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World's Most Expensive Drug Would Be A $2.1m Novartis Gene Therapy

World's Most Expensive Drug Would Be A $2.1m Novartis Gene Therapy
The one time treatment for spinal muscular atrophy gene therapy called Zolgensma, developed by the Swiss drugmaker Novartis and one that has been approved by the United States drugs authority, it priced at a record $2.125 million.
The treatment aimed for children under the age of two with SMA, including those not yet showing symptoms, was approved by the US Food and Drug Administration. The treatment called Zolgensma approval was given to include babies that possess the deadliest form of the inherited disease in addition to those medical conditions in which there is setting of the debilitating symptoms is seen much later.
One of the leading genetic cause of death in infants is SMA. Among babies born with the most serious type 1 form of the disease could lead to paralysis, breathing difficulty and death within months of being born. One in every 10,000 live births are affected by SMA and prevalence of type 1 is seen among such babies at the rate of between 50 per cent and 70 per cent.
“This is potentially a new standard of care for babies with the most serious form of SMA,” said Dr Emmanuelle Tiongson, a Los Angeles paediatric neurologist who has provided Zolgensma to patients under an expanded access program.
The very record pricing of the treatment has been defended by executives at Novartis. The company has argued that compared to those treatments that are expensive and long-term – the total cost of which often runs into several hundred thousand dollars every year, this one time treatment is much more valuable.  Under this therapy, a virus is used to provide a normal copy of the SMN1 gene to babies who are born with a defective gene. This is delivered by  infusion.
The independent Institute for Clinical and Economic Review (Icer) had ruled in April that Novartis’s previous $5m-per-patient value estimate for Zolgensma was excessive, after carrying out a review in April.
But on Friday, Icer said that it believed the drug fell within the upper bound of its range for cost-effectiveness when considering the additional clinical data provided by Novartis, the broad FDA label and the launch price of the treatment.
More than 150 patients with Zolgensma have been treated by it so far, Novartis said. Zolgensma was described to be a near-cure treatment for babies born with SMA if it is delivered soon after birth by Novartis’ chief executive, Vas Narasimhan. However, according to data, the durability of the drug only extends to about five years.
European and Japanese drug authorities would also give their approval for the drug by later this year, Novartis expects. Biogen’s Spinraza, the first approved treatment for SMA, would by the primary competitor of Zolgensma.
Spinrazawas given approval by various drug authorities in 2016 and its treatment requires the drug being infused into the spinal canal of patients once every four months. However Icer had also considered the price of this treatment to  be excessive – which come in at $750,000 for the first year and $375,000 annually every year subsequent to it.


Christopher J. Mitchell

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