During his first two months on the job, US Food and Drug Administration acting commissioner Ned Sharpless generally has come across as unflappable in his public appearances. However, press coverage of the approval of Novartis AG’s gene therapy Zolgensma (onasemnogene abeparvovec-xioi) for spinal muscular atrophy (SMA) has got him riled up.
Following the agency’s 24 May approval, many of the headlines focused on the therapeutic’s $2.1m price tag, as well as Novartis’ annuity-like payment model, under which the therapy would cost $425,000 annually for five years