FDA approves Voxzogo, first drug to improve growth in common form of dwarfism
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The FDA granted accelerated approval to vosoritide injection to improve growth among children aged 5 years or older with achondroplasia and open epiphyses.
This is the first drug approved for this common form of dwarfism.
“Today’s approval fulfills an unmet medical need for more than 10,000 children in the United States,” Theresa Kehoe, MD, director of the division of general endocrinology in the FDA’s Center for Drug Evaluation and Research, said in an agency-issued press release. “With this action, children with short stature due to achondroplasia have a treatment option that targets the underlying cause of their short stature.”
Achondroplasia is genetic condition characterized by severely short stature and disproportionate growth. Adults with achondroplasia attain an average height of approximately 4 feet.
Achondroplasia is associated with a genetic mutation that causes overactivity of a growth regulation gene called fibroblast growth factor receptor 3. This overactive gene inhibits normal bone growth.
Vosoritide (Voxzogo, BioMarin Pharmaceutical) binds to the natriuretic peptide receptor-B, which reduces the growth regulation gene’s activity and stimulates bone growth.
A randomized phase 3 study assessed vosoritide’s efficacy and safety for individuals aged 5 years or older with achondroplasia who had open epiphyses.
Researchers randomly assigned 121 study participants to vosoritide injections or placebo. After 1 year, researchers assessed growth velocity, or rate of height growth.
Study participants assigned vosoritide grew an average of 1.57 cm taller than those assigned placebo, according to the FDA release.
The most common adverse effects associated with vosoritide treatment included injection site reactions, vomiting and decreased blood pressure.
The agent’s label includes a warning and precaution about decreased blood pressure.
As a condition of accelerated approval, a post-marketing study will be required to assess study participants’ final adult height.
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