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Zolgensma: Bumpy Japan Review Holds Lessons For Applicants

‘Numerous And Extensive’ Problems

 
• By Ian Haydock

Product review report reveals multiple delays and missteps on route to market, holding valuable lessons for other gene therapy developers but also evidencing an ultimately welcoming environment.

Gene therapy
Lessons From Zolgensma's Japan Review • Source: Shutterstock

Following a November 2018 filing, Novartis AG’s novel gene therapy Zolgensma (onasemnogene abeparvovec) was launched in Japan in May this year, marking a watershed moment for patients and the country’s national health insurance (NHI) scheme.

The therapy for spinal muscular atrophy type 1 (SMA1) in children under two years of age represented a significant medical...

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US FDA Advisory Committee Supports Vaccine Safety, Scrutinizes AE Reporting Quality

 
• By Sue Sutter

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