COMPANY
Alexion Pharmaceuticals, Inc.
FDA-approved therapeutic company Clinical Phase 3
Our research in targeting the complement system focused on paroxysmal nocturnal haemoglobinuria (PNH), a rare and potentially life-threatening blood disorder. From company website Source · Oct 12, 2026
Timeline
- Trial start Phase 3: An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of ALXN2220 in Adults With Transthyretin Amyloid Cardiomyopathy source ↗
- Trial start Phase 3: Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity Study of Ravulizumab in Chinese Adults With Neuromyelitis Optica Spectrum Disorder (NMOSD) source ↗
- Trial start Phase 3: Efficacy and Safety of Ravulizumab in Chinese Adults Participants With Generalized Myasthenia Gravis (gMG) source ↗
- Trial start Phase 1: Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study of ALXN2230 in Healthy Participants source ↗
- Trial start Phase 4: A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab source ↗
- Trial start —: Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data source ↗
- Trial start Phase 4: Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS source ↗
- Trial start Phase 2: Safety and Efficacy of Tarperprumig in Adult Participants With Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis source ↗
- Trial start Phase 2: ALXN2420 Versus Placebo in Combination With Somatostatin Analogs in Participants With Acromegaly source ↗
- Trial start Phase 1/Phase 2: ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy source ↗
- Trial start Phase 2: Study of ALXN1920 in Adult Participants With Primary Membranous Nephropathy (PMN) source ↗
- Trial start 1: A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis. source ↗
- Trial start —: Long-term Safety of Danicopan: IPIG Registry-based Cohort Study source ↗
- Trial start —: Early Ravulizumab Treatment Of Anti- AChR Antibody-Positive Generalized Myasthenia Gravis source ↗
- Trial start Phase 3: Study of Ravulizumab in Pediatric Participants With Primary IgAN source ↗
- Trial start Phase 3: 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 Gra source ↗
- Trial start 2: Phase 2 Study of ALXN2030 in Patients With Antibody-Mediated Rejection After Kidney Transplantation source ↗
- FDA FDA New indication / efficacy supplement: SOLIRIS (eculizumab) — BLA125166/448, Feb 28, 2025. Applicant at approval: Alexion Pharmaceuticals, Inc. source ↗
- Trial start —: A Study Using Real-world Data to Examine Outcomes in Children and Adults Who Develop a Blood Clotting Disorder Called Thrombotic Microangiopathy (TMA) After Undergoing a Hematopoietic Stem Cell Tra source ↗
- Trial start —: Study of Ultomiris® (Ravulizumab) Safety in Pregnancy source ↗
- Trial start Phase 3: PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis source ↗
- Trial start Phase 3: Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) source ↗
- Trial start 3: Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP source ↗
- Trial start 3: Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa source ↗
- Trial start Phase 3: Study of Ravulizumab in Immunoglobulin A Nephropathy (IgAN) source ↗
Upcoming milestones
- Dec 15, 2026 (ESTIMATED) · Phase 3 · PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis · NCT06607627
- Dec 23, 2026 (ESTIMATED) · Phase 1 · Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study of ALXN2230 in Healthy Participants · NCT07352423
- Jun 25, 2027 (ESTIMATED) · Phase 4 · Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS · NCT07308574
- Jun 30, 2027 (ESTIMATED) · · Early Ravulizumab Treatment Of Anti- AChR Antibody-Positive Generalized Myasthenia Gravis · NCT06967480
- Jul 2, 2027 (ESTIMATED) · Phase 2 · Study of ALXN1920 in Adult Participants With Primary Membranous Nephropathy (PMN) · NCT07157787
- Aug 12, 2027 (ESTIMATED) · · A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM) · NCT05981092
- Aug 17, 2027 (ESTIMATED) · Phase 3 · Efficacy and Safety of Ravulizumab in Chinese Adults Participants With Generalized Myasthenia Gravis (gMG) · NCT07596784
- Aug 27, 2027 (ESTIMATED) · Phase 4 · A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab · NCT07221838
Clinical trials (lead sponsor) Showing 50 of 255
