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 Therapeutic 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

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

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

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

Metabolics

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

Danicopan (ALXN2040) for Geographic Atrophy (GA)

Danicopan (ALXN2040) is an investigational, oral, factor D inhibitor.

Other

Preclinical

Phase 1

Phase 2

Phase 3

ALXN2050 for PNH

ALXN2050 is an investigational, oral, factor D inhibitor.

Hematology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN2050 for gMG

ALXN2050 is an investigational, oral, factor D inhibitor.

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN2050 for Renal Diseases

ALXN2050 is an investigational, oral, factor D inhibitor.

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV for Renal Diseases

ULTOMIRIS is a long-acting C5 inhibitor.

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN1720 for gMG

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. 

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN1820

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

Hematology

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

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

ALXN1910

ALXN1910 is an investigational, next-generation, tissue-nonspecific alkaline phosphatase (TNSALP) enzyme replacement therapy (ERT).

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 07/29/22
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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