FDA approves Novartis' $2.1 million gene therapy — making it the world's most expensive drug

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The FDA approved Novartis’ $2.13 million gene therapy for spinal muscular atrophy on Friday. The treatment will cost $2.125 million — or $425,000 per year for five years.

In rationalizing the expensive price, Novartis said the one-time treatment costs 50% less than the 10-year cost of current chronic management for the disease.

"We believe by taking this responsible approach, we will help patients benefit from this transformative medical innovation and generate significant cost savings for the system over time," said Narasimhan, who hasThe Institute for Clinical and Economic Review, which evaluates drug prices, earlier this year said Zolgensma was worth up to only $1.5 million.

"Insurers were going to cover Zolgensma no matter the price, and Novartis has spoken publicly about considering prices that approached $5 million," ICER President Steven Pearson said in a statement. "It is a positive outcome for patients and the entire health system that Novartis instead chose to price Zolgensma at a level that more fairly aligns with the benefits for these children and their families.

"As a global leader in the treatment of spinal muscular atrophy, a life threatening, devastating disease, Biogen welcomes additional therapeutic options to help individuals with this rare disease," Biogen said in a statement. Acting FDA Commissioner Ned Sharpless lauded the approval, saying in a statement it marked "another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases. With each new approval, we see this exciting area of science continue to move beyond the concept phase into reality."

 

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