| Record | Title | Phase | Status | Start | Primary completion |
|---|---|---|---|---|---|
| NCT06291376 | Study of Ravulizumab in Immunoglobulin A Nephropathy (IgAN) | Phase 3 | Active, not recruiting | Mar 29, 2024 | Jan 5, 2026 (actual) |
| NCT07557420 | Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity Study of Ravulizumab in Chinese Adults With Neuromyelitis Optica Spectrum Disorder (NMOSD) | Phase 3 | Recruiting | Jul 20, 2026 | Sep 19, 2028 (estimated) |
| NCT07596784 | Efficacy and Safety of Ravulizumab in Chinese Adults Participants With Generalized Myasthenia Gravis (gMG) | Phase 3 | Recruiting | Jul 20, 2026 | Aug 17, 2027 (estimated) |
| NCT02306720 | Registry of Patients With Hypophosphatasia | Not listed | Enrolling by invitation | Jan 20, 2015 | Dec 31, 2031 (estimated) |
| NCT07789483 | An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of ALXN2220 in Adults With Transthyretin Amyloid Cardiomyopathy | Phase 3 | Not yet recruiting | Nov 23, 2027 | Jul 11, 2030 (estimated) |
| NCT07218887 | ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy | Phase 1/Phase 2 | Recruiting | Oct 24, 2025 | Jan 27, 2032 (estimated) |
| NCT06744647 | Phase 2 Study of ALXN2030 in Patients With Antibody-Mediated Rejection After Kidney Transplantation | 2 | Active, not recruiting | Mar 7, 2025 | Oct 11, 2027 (estimated) |
| NCT06183931 | Study of ALXN2220 Versus Placebo in Adults With ATTR-CM | Phase 3 | Active, not recruiting | Jan 11, 2024 | Dec 6, 2027 (estimated) |
| NCT07037420 | ALXN2420 Versus Placebo in Combination With Somatostatin Analogs in Participants With Acromegaly | Phase 2 | Terminated | Oct 28, 2025 | Jun 24, 2026 (actual) |
| NCT07024563 | Study of Ravulizumab in Pediatric Participants With Primary IgAN | Phase 3 | Recruiting | Jun 14, 2025 | Nov 29, 2028 (estimated) |
| NCT07732361 | A Study Using Real-world Data to Examine Outcomes in Children and Adults Who Develop a Blood Clotting Disorder Called Thrombotic Microangiopathy (TMA) After Undergoing a Hematopoietic Stem Cell Transplant (HSCT). | Not listed | Completed | Jan 22, 2025 | Mar 31, 2026 (actual) |
| NCT05746559 | ARTEMIS: Ravulizumab to Protect Patients With CKD From CSA-AKI and MAKE | Phase 3 | Terminated | Apr 6, 2023 | Oct 7, 2025 (actual) |
| NCT04933682 | Drug Interaction Study of ALXN2050 With Fluconazole and Rifampin in Healthy Adult Participants | Phase 1 | Completed | Jun 7, 2021 | Aug 23, 2021 (actual) |
| NCT04504825 | A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES) | Phase 3 | Active, not recruiting | Aug 25, 2020 | May 10, 2025 (actual) |
| NCT07081646 | A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis. | 1 | Recruiting | Aug 18, 2025 | Aug 15, 2029 (estimated) |
| NCT05346354 | Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD | Phase 2/Phase 3 | Active, not recruiting | Jun 23, 2022 | Jun 22, 2026 (estimated) |
| NCT01192425 | Paroxysmal Nocturnal Hemoglobinuria (PNH), Level of CD59 on Red and White Blood Cells in Bone Marrow Failure Syndromes | Not listed | Completed | Sep 1, 2010 | 2010-09 (actual) |
| NCT00546260 | Safety and Efficacy Study of Adjunctive Antiplatelet Therapy Prior to Primary PCI in Patients With STEMI | Phase 2 | Terminated | 2007-11 | 2008-07 (actual) |
| NCT06079281 | Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa | 3 | Active, not recruiting | Jan 3, 2024 | Jul 9, 2025 (actual) |
| NCT07157787 | Study of ALXN1920 in Adult Participants With Primary Membranous Nephropathy (PMN) | Phase 2 | Recruiting | Sep 19, 2025 | Jul 2, 2027 (estimated) |
| NCT04543591 | Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant | Phase 3 | Completed | Dec 10, 2020 | Sep 19, 2025 (actual) |
| NCT06830798 | 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 | Phase 3 | Recruiting | May 19, 2025 | Nov 30, 2027 (estimated) |
| NCT05966467 | Registry of Patients With AQP4+ NMOSD Treated With Alexion C5 Inhibitor Therapies | Not listed | Recruiting | Feb 1, 2024 | Sep 27, 2029 (estimated) |
| NCT05389449 | A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH | Phase 3 | Active, not recruiting | Oct 28, 2022 | Aug 24, 2026 (estimated) |
