BioMarin's $380K price tag on Vimizim is high, but far from the highest

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BioMarin ($BMRN) has slapped a price tag on its new drug Vimizim: $380,000 a year. That's hefty even for a rare disease treatment. But even at such nosebleed rates, Vimizim ranks in just third place on the list of the world's most expensive drugs.

Vimizim is the first FDA-approved drug for Morquio A syndrome, an enzyme deficiency that interferes with skeletal development and can trigger a host of other health problems. About 800 people suffer from the disease in the U.S., with 3,000 patients in the developed world, BioMarin says. At $380,000 per year, analysts expect it to bring in some $400 million to $500 million annually.

As the San Francisco Business Times points out, BioMarin would have to hike Vimizim's sticker price by $35,000 to even qualify for second place, now occupied by Cinryze, a therapy for familial hypercholesterolemia developed by ViroPharma ($VPHM). Shire ($SHPG) just snapped up that company for $4.2 billion, partly for that very drug. Not coincidentally, Shire's own rare disease drugs are among the globe's priciest. In fact, its Elaprase treatment for Hunter syndrome comes in fourth place at $375,000, per the Business Times.

To gain the top spot? BioMarin would have to raise the price by $60,000. Alexion Pharmaceuticals' ($ALXN) Soliris, long the world's most expensive drug at $440,000, treats paroxysmal nocturnal hemoglobinuria, among other rare maladies. Alexion has parlayed that one drug into blockbuster-level annual sales; for 2014, the company forecasts $2 billion in revenue. Like ViroPharma before the Shire sale, Alexion is constantly bandied about as a takeover target, but no deal, at least not yet.

One could say the same about BioMarin, rumored to be in Roche's ($RHHBY) sights last year. WIth rare diseases among the hottest fields in pharma these days--precisely because of the high prices they can command, and the captive markets they treat--companies that specialize in the field are equally hot commodities.

Vimizim is far from BioMarin's first foray into the business, too. Its Naglazyme--which treats another enzyme deficiency, Maroteaux-Lamy syndrome--ties with Shire's Elaprase for fourth place on the Times list, at $375,000. But by joining these rarified ranks of expensive meds, Vimizim could put BioMarin back in the rumor mill. In the meantime, we'll have to see how the launch goes.

- see the Business Times story

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