 2026 (American Cancer Society); approximately 2.6 per 100,000 people

Hunter Syndrome

Hunter syndrome is an X-linked lysosomal storage disorder caused by deficiency of iduronate-2-sulfatase enzyme, leading to accumulation of glycosaminoglycans in cells and tissues.

How common: 1 in 100,000 to 150,000 males; females with disease are rare

Hypoparathyroidism

Hypoparathyroidism is an endocrine disorder caused by insufficient production of parathyroid hormone (PTH), resulting in chronically low calcium and elevated phosphorus levels in the blood.

How common: Approximately 22 per 100,000 for postsurgical forms; estimated 70,000 to 77,000 people living with chronic hypoparathyroidism in the United States

Hypophosphatasia

Hypophosphatasia (HPP) is a rare inherited disorder in which the body makes too little of an enzyme called alkaline phosphatase, so bones and teeth do not harden properly.

How common: Severe HPP that shows up at birth or in infancy affects about 1 in 100,000 live births in Canada, the figure NORD and Soft Bones both use for North America

IgA Nephropathy

IgA nephropathy is the most common primary glomerulonephritis worldwide, caused by abnormal IgA1 antibodies depositing in the kidney's mesangium, activating complement pathways and triggering...

How common: About 2 new cases per 100,000 people each year in the U.S., and an estimated 199,000 to 208,000 Americans were living with IgA nephropathy in 2021 (Otsuka-funded study, Clinical Nephrology, 2025)

IgA Vasculitis with Nephritis

IgA vasculitis is a small-vessel vasculitis characterized by IgA immune complex deposition in kidneys and other organs.

How common: About 3 to 27 new cases per 100,000 children each year; the most common vasculitis of childhood

Light Chain Amyloidosis

Light Chain Amyloidosis is a rare, serious blood disorder caused by plasma cells producing misfolded immunoglobulin light chains that accumulate in tissues as amyloid fibrils, progressively damaging...

How common: Approximately 600-1,000 new cases per year in the United States; median age of diagnosis is 63 years

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

Methylmalonic Acidemia

Methylmalonic acidemia is a group of inherited metabolic disorders in which the body cannot properly break down certain proteins and fats, leading to toxic buildup of methylmalonic acid.

How common: 1 in 50,000 to 100,000 live births in the U.S.; identified through newborn screening in all 50 states

Mucopolysaccharidosis Type I

Mucopolysaccharidosis Type I (MPS I) is a lysosomal storage disorder caused by deficiency of the enzyme alpha-L-iduronidase, which breaks down glycosaminoglycans (GAGs).

How common: Approximately 1 per 100,000 live births; Hurler form is the most common phenotype

Sanfilippo Syndrome

Sanfilippo syndrome, formally mucopolysaccharidosis type III (MPS III), is a lysosomal storage disorder in which the body cannot break down a sugar chain called heparan sulfate.

How common: Approximately 1 per 70,000 to 1 per 100,000 live births; highest prevalence in certain populations including Australia and Scandinavia

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)

Myasthenia Gravis

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

How common: 1 in 5,000 people

Neurofibromatosis Type 1

Neurofibromatosis type 1 is a genetic disorder caused by mutations in the NF1 gene on chromosome 17, which encodes neurofibromin, a tumor suppressor protein.

How common: 1 in 2,500 to 3,500 people in the U.S., making it one of the most common genetic conditions

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

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

Pemphigus Vulgaris

Pemphigus Vulgaris is a rare, potentially life-threatening autoimmune blistering disorder where the body produces antibodies against cell-to-cell adhesion molecules, causing blisters and erosions in...

How common: Approximately 0.5-3.2 cases per 100,000 people; higher incidence in certain ethnic groups (Ashkenazi Jews, Mediterranean, Indian populations)

Peripartum Cardiomyopathy

Peripartum cardiomyopathy is a rare form of heart failure that develops during the last month of pregnancy or within 5 months after delivery in women with no prior history of heart disease.

How common: About 1 in 1,000 to 1 in 4,000 pregnancies in the U.S.; significantly more common in women of African descent

Sickle Cell Disease

Sickle cell disease is an inherited blood disorder where hemoglobin polymerizes under low oxygen, causing red blood cells to become rigid, sickle-shaped, and prone to hemolysis and vaso-occlusion.

How common: About 100,000 Americans with SCD; about 1 in 365 Black or African American births and 1 in 16,300 Hispanic American births

Warm Autoimmune Hemolytic Anemia

Warm autoimmune hemolytic anemia (wAIHA) is a rare autoimmune disease in which the immune system makes antibodies, usually of the IgG type, that stick to red blood cells at normal body temperature...

How common: About 1 to 3 new cases per 100,000 people each year, according to the FDA and NORD

Wild-Type ATTR Cardiac Amyloidosis

Wild-type ATTR cardiac amyloidosis is an age-related disease where normal transthyretin protein spontaneously misfolds and accumulates in the heart, causing progressive heart failure.

How common: Estimated to affect up to 13% of males aged 85 and older; substantially underdiagnosed

Wilson Disease

Wilson disease is a rare inherited disorder of copper metabolism caused by mutations in the ATP7B gene.

How common: Clinically diagnosed prevalence is about 1 in 30,000 people worldwide

Where to get support

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

Questions about Alexion Pharmaceuticals