FDA approves world's most expensive drug: 5 things to know

Alia Paavola - Print  | 

The FDA has approved Zolgensma, Novartis' $2.1 million gene therapy for spinal muscular atrophy.

Five things to know:

1. With the $2.1 million price tag, Zolgensma is the world's most expensive drug.

2. The therapy, approved May 24, is a one-time treatment for spinal muscular atrophy, an inherited muscle-wasting disease that it often fatal to patients before age 2.

3. Novartis previously said that a seven-figure price, between $1.5 million and $5 million, would be justified due to its potential to cure the disease.

4. The drugmaker told CNBC it was in "advanced discussions" with more than 15 insurers on payment options and working to "create five-year agreements based on success of the treatment as well as other novel pay-over-time options."

5. The issue of gene therapy cost is gaining importance as more of them become available in the U.S. The treatments have the potential to cure otherwise devastating illnesses in one treatment, but insurers say that their multimillion dollar price tags aren't sustainable.

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