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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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Potency Assay Issues Delayed US FDA Approval Of Mesoblast’s Ryoncil For Years

 
• By Sue Sutter

The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.

Mesoblast’s Ryoncil: US FDA Changed Its Mind On Need For A Randomized Trial

 
• By Sue Sutter

Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.

Ryoncil Chronology: Three Review Cycles, Two CRLs, One Dispute Resolution Request

 
• By Sue Sutter

The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.

Testosterone, TRAVERSE, And A Label Change 15 Years In The Making

 
• By Bridget Silverman

The Pink Sheet drug review profile investigates the long and rigorous process behind the FDA’s recent relaxation of the cardiovascular safety warning for testosterone products.

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