Vosoritide

In patients with achondroplasia, endochondral bone growth is negatively regulated due to a gain of function mutation in fibroblast growth factor receptor 3 ( FGFR3 ). Binding of vosoritide to natriuretic peptide receptor-B (NPR-B) antagonizes FGFR3 downstream signaling by inhibiting the extracellular signal-regulated kinases 1 and 2 (ERK1/2) in the mitogen-activated protein kinase (MAPK) pathway at the level of rapidly accelerating fibrosarcoma serine/threonine protein kinase (RAF-1).

• VOXZOGO is indicated to increase linear growth in pediatric patients with achondroplasia with open epiphyses. This indication is approved under accelerated approval based on an improvement in annualized growth velocity [see Clinical Studies (14) ] .

• None None.contraindicated

Ensure adequate food and fluid intake prior to administration. ( 2.1 ) Recommended dosage is based on patient's actual body weight. Administer VOXZOGO subcutaneously once daily. ( 2.2 ) Reconstitute prior to use. Injection volume is based on both patient's weight and concentration of reconstituted VOXZOGO. ( 2.2 ) Monitor growth and adjust dosage according to actual body weight. Permanently discontinue upon closure of epiphyses. ( 2.3 ) See full prescribing information for reconstitution, dilution, and administration instructions. ( 2.4 ) 2.1 Important Instructions Prior to Administration of VOXZOGO To reduce the risk of low blood pressure and its associated signs and symptoms, instruct the caregiver and patient that the patient should [see Warnings and Precautions (5.1) ] : Have adequate food intake prior to VOXZOGO administration. Drink approximately 240 to 300 mL of fluid in the hour prior to VOXZOGO administration. 2.2 Recommended Dosage and Administration The recommended dosage of VOXZOGO is based on the patient's actual body weight (see Table 1 ). VOXZOGO is administered by subcutaneous injection once daily [see Dosage and Administration (2.4) ] . Inject VOXZOGO at approximately the same time each day, if possible. The volume of VOXZOGO to be administered (injection volume) is based on the patient's actual body weight and the concentration of reconstituted VOXZOGO (0.8 mg/mL or 2 mg/mL) (see Table 1 ). VOXZOGO must be reconstituted prior to use [see Dosage and Administration (2.4) ] .

Risk of Low Blood Pressure : Transient decreases in blood pressure have been reported. Instruct patients to be well-hydrated and have adequate food intake prior to administration of VOXZOGO ( 5.1 ) 5.1 Risk of Low Blood Pressure Transient decreases in blood pressure were observed in clinical studies of VOXZOGO. Subjects with significant cardiac or vascular disease and patients on anti-hypertensive medicinal products were excluded from participation in VOXZOGO clinical trials. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, fatigue and/or nausea), instruct patients to be well hydrated and have adequate food intake prior to administration of VOXZOGO [see Dosage and Administration (2.1) and Adverse Reactions (6.1) ].

The following clinically significant adverse reactions are described elsewhere in the labeling: Risk of Low Blood Pressure [see Warnings and Precautions (5.1) ] Most common adverse reactions (>10%) are injection site erythema, injection site swelling, rash, vomiting, injection site urticaria, arthralgia, decreased blood pressure, and gastroenteritis. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact BioMarin Pharmaceutical Inc. at 1-866-906-6100, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. Pediatric Patients 5 Years of Age and Older VOXZOGO was studied in a 52-week, randomized, double-blind, placebo-controlled trial in 121 subjects with achondroplasia (Study 1) [see Clinical Studies (14) ] . The subjects' ages ranged from 5.1 to 14.9 years with a mean of 8.7 years. Sixty four (53%) subjects were male and 57 (47%) were female. Overall, 86 (71%) subjects were White, 23 (19%) were Asian, 5 (4%) were Black or African American, and 7 (6%) were classified as "multiple" race. The demographic and baseline characteristics were balanced between treatment groups. The subjects received either VOXZOGO 15 mcg/kg, or placebo administered subcutaneously once daily.

