The company logo is seen at Swiss pharmaceutical company Novartis’ new cell and gene therapy plant in Stein, Switzerland, November 28, 2019. (Arnd Wiegmann/Reuters)
Novartis AG on Thursday reported the deaths of two patients from acute liver failure following treatment with Zolgensma gene therapy, which is used to treat spinal muscular atrophy.
The company has notified health authorities in the markets where the drug is sold, including the FDA, and, as an additional step, relevant health professionals.
“While this is important safety information, we believe in the overall favorable risk/benefit profile of Zolgensma rather than in new safety signals,” Novartis said in a statement.
Two fatal cases of acute liver failure have been reported in Russia and Kazakhstan 5-6 weeks after Zolgensma injection and approximately 1-10 days after the initiation of corticosteroid taper.
Novartis’ Zolgensma, which received conditional EU approval in early 2020, costs more than $2 million per patient.
By Sneha Bhowmik and Ankur Banerjee