Evidence Grade A — Regulatory approved. 101 published studies. 15 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.
Vosoritide is also known by these brand and alternate names:
101 published studies: 65 human, 4 animal, 1 in-vitro, 27 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.
PeptideTrace tracks 15 registered clinical trials for Vosoritide sourced from ClinicalTrials.gov.
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
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
Vosoritide for Short Stature in Turner Syndrome
EMA Marketing Authorisation
FDA ORIG 1
FDA SUPPL 2
FDA SUPPL 4
Vosoritide is a 39-amino-acid CNP analogue: CNP37 with Pro-Gly N-terminal extension resisting NEP cleavage. MW ~4,354 Da. Retains 17-residue disulfide ring for receptor binding. First targeted pharmacotherapy for achondroplasia. SC 15 mcg/kg daily. Half-life ~30 minutes (>>native CNP ~3 min).
Binds NPR-B (transmembrane guanylyl cyclase) on growth plate chondrocytes. NPR-B generates cGMP, activating cGKII which phosphorylates RAF1 at Ser43, inhibiting FGFR3-activated RAS/MAPK cascade (RAF1-MEK-ERK). In achondroplasia, gain-of-function FGFR3 (Gly380Arg) constitutively activates MAPK suppressing chondrocyte proliferation. Vosoritide restores more normal endochondral ossification.
Marketed as Voxzogo. Approved November 19, 2021. Phase III (N=121, age 5-18): growth velocity +1.57 cm/year vs. placebo (P<0.0001; 5.19 vs. 3.62 cm/year). 2-year extension sustained (+1.40 cm/year). Phase II ages 0-<5: 7.16 vs. 4.61 cm/year. Indication: achondroplasia in pediatric patients >=5 years with open epiphyses.
Somatrogon is marketed as Ngenla (approved June 2023) for paediatric growth hormone deficiency in children aged 3 years and older. In the pivotal trial, once-weekly somatrogon produced growth rates equivalent to daily somatropin injections (10.1 cm/year versus 9.8 cm/year), confirming that reducing injection frequency does not compromise growth outcomes. Ngenla represents a meaningful advance for paediatric patients and their families, reducing injections from 365 to 52 per year. Treatment adherence has been a persistent challenge with daily growth hormone, and weekly dosing is expected to improve long-term outcomes through better compliance. Somatrogon competes directly with somapacitan (Sogroya), the other approved weekly growth hormone, creating a new generation of less burdensome treatment options.
Somapacitan is marketed as Sogroya (approved August 2020 for adult growth hormone deficiency; expanded April 2023 to paediatric growth hormone deficiency in children aged 2.5 years and older). It is the first once-weekly growth hormone approved for both adult and paediatric patients. In adults, clinical trials showed improvements in body composition including reduced truncal fat. In children, growth rates were comparable to daily somatropin. The albumin-binding approach provides predictable drug levels with lower peak-to-trough variation than some other long-acting growth hormone technologies. Sogroya competes with somatrogon (Ngenla) in the growing once-weekly growth hormone market, with both products expected to gradually replace daily injections as the standard of care.
ACE-031 has no marketing authorisation. A Phase I trial in healthy women showed increased lean mass and decreased fat mass. A Phase II trial in DMD (24 patients) showed lean body mass increases but was discontinued due to bleeding-related safety concerns (epistaxis, telangiectasias, and gum bleeding), likely caused by inhibition of BMP-9/10 vascular signalling. ACE-031 is a large fusion protein (~90 kDa), not a peptide. Its clinical development was halted. The non-selective ligand-trapping profile — capturing beneficial vascular signalling molecules alongside the intended muscle-growth targets — illustrates the challenge of targeting the TGF-beta superfamily. Acceleron subsequently developed more selective agents.
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