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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Applications to market Roche’s spinal muscular atrophy drug have already been submitted in eight countries including the US, where a decision is due in August.
Biotech gets $190m up front in partnership that will discover small molecule RNA-targeted therapies against a range of targets selected by Roche. Arrakis thinks its technology can drug difficult-to-reach targets.
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.