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The Food and Drug Administration on Friday approved a $ 2.125 million gene therapy for the treatment of a rare condition called spinal muscular atrophy in infants and toddlers.

Novartis has announced that the single-use drug Zolgensma would cost $ 425,000 a year over five years.

The FDA has approved Zolgensma in children under two years of age with spinal muscular atrophy, a rare hereditary disease caused by a mutation in the survival motor neuron (SMN) gene. This defect can disrupt the production of a protein and cause death of the motor neurons controlling the muscles.

About one in every 11,000 babies is born with spinal muscular atrophy. Children with the most severe form of the disease have difficulty holding their heads, swallowing or breathing. This disease is often fatal or may require the use of a mask or breathing machine by children.

Acting FDA Commissioner Ned Sharpless called the approval "a milestone in the transformative power of gene and cell therapies" that can treat a variety of diseases.

"The potential of gene therapy products to change the lives of these patients who may have been faced with a life-threatening illness or, worse, death, gives hope for the future," Sharpless said.

But the cost of therapy is likely to spark debate about the rising costs of prescription drugs and medical technology. Private and public insurers will also face the challenge of covering the costs of what is becoming the country's most expensive drug.

Novartis said the price of the drug was half the estimated cost of more than $ 4 million in the treatment of the disease for a decade. Zolgensma is a point therapy that uses a virus to provide a copy of the human NMS gene to prevent the death of motor neurons.

"We believe that by adopting this responsible approach, we will help patients take advantage of this transformative medical innovation and generate significant cost savings for the system," said Vas Narasimhan, Chief Executive Officer of Novartis.

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The Institute for Clinical and Economic Review, which analyzes the cost and effectiveness of drugs, announced Friday that its estimate of the "value-based price" of the drug ranged from 1.2 to 2.1 million of dollars.

"The insurers were going to cover Zolgensma, whatever the price, and Novartis has stated publicly that prices should be around $ 5 million," said Steven D. Pearson, president of ICER. "This is a positive result for patients and the entire health system Novartis has instead chosen to set the price of Zolgensma at a level more equitably to the benefits for these children and their families."

Although specialty drugs such as Zolgensma are taken by a small portion of the population, their costs can go beyond a family or an insurer.

Thomas Goetz is Head of Research at GoodRx, a consumer website and a mobile app that tracks the price of drugs.

Goetz said specialty drugs accounted for about 1% of prescriptions but 95% of their costs.

"These costs translate into higher premiums and more restrictive forms for everyone," Goetz said.

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