Voxzogo (vosoritide) / BioMarin - LARVOL DELTA

Three-dimensional craniofacial imaging in children with achondroplasia treated with vosoritide. (PubMed, Genet Med Open) - "Our findings indicate that vosoritide treatment leads to a measurable improvement in facial phenotype in achondroplasia, highlighting its potential utility beyond enhancing linear growth. We discuss the role of pathway-disrupting medications, such as vosoritide, in addressing the broader spectrum of medical issues experienced by children with achondroplasia."

Journal • CNS Disorders • Depression • Genetic Disorders • Orthopedics • Psychiatry • FGFR3

Functional Change in Three Adolescents With Achondroplasia Following 12 Months Treatment With Vosoritide-A Case Series. (PubMed, J Paediatr Child Health) - No abstract available

Journal • Genetic Disorders

Real-world safety and age-dependent effectiveness of vosoritide in achondroplasia: A single-center retrospective analysis of transition from growth hormone to vosoritide. (PubMed, Bone) - "Injection site pain occurred in three patients, leading to one discontinuation. Vosoritide improves linear growth in children with ACH, including those previously treated with GH, showing greater benefit when started at ≥4 years, though earlier use may offer advantages."

Journal • Real-world evidence • Retrospective data • Genetic Disorders • Orthopedics • Pain

Clinical Expert Opinion on the Impact of Vosoritide on Health and Function in Children With Achondroplasia (ISPOR-EU 2025) - "Clinical expert experiences offer new insights into vosoritide's impact on achondroplasia, including functional, mobility, emotional, social outcomes, and caregiver burden. These insights can help clinicians discuss treatment with patients and inform future studies."

Clinical • Genetic Disorders

Effect of Vosoritide therapy on IGF-I and Endogenous C-type Natriuretic Peptide in Hypochondroplasia. (PubMed, J Clin Endocrinol Metab) - "In children with HCH, IGF-1 levels are reduced and NTproCNP levels are elevated at baseline. A correlation between changes in these levels during treatment suggests a possible interaction between IGF-1 and CNP signaling."

Journal • IGF1

Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia (clinicaltrials.gov) - P3 | N=80 | Active, not recruiting | Sponsor: BioMarin Pharmaceutical | Enrolling by invitation ➔ Active, not recruiting

Enrollment closed

Thanatophoric Dysplasia Type 1 Treated with Vosoritide: A Case Report. (PubMed, Horm Res Paediatr) - "Vosoritide was well tolerated and improved growth velocity and lung function in this long‑term TD1 survivor, suggesting therapeutic potential even in severe FGFR3 overactivation. Given TD1's rarity, larger studies and further off‑label experience are essential to validate these findings."

Journal • Genetic Disorders • Orthopedics • FGFR3

Evaluation of sleep-related breathing disorders and polysomnography findings in pediatric patients with achondroplasia (ERS 2025) - "The most common symptom at presentation was snoring (67.6%), and 8 patients (23.5%) had witnessed apnea.In 11 patients(32.4%),no sleep symptoms were present.PSG results showed 32 patients (94.1%) with sleep apnea, 29(90.6%) of whom were diagnosed with obstructive sleep apnea syndrome, categorized as mild,moderate,or severe.Central sleep apnea syndrome was identified in 3 patients,two of whom had FMS on cranial imaging.Follow-up PSG in 8 patients showed no change in severity in 4 cases,improvement in 3, and worsening in one.AHI(Apnea-Hypopnea Index) did not significantly correlate with gender,adenoidectomy,or FMS presence.Patients not receiving vosoritide had a higher median AHI compared to those who received the treatment(p=0.042). This study highlights the high prevalence of SDB in achondroplasia and the importance of PSG, even in asymptomatic patients,at least one PSG evaluation should be performed, and repeated assessments should be considered when necessary."

Clinical • Genetic Disorders • Obstructive Sleep Apnea • Orthopedics • Pediatrics • Respiratory Diseases • Sleep Disorder • FGFR3

The CNP analogue vosoritide mediates PDE2-sensitive anti-arrhythmogenic effects in mouse hearts with STZ-induced type 1 diabetes. (PubMed, Basic Res Cardiol) - "Importantly, VO did not affect the QT interval, action potential duration, or contraction of cardiomyocytes from diabetic mice. Thus, the modified natriuretic peptide VO may serve as a promising therapeutic option to prevent lethal arrhythmias in susceptible diabetic patients."

Journal • Preclinical • Cardiomyopathy • Cardiovascular • Diabetes • Metabolic Disorders • Myocardial Infarction • Reperfusion Injury • Type 1 Diabetes Mellitus

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