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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.

“Today’s approval fulfills an unmet medical need for more than 10,000 children in the United States,” said Theresa Kehoe, director of FDA’s Division of General Endocrinology.

The approval is based on a Phase III placebo-controlled trial in which 121 participants were randomly assigned to receive either Voxzogo injections or a placebo.

“Researchers measured the participants’ annualized growth velocity, or rate of height growth, at the end of the year. Participants who received Voxzogo grew an average 1.57 centimeters taller compared to those who received a placebo,” the FDA said.

A condition of the accelerated approval is that BioMarin must run a post-marketing study that will assess the final adult height.

Jean-Jacques Bienaimé, chairman and CEO of BioMarin, said in a statement, “Voxzogo is a medical first that is rooted in BioMarin’s focus on molecular genetics and targets the underlying cause of the condition. More than a decade of scientific research underpins the medical advance that Voxzogo represents. We thank the FDA for recognizing its value as the first therapeutic treatment option for children with achondroplasia.”

But there’s an ethical question at the heart of the approval too. According to STAT News, organizations such as Little People of America have long sought to advocate for fairness while noting that achondroplasia, the most common form of dwarfism, doesn’t preclude them from a fulfilling life, and drugs like Voxzogo could be a threat to the community they’ve built.


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