Indicative of strong M&A appetite for orphan drug companies, Alexion Pharmaceuticals has agreed to acquire Synageva BioPharma in an US$8.4 B cash-and-stock deal. The key driver of the acquisition is Synageva’s lead product Kanuma™ (sebelipase alpha), an enzyme replacement therapy for lysosomal acid lipase deficiency that is under regulatory review in the US, Europe and Japan and which is regarded as a potential blockbuster. The deal will help reduce Alexion’s reliance on its sole marketed product Soliris® (eculizumab), although a number of analysts have questioned the very high purchase price.