Voxzogo (vosoritide) / BioMarin - LARVOL DELTA

CANOPY-HCH-EXT: Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia (clinicaltrials.gov) - P3 | N=140 | Enrolling by invitation | Sponsor: BioMarin Pharmaceutical

New P3 trial

Vosoritide (Voxzogo) for Achondroplasia: A Review of Clinical and Real-World Evidence. (PubMed, Cureus) - "Treatment with vosoritide may improve long-term outcomes for children with achondroplasia when compared with the current pharmacological standard of care. Ongoing studies are expected to clarify its effects on adult height, potential effects on skeletal deformities, and overall quality of life."

HEOR • Journal • Real-world evidence • Review • Genetic Disorders • Hypotension • Orthopedics • FGFR3

Continued Growth After Puberty in Participants With Achondroplasia Treated With Vosoritide in a Phase 3 Long-Term Extension Trial (ASBMR 2025) - No abstract available

Late-breaking abstract • P3 data • Genetic Disorders

Effect of Vosoritide on Spine Morphology in Young Children With Achondroplasia: 1-Year Results From a Double-Blind, Randomized Phase 2 Study (ASBMR 2025) - No abstract available

Clinical • Late-breaking abstract • P2 data • Genetic Disorders

Effect of Long-Term Vosoritide Treatment in Pediatric Participants With Achondroplasia on Bone Mineral Density and Bone Content: Results From Quantitative Computed Tomography Analyses (ASBMR 2025) - No abstract available

Clinical • Late-breaking abstract • Genetic Disorders • Pediatrics

Vosoritide, a C-Type natriuretic peptide (CNP) analog, alone and in combination with alendronate improves incidence of new fractures and long bone growth in the oim mouse model for Osteogenesis Imperfecta: A Promising Therapeutic Approach (ASBMR 2025) - No abstract available

Combination therapy • Preclinical • Genetic Disorders • Musculoskeletal Diseases • Orthopedics

Comparative Analysis of Vosoritide Treatment in Children with Achondroplasia: Transition from Growth Hormone Therapy Versus GH-Naïve Initiation (ASBMR 2025) - No abstract available

Clinical • Genetic Disorders

Integrating vosoritide therapy with limb surgery in paediatric patients with achondroplasia: real-life experiences. (PubMed, Orphanet J Rare Dis) - "This report provides the first clinical description of the combination of precision therapy with limb surgery in a relatively large multicentre cohort of paediatric patients with achondroplasia. These findings support continued exploration of the integration of different therapeutic approaches."

Journal • Genetic Disorders • Musculoskeletal Diseases • Orthopedics • Pediatrics • FGFR3

Sleep-disordered breathing in children with achondroplasia assessed by polysomnography: a retrospective chart review. (PubMed, Arch Dis Child) - "Sleep-disordered breathing was present in 85% of 80 children with achondroplasia, with 21% being asymptomatic. Respiratory parameters did not correlate with foramen magnum stenosis severity and improved after 1 year of treatment in those treated with a precision therapy."

Journal • Retrospective data • CNS Disorders • Genetic Disorders • Pediatrics • Respiratory Diseases • Sleep Apnea • Sleep Disorder

Highly Sensitive ELISA System Enables Direct Detection of C-Type Natriuretic Peptide-53 in Plasma Without Extraction (ENDO 2025) - "CNP induces endochondral ossification, vasorelaxation, antifibrosis, and so forth as a local regulator, and its analog, vosoritide, is used for the therapy for achondroplasia... The newly developed CLEIA for CNP-53 demonstrated superior sensitivity and quantitation capabilities, allowing for direct and highly accurate measurement of plasma irCNP-53 concentrations. This method provides a more reliable and reproducible approach than previously reported assays. Utilizing this CLEIA, more detailed physiological and pathophysiological changes of plasma CNP will be elucidated in near future."

Genetic Disorders

Trial of Vosoritide for Treatment of Short Stature in Turner syndrome: Preliminary Data (ENDO 2025) - "Preliminary data are encouraging for safety and efficacy of vosoritide injections in both GH naïve and in previously GH-treated girls with TS to improve height gain in the short term. Longer term data and ongoing enrollment are anticipated to clarify the potential role of vosoritide in TS."

Cardiovascular • Celiac Disease • Dermatology • Gastroenterology • Gastrointestinal Disorder • Genetic Disorders • Hypotension • Immunology • Inflammation • Inflammatory Bowel Disease • Turners Syndrome • FGFR3

Genetics of short stature. (PubMed, Curr Opin Pediatr) - "Incorporating genetic testing into the routine investigation of short stature improves diagnostic accuracy, enables early discussion of prognostic outcomes, and facilitates precision therapy. Timely identification of specific gene variants helps avoid ineffective treatments - such as growth hormone in resistant genotypes - and supports the adoption of personalized medicine interventions."

Journal • Review • Genetic Disorders • Idiopathic Short Stature • Orthopedics • ACAN • FGFR3 • LZTR1 • MATN3

Real-World Safety and Effectiveness of Vosoritide in Achondroplasia: Results from a Single Center in Portugal. (PubMed, Adv Ther) - "Vosoritide showed long-term effectiveness in a real-world Portuguese population of patients with achondroplasia. Vosoritide was also well tolerated, and patients showed good adherence to treatment. These findings were consistent with the outcomes of clinical trials and existing real-world experience."

