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Study 27 of 27Vosoritide literatureCureus · Case report2026

A Case of Inadvertent Overdose of Vosoritide Injection in a Three-Month-Old Infant With Achondroplasia.

A three-month-old infant received an accidental double dose of vosoritide but showed no adverse reactions, indicating potential variability in response among infants.

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Where it sits

this study against the rest of the vosoritide corpus
1
Preclinical
17
Observational · this one
0
Open-label
3
Randomised
6
Reviews

Summary and findings

A three-month-old infant with genetically confirmed achondroplasia accidentally received a double dose of vosoritide (0.24 mg; 53 μg/kg). The patient remained asymptomatic with no signs of hypotension or other adverse reactions during home monitoring. This case highlights the variability in hemodynamic sensitivity to vosoritide in early infancy.

How much of this paper we could read: full text read (0.80). We had a clear abstract, so the summary below closely tracks the paper. What this means →
0.24 mg vosoritide administered, equivalent to 53 μg/kg.2026

Abstract

The authors’ words, as Cureus supplied them

Vosoritide, a recombinant C-type natriuretic peptide analog that antagonizes fibroblast growth factor receptor 3 (<i>FGFR3</i>) signaling, is recommended for early initiation in infants with achondroplasia. However, clinical safety data for infants under six months remain limited, and concerns persist regarding its potential to induce symptomatic hypotension. We present a case of a three-month-old female infant with genetically confirmed achondroplasia (<i>FGFR3</i> p.Gly380Arg) who accidentally received an inadvertent double dose of vosoritide (0.24 mg; 53 μg/kg) during home self-injection therapy. This patient remained completely asymptomatic, exhibiting no signs of hypotension, lethargy, or facial pallor during home monitoring. While findings from a single patient cannot definitively establish safety, the complete absence of adverse reactions after an accidental overdose suggests substantial inter-individual variability in hemodynamic sensitivity to vosoritide during early infancy. This clinical observation provides insights into the drug's tolerability profile in young infants.

Elsewhere in the Vosoritide corpus

AA Phase 3 Trial of Vosoritide in Children with Hypochondroplasia.NEJM evidence · 2023 · n=81 · LSM difference of 2.33 cm/year between vosoritide and placebo; 95% CI, 1.85-2.82 cm/year; two-sided P<0.0001.HumanAA Phase 3 Trial of Vosoritide in Children with Hypochondroplasia.NEJM evidence · 2023 · n=81 · LSM difference of 2.33 cm/year between vosoritide and placebo, 95% CI 1.85-2.82 cm/year, two-sided P<0.0001.HumanBPerceived Impact of Vosoritide on Health-Related Quality of Life in Children with Selected Genetic Causes of Short Stature: A Qualitative Study of Children and Caregivers.Hormone research in paediatrics · 2026 · n=21 · 74% of caregivers and 100% of children reported changes in daily activities.HumanDFrom Small Data to Big Decisions: How Clinical Pharmacology Shapes Rare Disease Development.Journal of clinical pharmacology · 2026reviewASlipped Capital Femoral Epiphysis during Vosoritide Therapy for Short Stature: A Case Series.Hormone research in paediatrics · 2023 · n=5 · 2/12 participants with ACAN mutations developed SCFE.HumanBReal-World Evidence on the Effectiveness and Safety of Vosoritide in Latin American Patients With Achondroplasia (EVOLAC).American journal of medical genetics. Part A · 2023 · n=52 · Height Z-scores increased from 0.33 to 1.58 between 6 to 36 months, p<0.001 to p<0.05.Human