The FDA approves a gene therapy that is the most expensive drug in the world

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The gene therapy Zolgensma, priced at $2.1 million, treats infants with spinal muscular atrophy.

By Christopher Rowland Christopher Rowland Business reporter focused on the health-care economy's effects on patient health, costs, and privacy Email Bio Follow May 24 at 4:01 PM The Food and Drug Administration on Friday approved the most expensive drug in the world, a $2.1 million one-time gene therapy for treatment of a rare disease that afflicts infants.

Novartis said Friday that it would provide rebates to insurance companies if the drug did not work, but it did not provide details of what would be considered a failure of the drug. Even with the treatment, children do not gain normal muscle function and could still require wheelchairs and other support.

A competing drug, Biogen’s Spinraza, is already on the market but Spinraza must continue to be injected into the spines of patients for their whole lives, at a price of $750,000 for the first year and $375,000 a year after that. Novartis pointed out that the high cost of its drug is still 50 percent less than the more than $4 million for 10 years of treatment of Spinraza.

Novartis will set up five-year payment plans for states, small insurance companies and self-insured employers to help them cope with the high costs of the therapy.

 

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