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BioMarin Eyes Late 2021 Launch As Achondroplasia Drug Succeeds In Phase III

 
• By Mandy Jackson

R&D president Hank Fuchs said vosoritide can be filed based on height gains for children and safety in babies and toddlers with the most common form of dwarfism, but timing depends on meetings with regulators.

Red dart arrow hitting in the target center of dartboard with modern city and sunset background. Target business, achieve and victory concept .
Vosoritide hit a near-normal growth velocity mark in Phase III. • Source: Shutterstock

BioMarin Pharmaceutical Inc. will meet with regulators in the first half of 2020 to discuss filings for vosoritide in the treatment of achondroplasia – the most common cause of short stature, or dwarfism – now that the drug has succeeded in a 121-patient Phase III clinical trial.

The company said on 16 December that children who were treated with the therapy gained 1.6cm more in height over a year of treatment than those who received a placebo; a Phase II/III study in babies and toddlers is ongoing

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