Alexion sets the price of living well with PNH at roughly $400k a year
In a conference call that so stunned a Credit Suisse analyst he was left literally at a loss for words, Alexion Pharmaceuticals yesterday announced that the annual average wholesale price in the U.S. for Soliris, its newly approved drug to treat the rare disease Paroxsymal Nocturnal Hemoglobinuria (PNH), would be $389,000.
Wow.
Leonard Bell, Alexion’s CEO, was quick to defend the cost of the drug–interrupting another Alexion executive who started discussing acceptable cost-effectiveness ratios–by referencing the high cost of other therapies for so-called ultra-orphan diseases, such as Pompe (Myozyme, Genzyme) and Hunter (Elaprase, Shire HGT). But the costs he cited for these drugs (”over $400,000″ and “over $800,000″ annually) appear to be inflated figures, even when considering weight adjustments for adult patients. Generally speaking, the top end cost for drugs that target around 10,000 patients globally is under $300k a year. So Alexion seems to be exploring new price territory here.
This story is sure to be fodder for the WSJ (who more than once roasted Genzyme for their pricing) and, more importantly, for a newly Democratic Congress with drug costs on its mind.
But patients apparently need not worry about the high cost of Soliris. Alexion, as they reiterated at least five times on the call by my count, has a goal ”that every patient who can benefit from Soliris will have access to Soliris.” Not the snazziest marketing message, but at least it’s reassuring to patients. Isn’t it?

December 11th, 2007 at 12:45 pm
[...] 3. Eculizumab (Soliris, Alexion). Eculizumab is a humanized monoclonal antibody against terminal complement protein C5 that inhibits terminal complement activation. It is the first complement activation inhibitor approved for human use and is also the first drug indicated for Paroxsymal Nocturnal Hemoglobinuria (PNH), a rare, complement-mediated disease characterized by red blood cell destruction. Eculizumab was approved for its ability to stabilize hemoglobin (49% versus 0% with placebo) and reduce transfusion requirements (mean 3 U versus 11 U with placebo) in PNH. Eculizumab represents a tremendous clinical advance for PNH and represents clinical proof-of-concept for inhibition of complement activation generally. Its downside is its exorbitant cost of nearly $400k per year, which keeps this innovation out of my top 3. [...]