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 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 cardiac surgery-associated acute kidney injury (CSA-AKI)
ULTOMIRIS is a long-acting C5 inhibitor. |
Nephrology 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. |
Other Preclinical Phase 1 Phase 2 Phase 3 |
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ALXN2220 for ATTR-CM
Alexion has entered into an exclusive global collaboration and license agreement with Neurimmune AG for ALXN2220, an investigational TTR depleter. |
Cardiology Preclinical Phase 1 Phase 2 Phase 3 |
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Anselamimab for AL Amyloidosis
Anselamimab is an investigational first-in-class amyloid fibril targeted therapy. |
Cardiology Preclinical Phase 1 Phase 2 Phase 3 |
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Acoramidis for Transthyretin Amyloid Cardiomyopathy ATTR-CM (Japan Only)
Acoramidis 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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Efzimfotase alfa (ALXN1850) for Hypophosphatasia (HPP) Efzimfotase alfa (ALXN1850) 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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Gefurulimab for gMG Gefurulimab 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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VOYDEYA (danicopan) for PNH with extravascular hemolysis (EVH)
VOYDEYA (danicopan) is an investigational, oral, factor D inhibitor. |
Hematology Preclinical Phase 1 Phase 2 Phase 3 |
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VOYDEYA (danicopan) for Geographic Atrophy (GA) VOYDEYA (danicopan) is an investigational, oral, factor D inhibitor. |
Ophthalmology Preclinical Phase 1 Phase 2 Phase 3 |
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Vemircopan for gMG Vemircopan is an investigational, oral, factor D inhibitor. |
Neurology Preclinical Phase 1 Phase 2 Phase 3 |
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Vemircopan for Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN)
Vemircopan is an investigational, oral, factor D inhibitor. |
Nephrology Preclinical Phase 1 Phase 2 Phase 3 |
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ULTOMIRIS IV for Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN)
ULTOMIRIS is a long-acting C5 inhibitor. |
Nephrology 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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ALXN1920 ALXN1920 is a kidney-targeted factor H fusion protein. |
Nephrology 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 |
Phase III Projects: refers to assets that are pivotal in Phase II/III, or that have been submitted for regulatory approval, and may include assets that are now launched in one or more major markets (removed when launched in all applicable major markets).
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