PIPELINE
Advancing Our Pipeline Every Day
We’re building on our fundamental understanding of complement biology and advancing on our core therapeutic areas of hematology, nephrology, neurology, metabolics, and cardiology. We continue to evolve into new areas where there is great unmet need and opportunity to help people living with rare diseases and their families fully live their best lives.
| Therapy | Therapeutic Area | Preclinical | Phase 1 | Phase 2 | Phase 3 |
|---|---|---|---|---|---|
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SOLIRIS for Guillain-Barre syndrome (GBS)
SOLIRIS is a first-in-class C5 complement inhibitor. |
Neurology Preclinical Phase 1 Phase 2 Phase 3 |
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ULTOMIRIS® (ravulizumab-cwvz) Subcutaneous QW for Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS)
ULTOMIRIS is a long-acting C5 inhibitor. |
Hematology Preclinical Phase 1 Phase 2 Phase 3 |
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ULTOMIRIS IV for generalized Myasthenia Gravis (gMG)
ULTOMIRIS is a long-acting C5 inhibitor. |
Neurology Preclinical Phase 1 Phase 2 Phase 3 |
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ULTOMIRIS IV for Neuromyelitis Optica Spectrum Disorder (NMOSD)
ULTOMIRIS is a long-acting C5 inhibitor. |
Neurology Preclinical Phase 1 Phase 2 Phase 3 |
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ULTOMIRIS IV for Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy (HSCT‑TMA)
ULTOMIRIS is a long-acting C5 inhibitor. |
Nephrology Preclinical Phase 1 Phase 2 Phase 3 |
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ULTOMIRIS IV for Complement Mediated Thrombotic Microangiopathy (CM-TMA) ULTOMIRIS is a long-acting C5 inhibitor. |
Nephrology Preclinical Phase 1 Phase 2 Phase 3 |
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ALXN1840 for Wilson Disease ALXN1840 (bis-choline tetrathiomolybdate) is an investigational, oral, targeted de-coppering therapy. |
Metabolics Preclinical Phase 1 Phase 2 Phase 3 |
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CAEL-101 for AL Amyloidosis
CAEL-101 is an investigational first-in-class amyloid fibril targeted therapy. |
Cardiology Preclinical Phase 1 Phase 2 Phase 3 |
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Acoramidis (ALXN2060) for Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Acoramidis (ALXN2060) is an investigational, oral, small molecule. Alexion holds an exclusive license to develop and commercialize acoramidis in Japan. |
Cardiology Preclinical Phase 1 Phase 2 Phase 3 |
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Danicopan (ALXN2040) for PNH with extravascular hemolysis (EVH)
Danicopan (ALXN2040) is an investigational, oral, factor D inhibitor. |
Hematology Preclinical Phase 1 Phase 2 Phase 3 |
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KOSELUGO® (selumetinib) for Neurofibromatosis Type 1 (NF1) Plexiform Neurofibromas (PN) in adults KOSELUGO is kinase inhibitor. |
Metabolics Preclinical Phase 1 Phase 2 Phase 3 |
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ULTOMIRIS for Dermatomyositis (DM) ULTOMIRIS is a long-acting C5 inhibitor. |
Neurology Preclinical Phase 1 Phase 2 Phase 3 |
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Danicopan (ALXN2040) for Geographic Atrophy (GA) Danicopan (ALXN2040) is an investigational, oral, factor D inhibitor. |
Other Preclinical Phase 1 Phase 2 Phase 3 |
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ALXN2050 PNH ALXN2050 is an investigational, oral, factor D inhibitor. |
Hematology Preclinical Phase 1 Phase 2 Phase 3 |
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ALXN2050 Renal Diseases
ALXN2050 is an investigational, oral, factor D inhibitor. |
Nephrology Preclinical Phase 1 Phase 2 Phase 3 |
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ULTOMIRIS IV Renal Diseases
ULTOMIRIS is a long-acting C5 inhibitor. |
Nephrology Preclinical Phase 1 Phase 2 Phase 3 |
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ALXN1720 ALXN1720 is an investigational, anti-C5 albumin-binding bi-specific mini-body optimized for sub-cutaneous delivery that binds and prevents activation of human C5. |
Other Preclinical Phase 1 Phase 2 Phase 3 |
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ALXN1820 ALXN1820 is an investigational, bi-specific anti-properdin mini-body. |
Other Preclinical Phase 1 Phase 2 Phase 3 |
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ALXN1850 for Hypophosphatasia (HPP) ALXN1850 is an investigational, next generation asfotase alfa asset. |
Metabolics Preclinical Phase 1 Phase 2 Phase 3 |
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NI006 for ATTR-CM
Alexion has entered into an exclusive global collaboration and license agreement with Neurimmune AG for NI006, an investigational human monoclonal antibody. |
Cardiology Preclinical Phase 1 Phase 2 Phase 3 |
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Zealand Pharma A/S
Alexion is collaborating with Zealand Pharma A/S to discover and develop novel peptide therapies for up to four targets in the complement pathway. |
Other Preclinical Phase 1 Phase 2 Phase 3 |
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Dicerna - GalXCTM
Alexion is collaborating with Dicerna to jointly discover and develop subcutaneously delivered GalXCTM RNA interface (RNAi) candidates. |
Other Preclinical Phase 1 Phase 2 Phase 3 |
This experience has taught me to look at the small things that count. Now, I feel that I have something to look forward to and I am who I want to be.”ROBERTA LIVING WITH gMG