THIS MESSAGE (MATERIAL) IS CREATED AND (OR) DISTRIBUTED BY A FOREIGN MASS MEDIA PERFORMING THE FUNCTIONS OF A FOREIGN AGENT AND (OR) A RUSSIAN LEGAL ENTITY PERFORMING THE FUNCTIONS OF A FOREIGN AGENT.
Two children in Russia and Kazakhstan have died while being treated for spinal muscular atrophy (SMA) with Zolgensma, considered the world’s most expensive drug, Reuters reports citing a report by Swiss drugmaker Novartis.
Both deaths were due to acute liver failure following five to six weeks of infusion, the company said in a statement.
There is no reaction from the Russian Ministry of Health yet.
A single dose of Zolgensma, which was registered in the Russian Federation at the end of last year, costs more than $2 million and is the only way to treat SMA, a genetic disease that manifests itself as progressive muscle weakness. The patient’s ability to contract progressively decreases, which eventually leads to impaired breathing and swallowing. According to statistics, half of children with this diagnosis do not live up to two years.
Until recently, there were no radical treatments for SMA in the world, therapy was limited to symptomatic relief and lifelong use of expensive drugs. Zolgensma is a gene therapy that, in a single course of treatment, replaces a missing or defective gene with a functional copy.