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Home https://server7.kproxy.com/servlet/redirect.srv/sruj/smyrwpoii/p2/ Health https://server7.kproxy.com/servlet/redirect.srv/sruj/smyrwpoii/p2/ Novartis now has the most expensive substance ever after obtaining approval from the United States

Novartis now has the most expensive substance ever after obtaining approval from the United States



Basel headquarters of the Swiss pharmaceutical giant Novartis. Photo: SEBASTIEN BOZON / AFP

On Friday, Friday, the Swiss pharmaceutical company Novartis announced that it had received US regulatory approval for gene therapy that deals with a rare childhood disorder and has a price of $ 2.1 million, making it most expensive fabric in history.

The company said that Zolgensma was a one-time treatment for spinal muscular atrophy, a disease that affects approximately 10,000 people. One in 10,000 births results in death or the need for permanent ventilation at age two in 90 percent of cases.

But the message comes as the administration of US President Donald Trump has promised to tackle rising drug costs.

Novartis defended the pricing by saying that gene therapy was a transformative new type of treatment and was 50 percent cheaper than current treatments.

"Zolgensma is a historic advance in the treatment of SMA and a landmark one-time gene therapy," said Novartis CEO Vas Narasimhan in a statement that added that the company worked with the government and insurance companies to speed up coverage. [1

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The total cost will be $ 2,125 million paid over five years at $ 425,000 a year.

Zolgensma works by providing a functional copy of the defective gene responsible for SMA to stop the progression of the disease via a one-time intravenous infusion.

The US Food and Drug Administration said the drug's safety had been tested in an ongoing clinical trial and a completed clinical trial of 36 patients between two weeks and eight months.

Most of the evidence of its effectiveness was based on the results of the ongoing study, which showed that "patients treated with Zolgensma … showed significant improvement in their ability to reach developmental motor milestones" including head control and the ability to sit without support.

Jerry Mendell, a physician involved in the National Children's Hospital trial in Columbus, Ohio, added that "the level of efficacy delivered as a single one-time treatment is truly remarkable and provides a level of unprecedented hope for families. "

The most common side effects of Zolgensma are elevated liver enzymes and vomiting, the FDA said.

Gene and cell therapies utilize biology to reverse diseases ranging from congenital blindness to pediatric leukemia.


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