Novartis's Gene Therapy Zolgensma Has Durable Effects At Five Years
Intrathecal Therapy's 'Remarkable' Disease Scores
AveXis/Novartis’s one-time AAV9 vector-based intravenous gene therapy for spinal muscular atrophy, Zolgensma, continues to have beneficial effects up to five years after its administration to babies, and an intrathecal formulation has significant promise in older infants.
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Novartis’s AveXis unit has received a CHMP nod for the spinal muscular atrophy gene therapy at its latest meeting, sending it on the final steps to market in the EU.
If Zolgensma gets the thumbs up from the EMA this week, it will likely get formal EU approval within a couple of months.
With top-line data showing efficacy in type 1 SMA patients, risdiplam may be able to obtain a label that would address significantly more patients than Novartis’ Zolgensma. Final efficacy data will show how drug’s efficacy compares to Biogen’s Spinraza.