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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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More from Approvals

New EU Filings

 
• By Neena Brizmohun

Insulin efsitora alfa, Eli Lilly’s once-weekly, subcutaneous treatment for type 2 diabetes, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

Japan Roundup: Approvals Recommended For Ibtrozi, Voranigo And Ycanth

 
• By Lisa Takagi

Along with multiple new therapeutics for NSCLC, Astellas's Izervay and several products licensed from US companies receive approval recommendations in Japan.

First-Of-A-Kind Nasal Spray For Pain In Children Among 13 New EU Filings

 
• By Neena Brizmohun

Cessatech and Proveca’s investigational, sufentanil/ketamine fixed-dose combination analgesic nasal spray is among the latest drugs that the European Medicines Agency has started to review for potential pan-EU marketing authorization.

US FDA Upheaval Forces User Fee Talks To Begin Later Than Usual

 
• By Derrick Gingery

The fall start of PDUFA and GDUFA negotiations could impact the agreement review process and other parts of the schedule intended to ensure it reaches Congress on time.

More from Product Reviews

Japan Roundup: Approvals Recommended For Ibtrozi, Voranigo And Ycanth

 
• By Lisa Takagi

Along with multiple new therapeutics for NSCLC, Astellas's Izervay and several products licensed from US companies receive approval recommendations in Japan.

US FDA Compounding Advisory Panel Hit Hardest By Recent Purges

 
• By Sarah Karlin-Smith

A Pink Sheet review found 10 FDA advisory panel members recently pulled off committees despite having time left in their appointed terms.

First-Of-A-Kind Nasal Spray For Pain In Children Among 13 New EU Filings

 
• By Neena Brizmohun

Cessatech and Proveca’s investigational, sufentanil/ketamine fixed-dose combination analgesic nasal spray is among the latest drugs that the European Medicines Agency has started to review for potential pan-EU marketing authorization.