Novartis Reveals Two Deaths Related to SMA Drug Zolgensma

Novartis’ gene therapy for spinal muscular atrophy (SMA) has had a complicated history, of which there is a new chapter. Two patients receiving the therapy have died from acute liver failure, the company reported Thursday.

“While this is important safety information, it is not a new safety signal and we firmly believe in the overall favorable risk/benefit profile of Zolgensma, which to date has been used to treat more than 2,300 patients worldwide across clinical trials, managed access programs and in the commercial setting,” Novartis stated in a press release.

Novartis reported that the children developed acute liver failure about five to six weeks after receiving Zolgensma and about 1-10 days after initiation of corticosteroid taper. The corticosteroids are used to help manage risk from the therapy. The cases were in Russia and Kazakhstan.

The company indicated it has notified regulators in all countries where the therapy is approved and will inform healthcare providers where it is allowed. The company also plans to update the Zolgensma label to include mention of the deaths.

SMA is...