AveXis says label makes Zolgensma gene therapy ‘treatment of choice’ for newly diagnosed SMA

Gene therapy Zolgensma from Novartis’ AveXis unit received a label from FDA that will allow it to become the “treatment of choice” for newly diagnosed spinal muscular atrophy patients, according to the company, as well as an option for hundreds of patients who are being treated with Spinraza nusinersen from Biogen.

FDA Friday approved Zolgensma onasemnogene abeparvovec-xioi and granted a broad label, for treatment of children less than two years of age with SMA with bi-allelic mutations in the SMN1 gene. The indication includes symptomatic as well as presymptomatic infants identified through genetic screening tests.

Novartis AG

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