US FDA Puts IT Zolgensma Studies On Partial Clinical Hold
Zolgensma And IV Studies Unaffected
The roller coaster ride for Novartis with its high-profile gene therapy Zolgensma continues as the FDA places a partial hold on trials of an intrathecal version aimed at older SMA patients on the back of nerve inflammation seen in a preclinical study.
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Biogen said some spinal muscular atrophy patients are currently being treated with Spinraza after receiving the Novartis gene therapy – its main competitor – so it will start a study of the risk/benefit in 2021.
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.
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.