Voxzogo (vosoritide) / BioMarin - LARVOL DELTA

A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment (clinicaltrials.gov) - P2 | N=30 | Active, not recruiting | Sponsor: BioMarin Pharmaceutical | Recruiting ➔ Active, not recruiting

Enrollment closed • Genetic Disorders

A Case of Inadvertent Overdose of Vosoritide Injection in a Three-Month-Old Infant With Achondroplasia. (PubMed, Cureus) - "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."

Journal • Genetic Disorders • Hypotension • FGFR3

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 | Trial completion date: Dec 2040 ➔ Oct 2044 | Trial primary completion date: Dec 2040 ➔ Oct 2044

Trial completion date • Trial primary completion date

The Ethics of Growth-Promoting Pharmaceuticals for Achondroplasia. (PubMed, Am J Bioeth) - "This paper examines the key ethical issues surrounding vosoritide, including the distinction between its medical and social benefits, its potential impact on identity, the inclusion of disability communities in study design, the limits of parental decision-making, and the justification for public reimbursement. We argue that ethical evaluation requires robust empirical evidence on both medical and quality-of-life outcomes, inclusion of disability communities in the research process, and long-term psychosocial follow-up and mandated data sharing of treated children."

Journal • Endocrine Disorders • Genetic Disorders

A Phase 3 Trial of Vosoritide in Children with Hypochondroplasia. (PubMed, NEJM Evid) - P3 | "One year of vosoritide treatment significantly increased linear growth in children with hypochondroplasia. (Funded by BioMarin Pharmaceutical; ClinicalTrials.gov number, NCT06455059.)."

Journal • P3 data • Genetic Disorders • FGFR3

Clinical experience with vosoritide in infants and young children (ESPE 2026) - No abstract available

Clinical • Genetic Disorders

Holistic impact of vosoritide treatment (ESPE 2026) - No abstract available

Genetic Disorders

Vosoritide Increases Growth Velocity in Children With Hypochondroplasia: Phase 3 Trial Results (ESPE 2026) - No abstract available

Clinical • Late-breaking abstract • P3 data

Vosoritide safety and effectiveness in young children with achondroplasia aged ≤3 years and with up to 36 months of follow-up from the Japanese post-marketing safety surveillance study (111-604) (ESPE 2026) - No abstract available

Clinical • P4 data • Genetic Disorders

Vosoritide Phase 2 Extension Trial in Children with Hypochondroplasia: Three-Year Effects on Growth, Body Proportions and Collagen X Biomarker Activity (ESPE 2026) - No abstract available

Biomarker • Clinical • P2 data

Early IGF-1 Response is a Predictive Marker for Height Improvement in Children with Achondroplasia Following Two Years of Vosoritide Therapy (ESPE 2026) - No abstract available

Biomarker • Clinical • Genetic Disorders • IGF1

Early IGF-1 Response is a Predictive Marker for Height Improvement in Children with Achondroplasia Following Two Years of Vosoritide Therapy (ESPE 2026) - No abstract available

Biomarker • Clinical • Genetic Disorders • IGF1

Safety and Efficacy of Vosoritide for Short Stature in Turner Syndrome: Results from a Single Center Investigator-Initiated Trial (ESPE 2026) - No abstract available

Clinical • Genetic Disorders • Turners Syndrome

Slipped Capital Femoral Epiphyses during Vosoritide Therapy for Short Stature (ESPE 2026) - No abstract available

Late-breaking abstract

Vosoritide Improves Height in Children with Heterozygous Variants in NPR2: Year 2 Results from a Phase 2 Clinical Trial (ESPE 2026) - No abstract available

Clinical • P2 data

Long-term effects of vosoritide on bone density and body composition in children with hypochondroplasia: a 3-year prospective DXA study (ESPE 2026) - No abstract available

Clinical

Retrospective Study of Foramen Magnum Development in Patients With Achondroplasia Starting Vosoritide Before Age Three (ESPE 2026) - No abstract available

