Reuters
- 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.
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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.
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.
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Source: Business Insider – ecourt@businessinsider.com (Emma Court)