The world's most expensive drug has gone on sale and costs $ 2.1 million

The United States has approved the Swiss pharmaceutical company Novartis a genetical therapy which treats a rare childhood disorder that atrophies the motor capacity of babies and that usually causes death in two years. The drug, however, is not accessible at all: has a value of $ 2.1 million, making it the most expensive in history.

The company explained that Zolgensma is a unique treatment for spinal muscular atrophy (SMA). This illness affects 1 in 10,000 babies and causes death or the need for permanent ventilation for two years in 90% of cases.

To defend the high cost of the drug, Novartis explained that gene therapy was a new type of transformer processing.

"Zolgensma is a historic breakthrough for the treatment of SMA and historical gene therapy, "said Vas Narasimhan, the company's executive director, in a statement, adding that Novartis was working with the government and insurers to: speed up the coverage.

The total cost will be $ 2.125 million this can be paid in fees of $ 425,000 per year.

How it works

Zolgensma works by providing a functional copy of the gene defective SMA, which stops the progression of the disease by a single intravenous infusion.

The US Food and Drug Administration stated that the safety of this drug had been tested in the context of an ongoing clinical trial and a completed clinical trial in which 36 patients participated between the ages of two weeks and eight months.

The most effective evidence of its efficacy is based on the results of the current trial, which found that "patients treated with Zolgensma demonstrated significant improvement in its ability to achieve the "engine development milestones", including control of the head and the ability to sit without support.

Jerry Mendell, a physician involved in the clinical trial at Nationwide Children's Hospital in Columbus, Ohio, added that the level of effectiveness, offered as a punctual treatment, " is really remarkable and provides a level of unprecedented hope for families"

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

Genetic and cellular therapies use biology to reverse diseases ranging from conbad blindness to childhood leukemia.