Journal • Real-world evidence • Genetic Disorders • Orthopedics • FGFR3

Potential efficacy of vosoritide for foramen magnum stenosis in a patient with achondroplasia. (PubMed, Pediatr Int) - No abstract available

Journal • Genetic Disorders

An infant with achondroplasia worsening of the foramen magnum stenosis during early vosoritide treatment. (PubMed, Pediatr Int) - No abstract available

Journal • Genetic Disorders

Effect of Vosoritide Treatment on Growth Velocity and Clinical Outcomes in Children with Achondroplasia: A Single-Center Experience (ESPE-ESE 2025) - "Vosoritid treatment significantly increases the annual growth velocity in children with achondroplasia. No serious adverse effects were observed during the treatment period. These findings support the use of vosoritide as an effective option in the treatment of achondroplasia."

Clinical • Clinical data • Late-breaking abstract • Genetic Disorders • Pain • FGFR3

Vosoritide Therapy in Achondroplasia: Real-World Growth Trajectories in a Small Paediatric Cohort from Kazakhstan (ESPE-ESE 2025) - "Nevertheless, refinements in methodology are warranted. In particular, further evaluation is needed for patients exhibiting suboptimal response to treatment, including ongoing investigations into treatment adherence."

Clinical • Late-breaking abstract • Real-world • Real-world evidence • Genetic Disorders • Pediatrics

Real-World Outcomes of Vosoritide in Achondroplasia: A Single-Center Italian Study (ESPE-ESE 2025) - "The AGV remained stable at T6, T12, and T24, although there was a limit of only 7 patients with 24- month follow-up. Significant improvements in SH/H and SS/SI ratios were observed at T24, with a more pronounced increase in SH in prepubertal subjects compared to pubertal ones, though only 9 patients had Tanner Stage≥2."

Clinical • Late-breaking abstract • Real-world • Real-world evidence • Genetic Disorders

Design of a randomized, multicentre, phase 2 study of vosoritide in children with Turner syndrome, Noonan syndrome, or short stature homeobox-containing gene (SHOX) deficiency (ESPE-ESE 2025) - P2 | "Secondary endpoints include CFB height and height Z-score at 6 months; CFB height, height Z-score, and AGV over 24 months (therapeutic dose versus hGH); and adverse event incidence. This study may support vosoritide as a second-line treatment for these conditions if hGH treatment becomes inadequate."

Clinical • P2 data • Genetic Disorders • Turners Syndrome

Experience with Vosoritide for Achondroplasia: Insights from Kazakhstan (ESPE-ESE 2025) - "Additionally, monitoring and early intervention for complications, enhancing the quality of health professionals, and streamlined regulatory approval procedures are crucial to improving treatment outcomes. Keywords: Vosoritide, Achondroplasia"

CNS Disorders • Genetic Disorders • Mood Disorders • Orthopedics • Respiratory Diseases • Ventriculomegaly

A single center real world experience with Vosoritide for the treatment of children with Achondroplasia. (ESPE-ESE 2025) - "treatment was well tolerated. The difference in SDS based on Achondroplasia standards is more significant than based on WHO standards."

Clinical • Real-world • Real-world evidence • Genetic Disorders • Orthopedics • Pain • FGFR3

Design of a phase 2, randomized, controlled, multicentre study of vosoritide treatment in children with idiopathic short stature (ESPE-ESE 2025) - P2 | "The objectives of the dose-finding phase are to evaluate the safety and efficacy of 3 doses of vosoritide compared with placebo as measured by change from baseline in AGV after 6 months of treatment. The objectives of the long-term phase are to evaluate the safety and efficacy of the therapeutic dose of vosoritide versus hGH on height and height Z-score after long-term treatment."

Clinical • P2 data • Genetic Disorders • Idiopathic Short Stature • ACAN • FGFR3

Real-world data: Effectiveness and safety of Vosoritide in the treatment of Achondroplasia in Chinese population (ESPE-ESE 2025) - "Vosoritide effectively promotes linear growth and improves body proportions in Chinese children with ACH, providing an optimized treatment option. Further studies with larger sample sizes are needed to confirm these findings and explore potential racial differences in treatment efficacy."

Clinical • Real-world • Real-world evidence • Genetic Disorders • Orthopedics • FGFR3

Use of Vosoritide in Children with Achondroplasia - Clinical Experience (ESPE-ESE 2025) - "Although vosoritide increases the growth rate in children with achondroplasia, its effect on height SDS and body proportions is thought to be significant in longer follow-up."

Clinical • CNS Disorders • Genetic Disorders • Obstructive Sleep Apnea • Orthopedics • Respiratory Diseases • Sleep Apnea • Sleep Disorder • Ventriculomegaly • FGFR3 • IGF1

Evaluation of growth velocity in patients with achondroplasia treated with vosoritide (ESPE-ESE 2025) - "Vosoritide treatment had been started on eleven patients with achondroplasia (15 mcg/kg/dose subcutaneously daily). All patients had c.1138G>A heterozygous variant in the FGFR3 gene. The mean age at the start of treatment was 7.01±3.40 years (2.84-12.56 years)."

Clinical • Genetic Disorders • Orthopedics • FGFR3