Renal Impairment: Not recommended in patients with eGFR < 60 mL/min/1.73 m 2 . ( 8.6 ) 8.1 Pregnancy Risk Summary There are no available data on vosoritide use in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. In animal reproduction studies, there was no evidence of embryo-fetal toxicity or congenital malformations when pregnant rats and rabbits were administered vosoritide subcutaneously at doses equivalent to 14-times and 200-times, respectively, the exposure at the maximum recommended human dose (MRHD) (see Data ). The estimated background risk of major birth defects for the indicated population is higher than the general population. The estimated background risk of miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Data Animal Data In an embryofetal developmental toxicity study in rats, vosoritide was administered at 90, 270, 540 mcg/kg once daily by subcutaneous injection during the period of major organogenesis from gestation day (GD) 6 – 17.

Metabolism

The area under the concentration-time curve (AUC) and peak concentration (C max ) of vosoritide increased greater than proportionally following subcutaneous administration to pediatric subjects with achondroplasia in the dose range of 7.5 to 30.0 mcg/kg. The pharmacokinetics of vosoritide were evaluated in 58 subjects aged 5 to 13 years with achondroplasia who received subcutaneous injections of vosoritide 15 mcg/kg once daily for 52 weeks.

• Nov 19, 2021NDANDA214938Biomarin Pharm

1. 1Dose Calculation Error1100%
2. 2Product Preparation Error1100%
3. 3Product Prescribing Error1100%

The 10 most recently updated of 17 ClinicalTrials.gov registrations naming Vosoritide as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
  Recruiting · Phase 2 · Phase 3 · Interventional · 160 enrolled · BioMarin Pharmaceutical
  NCT07441876updated 2026-05-29
• A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
  Recruiting · Phase 2 · Interventional · 100 enrolled · BioMarin Pharmaceutical
  NCT06382155updated 2026-05-22
• A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
  Recruiting · Phase 2 · Interventional · 30 enrolled · BioMarin Pharmaceutical
  NCT06668805updated 2026-05-11
• A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
  Recruiting · Phase 2 · Interventional · 60 enrolled · BioMarin Pharmaceutical
  NCT07126262updated 2026-03-31
• Vosoritide for Selected Genetic Causes of Short Stature
  Active not recruiting · Phase 2 · Interventional · 56 enrolled · Andrew Dauber
  NCT04219007updated 2026-03-18
• A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
  Active not recruiting · Phase 2 · Interventional · 30 enrolled · BioMarin Pharmaceutical
  NCT02724228updated 2026-03-13
• An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
  Active not recruiting · Phase 3 · Interventional · 119 enrolled · BioMarin Pharmaceutical
  NCT03424018updated 2026-03-13
• A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
  Active not recruiting · Phase 2 · Interventional · 20 enrolled · BioMarin Pharmaceutical
  NCT04554940updated 2026-03-13
• An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
  Active not recruiting · Phase 2 · Interventional · 73 enrolled · BioMarin Pharmaceutical
  NCT03989947updated 2026-03-13
• Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI
  Active not recruiting · Phase 1 · Phase 2 · Interventional · 6 enrolled · University of California, San Francisco
  NCT05845749updated 2026-03-10

Frequently asked questions

How does Vosoritide work?

In patients with achondroplasia, endochondral bone growth is negatively regulated due to a gain of function mutation in fibroblast growth factor receptor 3 ( FGFR3 ). Binding of vosoritide to natriuretic peptide receptor-B (NPR-B) antagonizes FGFR3 downstream signaling by inhibiting the extracellular signal-regulated kinases 1 and 2 (ERK1/2) in the mitogen-activated protein kinase (MAPK) pathway at the level of rapidly accelerating fibrosarcoma serine/threonine protein kinase (RAF-1).

What is Vosoritide used for?

According to FDA labeling, Vosoritide carries indications including: VOXZOGO is indicated to increase linear growth in pediatric patients with achondroplasia with open epiphyses. This indication is approved under accelerated approval based on an improvement in annualized growth velocity [see Clinical Studies (14) ] .. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Vosoritide?

Vosoritide is classified as Other drugs affecting bone structure and mineralization, C-type Natriuretic Peptide Analog, Gene Editing or Modification, Decreased Fibroblast Migration.

What are the brand names for Vosoritide?

Vosoritide is marketed under brand names including Voxzogo.

What are the contraindications for Vosoritide?

Vosoritide labeling lists contraindications including: None None.. Always consult the full prescribing information and a clinician.