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The Swiss pharmaceutical company Novartis announced Friday that it has received regulatory approval from the United States for gene therapy that treats a rare childhood disorder and costs $ 2.1 million, making it the drug of choice. the most expensive in history.
Zolgensma was a unique treatment for spinal muscular atrophy, a condition that affects approximately 1 in 10,000 births and causes death or the need for permanent ventilation at 2 years of age in 90% of cases.
The announcement comes as the administration of US President Donald Trump has pledged to cope with soaring drug costs.
Novartis defended the award by saying that gene therapy was a new type of transformative treatment and that it was 50% cheaper than current treatments.
"Zolgensma is a historic breakthrough for the treatment of ADS and unique gene therapy," Novartis chairman Vas Narasimhan said in a statement, adding that the company is working with the government and insurers to improve coverage.
The total cost will be $ 2.125 million, payable over five years, at $ 425,000 per year.
Zolgensma works by providing a functional copy of the defective gene responsible for AMS in order to stop the progression of the disease via a single intravenous infusion.
The US Food and Drug Administration has stated that the safety of the drug had been tested in the context of an ongoing clinical trial and a completed clinical trial involving 36 two-week-old patients at eight months.
Most of the evidence for its effectiveness was based on the results of the ongoing trial that "patients treated with Zolgensma … have demonstrated a significant improvement in their ability to reach the milestones of development", including a master's degree. head and the ability to sit without support. .
Jerry Mendell, a doctor involved in the lawsuit at the Nationwide Children's Hospital Foundation in Columbus, Ohio, added that "the level of effectiveness, administered as a unique treatment and punctual, is truly remarkable and offers an unprecedented level of hope to families. "
The most common side effects of Zolgensma are an increase in liver enzymes and vomiting, the FDA said.
Gene and cell therapies rely on biology to reverse diseases ranging from congenital blindness to childhood leukemia.
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