BioMarin’s Voxzogo Predicted For Blockbuster Sales, But Market Must Be Built
Very early launch numbers look good in France and Germany, but US launch will be more challenging due to a fragmented patient base, the company said. Launch price is a flat $899 per vial.
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Vosoritide, a once daily injection, could be the first drug available for achondroplasia, the most common form of dwarfism.
BioMarin’s confidence that its first-ever hemophilia gene therapy would be cleared by the US FDA was shaken by a complete response letter that the company claims changed the requirements for approval.
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.