The Swiss pharmaceutical company Novartis announced on Friday that it has received the approval of the American regulatory authority for gene therapy, which treats a rare childhood disorder and has a price of USD 2.1 million, making it the most expensive drug in history.
The company claims that Zolgensma was a one-time treatment for spinal muscular atrophy, a disease that affects about 1 in 10,000 births and causes death or the need for constant ventilation at the age of two in 90 percent of cases.
But the announcement comes when the administration of US President Donald Trump has promised to deal with the rising cost of drugs.
Novartis defended prices, claiming that gene therapy is a new, transformative type of treatment and was 50 percent cheaper than current treatments.
"Zolgensma is a historic advancement in the treatment of SMA and ground-breaking one-off gene therapy," said General Director Novartis Vas Narasimhan in a statement, who added that the company works with the government and insurers to accelerate coverage.
The total cost will be $ 2.125 million payable over five years and be $ 425,000 per year.
Zolgensma works by providing a functional copy of the defective gene responsible for SMA to stop disease progression through a single intravenous infusion.
The US Food and Drug Administration has concluded that the safety of the medicine has been tested in an ongoing clinical trial and completed clinical trial in 36 patients aged from two weeks to eight months.
Most of the evidence of its efficacy was based on the results of an ongoing study that stated that "patients treated with Zolgensma … showed significant improvement in achieving developmental milestones", including head control and the ability to sit without support.
Jerry Mendell, a physician at the Nationwide Children & # 39; s Hospital in Columbus, Ohio, added that "the level of effectiveness, delivered as a one-off, one-off therapy, is truly remarkable and provides families with an unprecedented level of hope."
The most common side effects of Zolgensma are elevated levels of liver enzymes and vomiting, the FDA said.
Gene and cell therapies use biology to turn diseases away from congenital blindness to childhood leukemia.