Voxzogo
Evidence Grade A — Regulatory approved. 94 published studies. 17 registered clinical trials.
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Vosoritide (sold as Voxzogo) is the first targeted treatment for achondroplasia, the most common form of dwarfism affecting approximately 1 in 25,000 births. Given as a daily injection to children with open growth plates, it works by counteracting the overactive signal that restricts bone growth in this condition. It represents a shift from surgical limb-lengthening to pharmacological treatment.
94 published studies: 62 human, 4 animal, 1 in-vitro, 25 reviews
Vosoritide is marketed as Voxzogo (approved November 2021) for achondroplasia in paediatric patients aged 5 years and older with open growth plates. Administered as a daily subcutaneous injection dosed by weight.
In the pivotal trial, children receiving vosoritide grew an average of 1.57 cm per year faster than placebo (5.19 versus 3.62 cm/year), and this increased growth rate was sustained through two-year follow-up. Studies in younger children (under 5) have also shown increased growth velocity. The most common side effects are injection-site reactions and transient blood pressure decreases. Vosoritide represents a fundamental shift from surgical limb-lengthening to pharmacological treatment for achondroplasia.
Achondroplasia is caused by a single mutation in the FGFR3 gene that makes the receptor constantly active, sending a 'stop growing' signal to growth plate cartilage cells. In normal development, CNP counterbalances this signal through a separate pathway. Vosoritide amplifies the CNP pathway, overriding the excessive FGFR3 brake and restoring a more normal rate of bone growth. A two-amino-acid extension at one end protects it from rapid enzymatic breakdown, extending its duration compared to the body's own CNP.
In the pivotal trial, children on vosoritide grew 1.57 cm per year faster than those on placebo (5.19 versus 3.62 cm/year), and this increased growth rate was sustained through two-year follow-up. Studies in younger children have also shown increased growth velocity, potentially extending the treatment window. The most common side effects are injection-site reactions (71%) and temporary blood pressure drops — patients are advised to drink plenty of fluids before injection. A key unknown is the ultimate impact on final adult height, which will take years to determine as treated children reach maturity. The treatment is only effective while growth plates remain open and has not been studied in adults with achondroplasia. Research is exploring use in other skeletal conditions.
A Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
A Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
EMA Marketing Authorisation
FDA ORIG 1
FDA SUPPL 2
FDA SUPPL 4
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