Here is some background info on Alexion. It has a market cap of $36bn. Its main drug is Soliris and its main indication of PNH. RVX's announcement today is basically a 'shot across the bows' of one of the largest ODD specialist drug companies there is. Alexion is trying to expand Soliris's reach into neurological and kidney disorders (this is exactly what RVX is trying to do; note DM's remarks on CKD and also that BOM will have a CKD trial embedded into it). Alexion has also made orphan focused acquisitions.....

Alexion Pharmaceuticals specializes in the development and marketing of drugs for life-threatening medical conditions. The firm's key product is Soliris, the first approved therapy for paroxysmal nocturnal hemoglobinuria, an ultra-rare blood disorder. Soliris was also approved for the treatment of atypical hemolytic uremic syndrome. Alexion's pipeline includes other research projects that focus on complement system disorders, and Strensiq and Kanuma, which target other ultra-rare diseases.

Alexion Pharmaceuticals' strategy is to develop drugs that significantly improve the condition of those suffering from rare (or "orphan") diseases. Its lead drug Soliris has achieved blockbuster status as the only approved treatment for two ultra-rare disorders. With the potential for Soliris to expand into other indications and a pipeline of other drugs all targeting orphan opportunities.

The key to Soliris’ success is its strong pricing power. The drug has dramatic, life-saving effect on paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two rare genetic blood disorders that both affect only 5,000-10,000 people in North America and Europe. Despite this small target population, Soliris’ high price (more than $400,000 annually) and the need for subsequent lifelong chronic use have quickly turned the drug into a blockbuster. As an orphan drug, Soliris also benefits from a period of marketing exclusivity and favorable reimbursement policies.

Alexion's pipeline is focused on expanding Soliris’ reach into neurological and kidney disease indications, with several Phase II and III trials ongoing. Alexion has also made orphan-focused acquisitions, most notably the recently announced Synageva BioPharma, to diversify its pipeline and to leverage its rare-disease expertise across its clinical, regulatory and commercial infrastructure.