- Less than three months after completing the $8.4 billion acquisition of Synageva Biopharma, Alexion got an approval for Kanuma.
- Kanuma has been approved for use in patients with lysosomal acid lipase deficiency (LAL-D), a progressive metabolic disease, which often leads to liver failure, multi-organ failure and premature death.
- Alexion also received an approval in the EU for another drug acquired as part of an acquisition—Strensiq for patients with pediatric-onset hypophosphatasia (HPP).
Both Kanuma and Strensiq are under review by the FDA. Alexion's new CEO, David Hallal, is encouraged by his company's recent wins. For quite some time, Alexion has been known as a one-drug company. Granted that one drug—Soliris, another rare disease drug—costs roughly half a million dollars per year, giving Alexion $2.23 billion in revenues last year. As for the recently approved drugs, Hallal is expecting more than $1 billion in sales for Kanuma, but has not yet established target revenues for Stresiq. In addition, Alexion is evaluating Soliris in clinical tirals for additional indications.
Alexion is one example of a company focused on rare disease drugs in which there is an unmet medical need and a large financial upside for the company.