The world's most expensive drug: Alexion Pharmaceutical's Soliris, at $409,500 per year

Alexion Pharmaceuticals (Cheshire, CT), on the market for two years with Soliris (eculizumab), gets the prize—if that’s the right term—for the world’s most expensive drug, according to market and analysts’ data gathered by the magazine. The cost claimed for the drug (or any of the others) isn’t necessarily an average sales price, average manufacturer’s price, or any other “standard” average, but it is likely to put Soliris at the top of some list. Soliris is a monoclonal antibody that treats a blood disorder known as paroxysymal nocturnal hemoglobinuria (PNH) that afflicts some 8,000 Americans annually.

The Top Nine, according to Forbes:

• Soliris (eculizumab), for PNH, annual cost: $409,500. Manufacturer: Alexion Pharmaceuticals
• Elaprase (idursulfase), for Hunter syndrome, annual cost: $375,000. Manufacturer: Shire Pharmaceuticals
• Naglazyme (galsulfase), for Maroteaux-Lamy syndrome (mucopolysccharidosis IV), annual cost: $365,000. Manufacturer: BioMarin Pharmaceutical
• Cinryze (C1 esterase inhibitor), for hereditary angiodema, annual cost: $350,000. Manufacturer: ViroPharma
• Myozyme (alglucosidase alpha), for Pompe disease, annual cost: $300,000. Manufacturer: Genyzme
• Arcalyst (rilonacept), for cryopyrin-associated periodic syndromes, annual cost: $250,000. Manufacturer: Regeneron
• Fabrazyme (agalsidase beta), for Fabry disease, annual cost: $200,000. Manufacturer: Genzyme
• Cerezyme (imiglucerase), for Gaucher disease, annual cost: $200,000. Manufacturer: Genzyme
• Aldurazyme (laronidase), for Hurler syndrome (mucopolysaccharidosis I), annual cost: $200,000. Manufacturer: Genzyme, BioMarin Pharmaceutical.

There are several interesting aspects to this list. Except for Shire, all the companies are US-based. Most of the products are biotech (although it’s worth noting that Cinryze is derived from human plasma), and most treat genetic disorders which, if found during infancy, imply a lifetime of treatment. All of the drugs qualify as “orphan” and most are “ultra-orphan”—the smallest group of patients is for Aldurazyme, which Forbes says applies to 600 patients worldwide. However, a hallmark of many ultra-orphan diseases is that it can be hard to find doctors who know how to diagnose them.

“The success of specialty drugs for rare diseases comes at a time when the traditional business of selling medicines to the masses is in decline,” opines the Forbes writer, who goes on to quote a Wall Street analyst as saying that these drugs are “the future of the biotechnology industry.”