Soliris - Alexion

Cost: $536,629 per U.S. patient per year

When it comes to the world's most expensive drugs, Soliris takes the cake. And with it, Alexion's ($ALXN) treatment has all the makings for rare-disease drug success: an essential treatment, a frightening disease, a lack of market rivals and a very, very hefty price tag.

Soliris was first indicated as a therapy for the debilitating and life-threatening blood disorder paroxysmal nocturnal hemoglobinuria, a malady that only affects about 8,000 Americans. But at over $500,000 per year, Soliris doesn't have to reach many patients to pass the blockbuster barrier.

That doesn't mean Alexion isn't working to expand its market. The drug now has an additional indication, as a treatment for atypical hemolytic uremic syndrome, an ultrarare genetic disorder that can lead to kidney failure, stroke, heart attack and death. And the company is testing the drug in a bevy of Phase II studies for uses in nephrology and neurology.

Despite Soliris' lifesaving capabilities, the U.K.'s cost-effectiveness gatekeeper, the National Institute of Health and Care Excellence (NICE), demanded an explanation back in March for the med's sky-high price. In September, it issued a recommendation for the drug, but it came along with a warning: Soliris should only be funded "if important conditions are met," including coordinating prescriptions through an "expert center."

For more:
Special Report: Top 20 orphan drugs by 2018 - Soliris
NICE slaps unusual rules on Alexion's pricey Soliris after atypical cost review
That's some reimbursement deal: Alexion hikes EPS, sales forecast on French agreement
Why does Alexion's Soliris cost $500K-plus? U.K. gatekeepers want to know

-- Carly Helfand (email | Twitter)

Soliris - Alexion