Alexion Pharmaceuticals

Alexion Pharmaceuticals, now the rare disease group within AstraZeneca, operates as a global biopharmaceutical firm with a sharp focus on therapies for rare and ultra-rare diseases. Founded in 1992 by Dr. Leonard Bell and Steven Squinto, the company originated from academic roots at Yale University. Dr. Bell, a physician and professor at Yale School of Medicine, became interested in the complement system, a component of the immune system that, when uncontrolled, can cause inflammatory disorders. This scientific curiosity formed the bedrock of the company, leading to the development of its core therapeutic focus.

The company's business model is centered on the research, development, and commercialization of high-value therapies for diseases that affect small patient populations. Revenue is generated through the sales of its portfolio of drugs, which command premium pricing due to the significant research investment and the life-altering nature of the treatments for conditions with few or no alternatives. Alexion strategically reinvests in its pipeline through in-house R&D and a series of acquisitions, such as the purchases of Enobia Pharma, Achillion Pharmaceuticals, and Portola Pharmaceuticals, to broaden its therapeutic reach.

Alexion's pioneering work lies in translating the complex biology of the complement cascade into viable medicines. Its flagship product, Soliris (eculizumab), was first approved by the FDA in 2007 and is a terminal complement inhibitor that treats rare disorders like paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Another key product is Ultomiris (ravulizumab-cwvz), a long-acting C5 complement inhibitor, which offers a less frequent dosing schedule. The portfolio also includes Strensiq for hypophosphatasia (HPP) and Kanuma for lysosomal acid lipase deficiency (LAL-D), among others. These therapies are highly targeted, often representing the first and only approved treatments for their respective conditions.

A significant milestone in the company's history was its acquisition by AstraZeneca, which was completed in July 2021 for approximately $39 billion. This transaction established Alexion as AstraZeneca's dedicated rare disease unit, headquartered in Boston, Massachusetts. The acquisition combined Alexion's expertise in complement biology and its robust commercial portfolio with AstraZeneca's global scale, aiming to accelerate the development and delivery of medicines for rare diseases to a broader international market.

Keywords: Alexion Pharmaceuticals, AstraZeneca Rare Disease, rare diseases, orphan drugs, complement inhibitor, Soliris, eculizumab, Ultomiris, ravulizumab, paroxysmal nocturnal hemoglobinuria, PNH, atypical hemolytic uremic syndrome, aHUS, Leonard Bell, biopharmaceutical, complement cascade, immunology, Strensiq, Kanuma, metabolic disorders, neurology, nephrology, hematology, C5 inhibitor, life-changing therapies, ultra-rare disorders