Alexion Pharmaceuticals

Alexion Pharmaceuticals, Inc., a subsidiary of AstraZeneca, is a pharmaceutical company headquartered in Boston, Massachusetts that specializes in orphan drugs to treat rare diseases.

Its products include eculizumab (Soliris) and ravulizumab (Ultomiris), both used to treat the rare disorders of atypical hemolytic uremic syndrome (aHUS) and paroxysmal nocturnal hemoglobinuria (PNH); asfotase alfa (Strensiq), used to treat hypophosphatasia; sebelipase alfa (Kanuma), used to treat lysosomal acid lipase deficiency, and andexanet alfa (Andexxa), used to stop life threatening or uncontrollable bleeding in people who are taking rivaroxaban or apixaban.

With costs that can reach as much as $2 million per year, the drugs manufactured by Alexion are some of the most expensive drugs worldwide.

Alexion Pharmaceuticals was founded in 1992 at Science Park in New Haven, Connecticut by Steven Squinto and Leonard Bell, a physician at Yale New Haven Hospital and assistant professor of medicine and pathology at Yale School of Medicine.

In 2000, Alexion moved its headquarters from New Haven to Cheshire, Connecticut.

Alexion received U.S. Food and Drug Administration (FDA) approval for Soliris in 2007. It was initially approved to treat paroxysmal nocturnal hemoglobinuria, a rare blood disorder.

In June 2010, there was an outbreak of hemolytic–uremic syndrome caused by shigatoxigenic and verotoxigenic Escherichia coli (EHEC) in Germany. Soliris was tested as a treatment option because of its effectiveness in treating atypical hemolytic uremic syndrome, an illness similar to that caused by the EHEC infection.

In January 2014, the company paid Moderna $100 million for ten product options to develop rare disease treatments, including for Crigler–Najjar syndrome, using Moderna's mRNA therapeutics platform; however, the program was scrapped in January 2017 after animal trials showed that Moderna's treatment would never be safe enough for humans.