Genentech’s SMA Type 1 Data Strengthen Case Backing Risdiplam Approval
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.
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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.
Biogen confirmed that some patients treated with Spinraza are temporarily having their dosing postponed as hospital systems brace for COVID-19, potentially a broader issue for the industry.
CEO Vas Narasimhan is confident the Swiss major's gene therapy for spinal muscular atrophy will outperform present and future rivals – Biogen's Spinraza and Roche's risdiplam, respectively – as "we have a better profile simply because we are a one-time therapy."