One-time treatment for a rare genetic disease that targets infants now costs a whopping $2.1 million in the United States, making it the world's most expensive gene therapy.

On Friday, the Food and Drug Administration approved the use of the Novartis drug Zolgensma for treatment of spinal muscular atrophy, the leading genetic cause of death in children worldwide.

Zolgensma, which will be marketed by AveXis under the Swiss company Novartis, will be used to treat infants under the age of two diagnosed with spinal muscular atrophy. Included in the treatment are infants whose symptoms are not yet showing.

Dr. Ned Sharpless, the acting commissioner of the FDA, said the approval marks another milestone in the power of gene and cell therapies to treat diseases.

Novartis Drug Zolgensma Is World's Most Expensive Gene Therapy

Dr. Emmanuelle Tiongson from the Children's Hospital in Los Angeles believes Zolgensma is potentially a new standard of care for babies with the most serious form of spinal muscular atrophy.

"The job now is trying to negotiate with insurers that this would be a long-term savings," said Tiongson, who provided Zolgensma to patients under an expanded access program.

Indeed, the price of the gene therapy is quite astonishing. Executives from Novartis have tried to defend the price, explaining that a one-time treatment is more valuable than long-term treatments that are more expensive and actually cost several hundred thousand dollars a year.

Novartis said the gene therapy would be cost-effective at up to $5 million per patient.

But a review in April by the Institute for Clinical and Economic Review, an independent group in the United States, concluded that the value estimate for Zolgensma is excessive.

Moreover, based on additional clinical data from Novartis, the launch price, and the FDA label, ICER believes the Zolgensma drug fell within the upper bound of its range for cost-effectiveness.

Meanwhile, the advocacy group Patients for Affordable Drugs also criticized the cost of Zolgensma.

David Mitchell, the group's founder, said the rising costs of drugs is a symptom of how the broken system forces people to pay whatever price drug corporations demand for saving people's lives.

"We didn't pay for the polio vaccine based on the future cost savings for kids who didn't need to live in iron lungs," said Mitchell.

Rival Treatments For Spinal Muscular Atrophy

The Swiss company's chief executive, Vas Narasimhan, said Zolgensma is a near-cure for spinal muscular atrophy if delivered soon after birth. Data shows that the drug's durability extends to about five years.

Zolgensma, which is delivered by infusion, uses a virus to provide a normal copy of the SMN1 gene to babies who were born with the defective gene.

As Novartis expects Japanese and European approval later this year, the drug will compete with Spinraza, the first approved treatment for spinal muscular atrophy that is manufactured by Biogen Inc.

Spinal muscular atrophy often leads to difficulty breathing, paralysis, and death within months for babies born with the Type I form. The genetic disease affects about one in every 10,000 births, with at least 50 percent to 70 percent diagnosed with Type I form.

Approved in 2016, Spinraza also requires infusion into the spinal canal every four months and is priced at $750,000 for the first year, and $375,000 annually. It has also been deemed "excessive" by ICER.

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