Swiss drugmaker Novartis said on Friday it has received regulatory approval from the US for gene therapy treating a rare childhood disorder, priced at $ 2.1 million, making it the most expensive drug in history.
The company said Zolgensma was a unique treatment for spinal muscular atrophy, a disease that affects about 1 in 10,000 births and results in death or need for permanent ventilation at two years of age in 90% of cases.
But the announcement comes as the administration of US President Donald Trump has vowed to meet rising drug costs.
Novartis defended the price by saying that gene therapy was a new type of transformative treatment and 50% cheaper than current treatments.
"Zolgensma is a historic breakthrough for the treatment of SMA and single gene therapy," said Novartis CEO Vas Narasimhan in a statement, adding that the company was working with government and insurance companies to accelerate coverage.
The total cost will be $ 2,125 million payable in five years at $ 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 said the safety of the drug was tested in an ongoing clinical study and that a full clinical study involved 36 patients aged between two weeks and eight months.
Most evidence of its efficacy was based on the results of the ongoing study, which concluded that "patients treated with Zolgensma demonstrated significant improvement in their ability to achieve developmental milestones," including control of the head and ability to sit without support. .
Jerry Mendell, a physician involved in the study of Nationwide Children's Hospital in Columbus, Ohio, added that "the level of efficacy, administered as a single and unique therapy, is truly remarkable and provides an unprecedented level of hope for families."
The most common side effects of Zolgensma are elevated liver enzymes and vomiting, according to the FDA.
Genetic and cellular therapies leverage biology to reverse diseases that range from congenital blindness to pediatric leukemia.
May 25, 2019 1:26 PM IST