Retrospective data • Genetic Disorders

The real world effects of Vosoritide on craniofacial shape and function in achondroplasia: a multinational survey (ESPE 2026) - No abstract available

Clinical • Real-world • Real-world effectiveness • Real-world evidence • Genetic Disorders

A phase 3, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of vosoritide in children with hypochondroplasia: CANOPY HCH-3 study design. (PubMed, Ther Adv Endocrinol Metab) - "Written informed consent from the participant, or parent or legal guardian, was obtained prior to any study-related procedures being performed. CANOPY HCH-3 will provide further evidence for the efficacy and safety of vosoritide in children with hypochondroplasia."

Clinical • Journal • P3 data • Genetic Disorders • Orthopedics • FGFR3

Perceived 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. (PubMed, Horm Res Paediatr) - "Genetic short stature significantly impacts health-related quality of life, as reported by both children and their caregivers. Both children and caregivers noted emotional, physical, and social impacts as well as impacts on daily activities. Vosoritide treatment was associated with meaningful improvements in physical impacts and impacts on daily activities."

HEOR • Journal • Genetic Disorders • ACAN

From FGFR3 Hyperactivation to Disease-Modifying Therapy in Pediatric Achondroplasia: Molecular Mechanisms, Clinical Evidence, and Emerging Treatments. (PubMed, Children (Basel)) - "Achondroplasia is increasingly understood as a disorder of dysregulated growth plate signaling rather than solely a condition of reduced stature. Although vosoritide has established the feasibility of disease-modifying treatment, substantial uncertainty remains regarding final adult height, skeletal proportionality, cranio-spinal development, orthopedic outcomes, and long-term safety. Future progress will depend on mechanistically informed therapeutic combinations, improved biomarkers, advanced cellular and animal models, and long-term clinical and real-world evidence."

Journal • Review • Genetic Disorders • Orthopedics • Pediatrics • CTNNB1 • DKK1 • FGFR3

Slipped Capital Femoral Epiphysis during Vosoritide Therapy for Short Stature: A Case Series. (PubMed, Horm Res Paediatr) - P2 | "Vosoritide treatment may be associated with a higher risk of SCFE in some individuals with genetic forms of short stature such as Turner syndrome and Aggrecan mutations. The causal mechanisms and contributory factors to the predisposition for valgus deformities noted with vosoritide therapy in children with these genetic conditions warrant further study."

Journal • Genetic Disorders • Turners Syndrome • ACAN

Real-World Evidence on the Effectiveness and Safety of Vosoritide in Latin American Patients With Achondroplasia (EVOLAC). (PubMed, Am J Med Genet A) - "In real-world clinical practice across Latin America, vosoritide demonstrated rapid and sustained gains in linear growth, favorable tolerability, high adherence, and meaningful benefits as perceived by caregivers. These results reinforce the generalizability of vosoritide therapy in heterogeneous healthcare systems and highlight the relevance of real-world evidence for informing treatment decisions in rare diseases."

Journal • Genetic Disorders • Orthopedics • Rare Diseases • FGFR3

Vosoritide Increases Growth Velocity in Children With Hypochondroplasia: Phase 3 Trial Results (ASBMR 2026) - No abstract available

Clinical • Late-breaking abstract • P3 data

Healthcare and Psychosocial Needs in Achondroplasia Across the Lifespan: Developmental Functioning, Multidisciplinary Care, and Family-Centered Outcomes. (PubMed, Healthcare (Basel)) - "Achondroplasia should be managed as a lifelong multisystem condition rather than solely as a disorder of short stature. Standardized surveillance, multidisciplinary coordination, planned transition to adult care, and patient- and family-centered management are essential for improving function, autonomy, long-term health outcomes, and quality of life."

Journal • Review • Cardiovascular • CNS Disorders • Genetic Disorders • Musculoskeletal Diseases • Obesity • Orthopedics • Otorhinolaryngology • Pain • Sleep Disorder • FGFR3