Alexion Pharmaceuticals (AstraZeneca Rare Disease)

Alexion, AstraZeneca Rare Disease is the rare disease division of AstraZeneca, formed following AstraZeneca's 2021 acquisition of Alexion Pharmaceuticals, Inc., a Boston-headquartered biopharmaceutical company founded in 1992 by Dr. Leonard Bell. Alexion has spent more than three decades establishing itself as a global leader in orphan drugs, and was the first company to translate the complex biology of the complement cascade into approved medicines for severe, life-threatening conditions.

The division's therapeutic focus spans haematology, nephrology, neurology, metabolic disorders, cardiology, and ophthalmology. Its key marketed products include eculizumab (Soliris) and ravulizumab (Ultomiris) for paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome, asfotase alfa (Strensiq) for hypophosphatasia, sebelipase alfa (Kanuma) for lysosomal acid lipase deficiency, and andexanet alfa (Andexxa) for reversal of anticoagulant-related bleeding.

In 2024, Alexion generated total revenue of $8,768 million, up 13% year-over-year, driven by durable growth in C5 inhibition and expanding demand across its portfolio. The division serves patients in more than 70 countries, with AstraZeneca's broader network operating in over 100 countries. In July 2024, AstraZeneca acquired Amolyt Pharma to bolster Alexion's late-stage pipeline in rare endocrine and bone metabolism diseases.

Given Alexion's core focus on complement biology and rare genetic disorders, innovations in areas such as gene therapy, novel modality drug delivery, or complement pathway targeting are directly relevant to its mission of addressing significant unmet need across an estimated 400 million people globally living with rare diseases.