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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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 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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KOSELUGO® (selumetinib) for Neurofibromatosis Type 1 (NF1) Plexiform Neurofibromas (PN) in adults
KOSELUGO is a kinase inhibitor. |
Bone Metabolism 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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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. |
Ophthalmology Preclinical Phase 1 Phase 2 Phase 3 |
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Vemircopan (ALXN2050) for PNH Vemircopan (ALXN2050) is an investigational, oral, factor D inhibitor. |
Hematology Preclinical Phase 1 Phase 2 Phase 3 |
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Vemircopan (ALXN2050) for gMG Vemircopan (ALXN2050) is an investigational, oral, factor D inhibitor. |
Neurology Preclinical Phase 1 Phase 2 Phase 3 |
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Vemircopan (ALXN2050) for Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN)
Vemircopan (ALXN2050) is an investigational, oral, factor D inhibitor. |
Nephrology Preclinical Phase 1 Phase 2 Phase 3 |
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ULTOMIRIS IV for Renal Diseases
ULTOMIRIS is a long-acting C5 inhibitor. |
Nephrology Preclinical Phase 1 Phase 2 Phase 3 |
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Gefurulimab (ALXN1720) for gMG Gefurulimab (ALXN1720) is an investigational, anti-C5 albumin-binding humanized bispecific VHH antibody optimized for sub-cutaneous delivery. |
Neurology Preclinical Phase 1 Phase 2 Phase 3 |
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ALXN1820 ALXN1820 is an investigational, anti-properdin humanized bispecific VHH antibody. |
Hematology Preclinical Phase 1 Phase 2 Phase 3 |
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ALXN1850 for Hypophosphatasia (HPP) ALXN1850 is an investigational, next-generation asfotase alfa asset. |
Bone Metabolism 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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ALXN1910 ALXN1910 is an investigational, next-generation, tissue-nonspecific alkaline phosphatase (TNSALP) enzyme replacement therapy (ERT). |
Bone Metabolism Preclinical Phase 1 Phase 2 Phase 3 |
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ALXN2030 ALXN2030 is an siRNA targeting the C3 complement protein. |
Nephrology Preclinical Phase 1 Phase 2 Phase 3 |
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ALXN2080 ALXN2080 is an investigational, oral, factor D inhibitor. |
Other 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