Jan 17, 2020

A new multimillion dollar drug

A lab technician prepares to thawing blood bag before genetically modifying a patients immune cells

A lab tech thaws a blood bag before genetically modifying a patient's immune cells. Photo: Gerard Julien/AFP via Getty Images

BioMarin Pharmaceutical is eyeing a $2 million–$3 million price tag for its hemophilia gene therapy if it's approved, which could make it the world's most expensive drug, the Wall Street Journal reports.

Why it matters: It's a good reminder that today's pipeline is likely to cause a giant shock to the health care system over the next few years.

  • The company's CEO said insurers have indicated that they're OK with the price range.
  • The most expensive drug in the world right now is Zolgensma, a $2.1 million-dollar gene therapy that initially ran into some coverage problems.

BioMarin argues that the lifetime cost of treating hemophilia is $25 million, making the company's gene therapy a relative bargain.

  • Yes, but: There have been some concerns that the treatment won't last for a lifetime.

Go deeper: Health care industry grapples with staggering gene therapy costs

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