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It's Official: Novartis SMA Gene Therapy Zolgensma Is World's Most Expensive Drug

 
• By Mandy Jackson

The total cost of Zolgensma is more than $2m – in line with estimates – or $425,000 annually for five years if payers choose an annuity-like payment plan. Novartis is also negotiating value-based reimbursement agreements for infants treated with the one-time infusion.

Neurons and nervous system. 3d render of nerve cells
Zolgensma prevents the loss of neurons in SMA patients. • Source: Shutterstock

Novartis AG is setting new records as it prepares to launch Zolgensma, the spinal muscular atrophy (SMA) gene therapy developed by Novartis subsidiary AveXis Inc. The 24 May approval makes it the second gene therapy to clear the US FDA, and the largely expected price of about $2m makes it the most expensive drug in the world.

Novartis anticipates that some payers will agree to an annuity-like model under which Zolgensma (onasemnogene abeparvovec-xioi) will cost $425,000 annually for five years, but such agreements may be difficult to implement

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