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BioMarin’s Voxzogo Accelerated Approval Based Mostly On One-Year Data

 
• By Bridget Silverman

US FDA advisory committee had encouraged a two-year endpoint for achondroplasia treatments, but BioMarin attributes success to having ‘partnered closely’ with the agency. Confirmatory trial still being discussed.

Vosoritide approval (Nielsen Hobbs; the Pink Sheet | Alamy images)
• Source: Nielsen Hobbs; the Pink Sheet | Alamy images

The US Food & Drug Administration approved BioMarin Pharmaceutical Inc.’s C-type natriuretic peptide analog Voxzogo (vosoritide) on 19 November to improve growth in children five years of age and older with achondroplasia and open epiphyses. Voxzogo is “the first therapeutic treatment option for children with achondroplasia,” BioMarin emphasized.

The agency granted accelerated approval based upon an improvement in annualized growth velocity (AGV), with a required post-marketing study that...

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