Avexis Receives FDA Approval For Zolgensma, First And Only Gene Therapy For Pediatric Patients With Spinal Muscular Atrophy

The Food and Drug Administration approved on May 24th Novartis’ $2.1 million gene therapy for spinal muscular atrophy — making it the world’s most expensive drug. The therapy, Zolgensma, is a one-time treatment for spinal muscular atrophy, a muscle-wasting disease and leading genetic cause of infant mortality that affects one in every 11,000 births. Novartis had previously said it could price the treatment between $1.5 million to $5 million. Novartis said the treatment will cost $2.1 million — or $425,000 a year spread out over five years. The company said it’s “working closely with insurers to create 5-year agreements based on success of the treatment as well as other novel pay-over-time options.” It’s currently in “advanced discussions” with more than 15 insurers on payment options. Shares of Novartis were up nearly 4% late-afternoon Friday. This marks a new era in medicine where new therapies can cure patients in a single treatment — but at a high price. Insurers and governments will need to figure out how to pay for these therapies and society will need to decide whether any drug, even lifesaving ones, are worth millions of dollars.

For further information see CNBC (https://www.cnbc.com/2019/05/24/fda-approves-novartis-2-million-spinal-muscular-atrophy-gene-therapy.html)