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

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV for Immunoglobulin A Nephropathy (IgAN)

ULTOMIRIS is a long-acting C5 inhibitor.

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

Other

Preclinical

Phase 1

Phase 2

Phase 3

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

Anselamimab for AL Amyloidosis

Anselamimab is an investigational first-in-class amyloid fibril targeted therapy.

Cardiology

Preclinical

Phase 1

Phase 2

Phase 3

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

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

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

Danicopan for Geographic Atrophy (GA)

Danicopan is an investigational, oral, factor D inhibitor.

Ophthalmology

Preclinical

Phase 1

Phase 2

Phase 3

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

ULTOMIRIS IV for Lupus Nephritis (LN)

ULTOMIRIS is a long-acting C5 inhibitor.

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

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

ALXN1920

ALXN1920 is a kidney-targeted factor H fusion protein.

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN2030

ALXN2030 is an siRNA targeting the C3 complement protein.

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN2080

ALXN2080 is an investigational, oral, factor D inhibitor.

Other

Preclinical

Phase 1

Phase 2

Phase 3

Last updated 4/25/24

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).

Related Topics

See our approved medicines

MEDICINES

Liz
It was a relief to have an answer. Now I knew why I was feeling what I was feeling, so we could make a plan.”
LIZ LIVING WITH gMG