Swiss pharmaceutical company Novartis on Friday announced it had received US regulatory approval for a gene therapy that treats a rare childhood disorder and has a price tag of $2.1 million, making it the most expensive drug in history. Novartis defended the pricing by saying that gene therapy was a transformative new type of treatment and was 50 percent cheaper than current treatments. "Zolgensma is a historic advance for the treatment of SMA and a landmark one-time gene therapy," Novartis CEO Vas Narasimhan said in a statement, which added that the company was working with government and insurers to accelerate coverage. Zolgensma works by providing a functional copy of the defective gene responsible for SMA to halt the disease's progression via a one-time intravenous infusion. Gene and cell therapies leverage the biology to reverse diseases ranging from congenital blindness to pediatric leukemia.
Source: Mint May 25, 2019 04:41 UTC