The FDA just approved a new treatment for a devastating disease. The price tag is more than $2 million.

FILE PHOTO: CEO Vas Narasimhan of Swiss drugmaker Novartis addresses the company's annual news conference in Basel, Switzerland January 30, 2019. REUTERS/Arnd WiegmannCEO Vas Narasimhan of Swiss drugmaker Novartis addresses the company's annual news conference in BaselReuters

  • The US FDA just approved a new treatment for a devastating rare genetic disease.
  • Called Zolgensma, it is a type of cutting-edge, one-time treatment called a "gene therapy" that treats the disease spinal muscular atrophy at its genetic root.
  • That means it should work longer than the typical drug, but has also translated to a much higher price tag, too.
  • Zolgensma, which is made by drugmaker Novartis's AveXis unit, will cost $2.125 million, the company said on Friday.
  • Visit Business Insider's homepage for more stories.

The FDA just approved a cutting-edge new treatment for a devastating rare genetic disease called spinal muscular atrophy.

The treatment, Novartis's Zolgensma, is a one-time therapy that works to treat the disease at the genetic level. That meants the drug's effects should last for a long time, though it's still not known if the treatment will be permanent.

Read more: Drugs that cost as much as a house are on the way to treat rare and devastating diseases. The US is scrambling to figure out how to pay for them.

Novartis priced the product at $2.125 million, or $425,000 a year if paid over a five-year installment plan. Novartis called the price fair and reasonable on a conference call with reporters on Friday.

ICER, a group that measures the cost-effectiveness of new drugs, has said that a fair price range for Zolgensma is likely between $310,000 to $900,000, and could be up to $1.5 million.

Read more: A revolutionary drug that could treat a rare and devastating disease is prohibitively expensive. But one state has a plan to pay for its potential $5 million price tag.


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