Novartis believes its new gene therapy is worth more than $4 million for a one-time dose, and the company has some data to back that up. But, with a global spotlight on the escalating cost of medicine, is it politically viable to set a new record for the world’s most expensive drug?
The treatment, called AVXS-101, has demonstrated dramatic effects in spinal muscular atrophy, a rare genetic disorder whose most severe form is fatal for almost all patients before age 2. In a 15-patient trial, infants with SMA who got AVXS-101 had a 100 percent survival rate after 24 months, data that convinced Novartis to pay $8.7 billion for the gene therapy’s inventor.
And those data are why Novartis believes AVXS-101 will be worth upward of $4 million per dose if and when it wins approval next year, the company said in a presentation to investors Monday — while stressing that the actual price would not be disclosed until after approval.
This article is exclusive to STAT+ subscribers
Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.
Already have an account? Log in
About the Author Reprints
To submit a correction request, please visit our Contact Us page.
