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.

FILTER

Therapeutic Area

Development Phases

Therapy Theraputic Area Preclinical Phase 1 Phase 2 Phase 3

SOLIRIS for Guillain-Barre syndrome (GBS)

SOLIRIS is a first-in-class C5 complement inhibitor. 

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS® (ravulizumab-cwvz) Subcutaneous QW

ULTOMIRIS is a long-acting C5 inhibitor.

Hematology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV for generalized Myasthenia Gravis (gMG)

ULTOMIRIS is a long-acting C5 inhibitor.

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN1840 for Wilson Disease

ALXN1840 (bis-choline tetrathiomolybdate) is an investigational, oral, targeted de-coppering therapy. 

Metabolics

Preclinical

Phase 1

Phase 2

Phase 3

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

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

ALXN2040 (Danicopan) for PNH with extravascular hemolysis (EVH)

ALXN2040 is an investigational, oral, factor D inhibitor.

Hematology

Preclinical

Phase 1

Phase 2

Phase 3

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

ULTOMIRIS for Dermatomyositis (DM)

ULTOMIRIS is a long-acting C5 inhibitor.

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN2040 (Danicopan) for Geographic Atrophy (GA)

ALXN2040 is an investigational, oral, factor D inhibitor.

Other

Preclinical

Phase 1

Phase 2

Phase 3

ALXN2050 PNH

ALXN2050 is an investigational, oral, factor D inhibitor.

Hematology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN2050 Renal Diseases

ALXN2050 is an investigational, oral, factor D inhibitor.

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV Renal Diseases

ULTOMIRIS is a long-acting C5 inhibitor.

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

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

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

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

ALXN2075 (cerdulatinib)

Cerdulatinib is a dual spleen tyrosine kinase and janus kinase (SYK/JAK) inhibitor.

Other

Preclinical

Phase 1

Phase 2

Phase 3

ALXN1820

ALXN1820 is an investigational, bi-specific anti-properdin mini-body.

Other

Preclinical

Phase 1

Phase 2

Phase 3

ALXN1850 for Hypophosphatasia (HPP)

ALXN1850 is an investigational, next generation asfotase alfa asset.

Metabolics

Preclinical

Phase 1

Phase 2

Phase 3

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

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

Last updated 11/12/21
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MEDICINES

Roberta, diagnosed with gMG at 16 years old
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