Alexion Pharmaceuticals
Trial Pipeline
ALXN2420 Versus Placebo in Combination With Somatostatin Analogs in Participants With Acromegaly
NCT07037420
ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy
NCT07218887
Study of ALXN1920 in Adult Participants With Primary Membranous Nephropathy (PMN)
NCT07157787
A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis.
NCT07081646
Study of Ravulizumab in Pediatric Participants With Primary IgAN
NCT07024563
Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab Administered Intravenously in Adult Participants at High Risk of Delayed Graft Function After Kidney Transplantation
NCT06830798
Phase 2 Study of ALXN2030 in Patients With Antibody-Mediated Rejection After Kidney Transplantation
NCT06744647
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
NCT06312644
PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
NCT06607627
Study of Ravulizumab in Immunoglobulin A Nephropathy (IgAN)
NCT06291376
Registry of Patients With AQP4+ NMOSD Treated With Alexion C5 Inhibitor Therapies
NCT05966467
A Prospective Sub-Study of the Global Hypophosphatasia Registry
NCT05234567
Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD
NCT05346354
Registry of Participants With Generalized Myasthenia Gravis Treated With Alexion C5 Inhibition Therapies (C5ITs)
NCT04202341
Lysosomal Acid Lipase (LAL) Deficiency Registry
NCT01633489
Atypical Hemolytic-Uremic Syndrome (aHUS) Registry
NCT01522183
Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data
NCT07413250
Long-term Safety of Danicopan: IPIG Registry-based Cohort Study
NCT07413679
Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP
NCT06079359
Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa
NCT06079372
Study of ALXN2220 Versus Placebo in Adults With ATTR-CM
NCT06183931
Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa
NCT06079281
Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)
NCT05644561
Evaluation of the Safety and Efficacy of Eneboparatide (AZP-3601) in Patients With Chronic Hypoparathyroidism
NCT05778071
ARTEMIS: Ravulizumab to Protect Patients With CKD From CSA-AKI and MAKE
NCT05746559
Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis
NCT05556096
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
NCT05389449
A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)
NCT05981092
Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy
NCT05506254
Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant
NCT04543591
A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)
NCT04512235
A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES)
NCT04504825
Drug-Drug Interaction (DDI) Study of ALXN2050 in Healthy Adult Participants
NCT05202145
A Study to Assess ACH-0145228 When Administered as Immediate Release Tablet Versus Powder-In-Capsule in Healthy Adult Participants
NCT04551586
Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
NCT03748823
A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria
NCT03406507
Study of Danicopan in Participants With Normal Kidney Function and Participants With Kidney Dysfunction
NCT04935294
ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
NCT03056040
ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT02946463
The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment
NCT02763215
Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)
NCT02324049
Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency
NCT02112994
A Study of ALXN1840 (Non-coated) Administered With And Without Omeprazole In Healthy Adults
NCT05319899
Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency
NCT01757184
A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)
NCT01419028
Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)
NCT01205152
An Open-Label Treatment Protocol to Provide Continued Elvucitabine Treatment
NCT00675844
Elvucitabine/Efavirenz/Tenofovir Versus Lamivudine/Efavirenz/Tenofovir in Human Immunodeficiency Virus (HIV)-1 Infected, Treatment-naive Participants
NCT00350272
Study of Once Daily Elvucitabine Versus Lamivudine in Participants With a Documented M184V Mutation
NCT00312039
Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT00122317
Pexelizumab in Patients Undergoing Coronary Artery Bypass Grafting With Cardiopulmonary Bypass (PRIMO-CABG II)
NCT00088179
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 8 |
| Phase 2 | 8 |
| Phase 3 | 23 |
What the Pipeline for Alexion Pharmaceuticals Shows
According to the ClinicalTrials.gov registry, Alexion Pharmaceuticals is linked to 109 US clinical trials across every stage of research activity. Of those, 74 studies are currently recruiting — about 68% of the sponsor's indexed portfolio — and 19 are already marked complete, representing roughly 17% of the total. Recruiting share is one of the more practical signals here: it reflects how much of a sponsor's research is presently open to new participants, while the completed share indicates the depth of finished work that has already contributed registry results. Both counts come directly from the public ClinicalTrials.gov dataset and are refreshed on the registry side; this page mirrors the latest data pull without altering it.
The phase mix for Alexion Pharmaceuticals reports 23 late-stage studies (Phase 3 and Phase 4 combined) and 16 earlier-phase studies (Phase 1 and Phase 2). A portfolio weighted toward Phase 3 usually reflects an organization advancing candidates toward regulatory review, where the research centers on comparative efficacy and broader safety across larger populations. A heavier Phase 1 and Phase 2 tilt generally indicates exploratory work — safety, dosing, and early signal detection — and is common among research-forward sponsors that seed many early programs. Phase 4 entries, when present, track interventions already in real-world use and typically focus on long-term safety, effectiveness across subgroups, or formulation comparisons.
The top therapeutic focus area indexed for Alexion Pharmaceuticals is Paroxysmal Nocturnal Hemoglobinuria with 5 linked trials, and 9 other condition areas appear in the top list above. That distribution is a quick read of where the organization concentrates its research attention; it does not imply product availability, market share, or any clinical endorsement. All numbers on this page come from ClinicalTrials.gov maintained by the National Library of Medicine, and counts can shift as new studies are registered or existing ones update their status. This information is provided for reference and educational purposes only, not as medical, investment, or regulatory advice — verify current details directly with ClinicalTrials.gov before relying on any figure here.
Read our methodology — how this data is sourced, computed, and verified.