FDA opens up an ethical can of worms with accelerated approval for first drug for underlying genetic cause of dwarfism
The FDA on Friday signed off on an accelerated approval for BioMarin’s Voxzogo (vosoritide) injection, the first treatment to target the underlying genetics of dwarfism, which can increase the height of children five years of age and older with the condition.
The injection, which Biomarin said will have an annual net price of $240,000, works by binding to a specific receptor called the natriuretic peptide receptor-B, which reduces the growth regulation gene’s activity and stimulates bone growth in children.
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