Naglazyme - BioMarin Pharmaceutical

Cost: $485,747 per U.S. patient per year 

Naglazyme has been around for nearly a decade but continues to rank just below the top of the list of most expensive drugs. The orphan drug was approved in the U.S. in 2005, followed shortly after by an EU approval to treat mucopolysaccharidosis type VI (MPS VI), also known as Maroteaux-Lamy syndrome.

MPS VI is a progressive condition that causes many tissues and organs to enlarge and become inflamed or scarred and often includes skeletal abnormalities. BioMarin's ($BMRN) recombinant enzyme replacement therapy helps patients with the rare genetic disease to walk and climb stairs better. The drugmaker upped the price just 2% last year, and it generated $271.2 million for the year, but the drug has a minuscule patient pool to sell into. EvaluatePharma says the drug was prescribed to only 64 people in the U.S. last year.

BioMarin has continued in this realm, having gotten approval this year of Vimizim, its drug to treat mucopolysaccharidosis IV, or Morquio syndrome. It put a price of $380,000 on that drug and has sold about $15 million worth of it since its approval in February. Forecasts are that it could hit peak sales of $400 million to $500 million annually.

The drugmaker's success with these specialty products has often led analysts to suggest it is prime for a takeover. Even Roche ($RHHBY) was rumored to be looking at it, but so far no bids have been made, at least not publicly.

For more:
BioMarin's $380K price tag on Vimizim is high, but far from the highest
Vimizim panel nod fuels new round of BioMarin buyout rumors
BioMarin skates through FDA panel for rare disease drug
Roche chief tamps down talk of $13.5B BioMarin buyout

-- Eric Palmer (email | Twitter)

Naglazyme - BioMarin Pharmaceutical