Alexion is focused on developing drugs for rare disorders. Its lead product, Soliris, is approved for paroxysmal nocturnal hemoglobinuria (PNH), a disease estimated to afflict over 20 000 patients worldwide. The product was approved in the US and Europe in 2007 with a broad label. The launch has gone extremely well to date given Alexion’s success at facilitating patient access and reimbursement and increasing physician awareness and screening to identify new PNH patients. We expect launch in other key territories, as well as continued penetration in the US and Europe, to enable Soliris sales in PNH to reach over USD 1.0 bn. Atypical hemolytic uremic syndrome (aHUS) is the next indication for which we expect Soliris to gain approval. We estimate an annual incidence of 1 200 to 1 800 patients in the US and Europe.

In October 2010, the company announced highly positive interim data from four Phase II trials that we expect will support approval for aHUS in the US and Europe in the second half of 2012 and the first half of 2013. We estimate an additional USD 500 mn to USD 1.0 bn market opportunity for Soliris. Soliris is also in early-stage testing in a number of other orphan indications that could generate a larger market opportunity than we expect if successful results are reported in 2011.

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