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 | Theraputic 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
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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ALXN2060 (AG10) for Transthyretin Amyloidosis (ATTR) ALXN2060 (AG10) is an investigational, oral, small molecule. Alexion holds an exclusive license to develop and commercialize AG10 in Japan. |
Cardiology Preclinical Phase 1 Phase 2 Phase 3 |
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ALXN2040 (Danicopan) for PNH with extravascular hemolysis (EVH)
ALXN2040 is an investigational, oral, factor D inhibitor. |
Hematology Preclinical Phase 1 Phase 2 Phase 3 |
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ANDEXXA for Acute Intracranial Hemorrhage (ICH) ANDEXXA has conditional approval in the U.S. and EU (marketed as ONDEXXYA in the EU) for the reversal of anticoagulation in patients experiencing life-threatening or uncontrolled bleeding who are treated with rivaroxaban or apixaban. |
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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ALXN2040 (Danicopan) for Geographic Atrophy (GA) 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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ANDEXXA Urgent Surgery ANDEXXA has conditional approval in the U.S. and EU (marketed as ONDEXXYA in the EU) for the reversal of anticoagulation in patients experiencing life-threatening or uncontrolled bleeding who are treated with rivaroxaban or apixaban. |
Other Preclinical Phase 1 Phase 2 Phase 3 |
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ALXN1830
ALXN1830 is an investigational, humanized monoclonal antibody that inhibits the interaction of neonatal Fc receptor (FcRn) with Immunoglobulin G (IgG) and IgG immune complexes. |
Other 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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ALXN2075 (cerdulatinib) Cerdulatinib is a dual spleen tyrosine kinase and janus kinase (SYK/JAK) inhibitor. |
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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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