News ID: 39841
Publish Date: 17:31 - 25 May 2019
TEHRAN, May 25 -The Food and Drug Administration on Friday approved a $2.125 million treatment for spinal muscular atrophy, a genetic illness known as muscle-wasting disease that kills many babies before they turn 2.

FDA approves gene therapy with $2.1M price tagTEHRAN, Young Journalists Club (YJC) -The price tag makes the gene therapy the most expensive drug in history. 

One in 11,000 babies is born with spinal muscular atrophy, a genetic condition that destroys the nerves that control muscles. It is a leading genetic cause of infant mortality.

"Today's approval marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases," said Ned Sharpless, the FDA acting commissioner.

The drug, Zolgensma, is a one-time treatment, while existing treatments that cost hundreds of thousands of dollars a year must be taken repeatedly. Novartis, which makes Zolgensma, said it costs about the same as five years worth of existing drugs for the condition.

Despite the innovation, children who receive the treatment still will not have normal muscle function and may require wheelchairs, The Washington Post reported.

The treatment is specifically for children less than 2 years old with SMA.

"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," Novartis CEO Vas Narasimhan said in a statement.

"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."

The company said it is working with as least 12 insurance companies to provide coverage for the drug.


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