Dr. Vasant Narasimhan, CEO of Novartis, speaking at the Healthy Returns conference in New York City on May 21, 2019.
Astrid Stawiarz | CNBC
The Food and Drug Administration on Friday approved Novartis' $2.13 million gene therapy for spinal muscular atrophy.
The company's shares traded in the U.S. rose more than 4% in midday trading Friday.
The therapy, Zolgensma, is a one-time treatment for spinal muscular atrophy — a muscle-wasting disease and leading genetic cause of infant mortality, affecting one in every 11,000 live births. Novartis had previously said it could price the treatment between $1.5 million to $5 million.
Novartis CEO Vas Narasimhan has called for new ways to pay for innovative gene therapies.
"Zolgensma is a historic advance for the treatment of SMA and a landmark one-time gene therapy. Our goal is to ensure broad patient access to this transformational medicine and to share value with the healthcare system," Vas Narasimhan said a statement Friday.
The company in explaining the rationale for its price said the one-time treatment is 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," Narasimhan said.
The company said it's in "advanced discussions" with "more than 15 payers" on terms.
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