Intrathecal Zolgensma Delay Could Allow Competitors To Shine

US FDA Requests Confirmatory Clinical Study

 
• By John Davis
  
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The rollercoaster ride continues for Novartis’s gene therapy Zolgensma, with the company committing to providing further clinical data on an intrathecal formulation for older spinal muscular atrophy patients.  

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The shine on Novartis AG’s new spinal muscular atrophy (SMA) therapy, Zolgensma, and perhaps on gene therapies in general, has been dulled by the US Food and Drug Administration requesting another study involving its intrathecal administration to older patients. Further data were needed to confirm the results of the previous STRONG study of intrathecal Zolgensma (AVXS-101, onasemnogene abeparvovec) in older SMA patients, US regulators explained to Novartis Gene Therapies (formerly AveXis).

The confirmatory study will indeed be initiated by Novartis Gene Therapies, the company said, although when it will start is...

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