Alexion, AstraZeneca Rare Disease
About Alexion
Alexion, AstraZeneca Rare Disease is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and delivery of life-changing medicines. A pioneering leader in rare disease for more than three decades, Alexion was the first to translate the complex biology of the complement system into transformative medicines, and today it continues to build a diversified pipeline across disease areas with significant unmet need, using an array of innovative modalities. As part of AstraZeneca, Alexion is continually expanding its global geographic footprint to serve more rare disease patients around the world. It is headquartered in Boston, US.

Rare Disease (as of July 2025)
ALXN1920
nephrology
ALXN2030
nephrology
ALXN2080
healthy volunteers
MEDI1341
multiple system atrophy/Parkinson's disease
anselamimab
amyloid light-chain amyloidosis
cliramitug (ALXN2220) DepleTTR-CM
transthyretin amyloid cardiomyopathy
efzimfotase alfa
hypophosphatasia
eneboparatide CALYPSO
hypoparathyroidism
gefurulimab PREVAIL
generalised myasthenia gravis