| NCT04512235 | A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES) | Phase 3 | Active, not recruiting | Nov 3, 2020 | Apr 8, 2025 (actual) |
| NCT05556096 | Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis | Phase 3 | Active, not recruiting | Nov 21, 2022 | May 27, 2025 (actual) |
| NCT07160608 | Safety and Efficacy of Tarperprumig in Adult Participants With Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis | Phase 2 | Recruiting | Nov 26, 2025 | Oct 11, 2027 (estimated) |
| NCT05981092 | A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM) | Not listed | Active, not recruiting | Oct 14, 2022 | Aug 12, 2027 (estimated) |
| NCT04622046 | A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM | 3 | Completed | Nov 13, 2020 | Nov 8, 2023 (actual) |
| NCT06578949 | Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 3 | Completed | Oct 10, 2024 | May 15, 2025 (actual) |
| NCT05982938 | Danicopan Early Access Program | Not listed | Available | Not listed | Not listed |
| NCT05886244 | Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China | Phase 3 | Completed | Jul 5, 2023 | Apr 14, 2025 (actual) |
| NCT06079359 | Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP | 3 | Active, not recruiting | May 14, 2024 | Jan 6, 2026 (actual) |
| NCT05274633 | Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria | Not listed | Completed | Jun 17, 2022 | Jun 30, 2024 (actual) |
| NCT06079372 | Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa | 3 | Active, not recruiting | Apr 2, 2024 | Jul 23, 2025 (actual) |
| NCT07308574 | Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS | Phase 4 | Recruiting | Dec 19, 2025 | Jun 25, 2027 (estimated) |
| NCT05644561 | Evaluation of PK, PD, Efficacy, Safety, and Immunogenicity of IV Ravulizumab in Pediatric Participants With Generalized Myasthenia Gravis | Phase 3 | Active, not recruiting | Jun 24, 2023 | Jul 15, 2026 (estimated) |
| NCT06312644 | Study of Ultomiris® (Ravulizumab) Safety in Pregnancy | Not listed | Recruiting | Dec 16, 2024 | Jul 11, 2034 (estimated) |
| NCT07352423 | Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study of ALXN2230 in Healthy Participants | Phase 1 | Recruiting | Mar 26, 2026 | Dec 23, 2026 (estimated) |
| NCT05501717 | Safety, PK/PD, and Immunogenicity Study of SC ALXN2030 in Healthy Participants | Phase 1 | Active, not recruiting | Aug 16, 2022 | Oct 9, 2025 (actual) |
| NCT07221838 | A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab | Phase 4 | Recruiting | Mar 4, 2026 | Aug 27, 2027 (estimated) |
| NCT06607627 | PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis | Phase 3 | Recruiting | Nov 13, 2024 | Dec 15, 2026 (estimated) |
| NCT04958135 | Study to Assess the Effect of Food on a Single Dose of Acoramidis in Healthy Adult Participants | Phase 1 | Completed | May 30, 2021 | Aug 4, 2021 (actual) |
| NCT01633489 | Lysosomal Acid Lipase (LAL) Deficiency Registry | Not listed | Recruiting | May 30, 2013 | Aug 30, 2029 (estimated) |
| NCT04202341 | Registry of Participants With Generalized Myasthenia Gravis Treated With Alexion C5 Inhibition Therapies (C5ITs) | Not listed | Recruiting | Dec 2, 2019 | Oct 1, 2029 (estimated) |
| NCT07413250 | Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data | Not listed | Active, not recruiting | Jan 14, 2026 | Jan 15, 2030 (estimated) |
| NCT07413679 | Long-term Safety of Danicopan: IPIG Registry-based Cohort Study | Not listed | Active, not recruiting | Aug 18, 2025 | Jul 1, 2029 (estimated) |
| NCT05234567 | A Prospective Sub-Study of the Global Hypophosphatasia Registry | Not listed | Recruiting | Aug 25, 2022 | Jul 18, 2028 (estimated) |
| NCT06967480 | Early Ravulizumab Treatment Of Anti- AChR Antibody-Positive Generalized Myasthenia Gravis | Not listed | Recruiting | Jul 31, 2025 | Jun 30, 2027 (estimated) |
| NCT05506254 | Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy | Not listed | Active, not recruiting | Jul 15, 2022 | Jan 1, 2037 (estimated) |
FDA records (last 2 years)
- FDA New indication / efficacy supplement: SOLIRIS (eculizumab) — BLA125166/448, Feb 28, 2025. Applicant at approval: Alexion Pharmaceuticals, Inc. source ↗
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