VOXZOGO Sales Forecast, and Market Size Analysis - 2034

Key Factors Driving VOXZOGO Growth

1. Market Share Gains and New Patient Starts

• VOXZOGO is rapidly gaining market share in the achondroplasia treatment space, establishing itself as the first disease-modifying therapy for this rare genetic condition.
• Rising diagnosis rates and early genetic testing are driving strong growth in new patient starts, particularly in pediatric populations.
• BioMarin's global physician education, patient advocacy engagement, and newborn screening initiatives are accelerating awareness and adoption.

2. Expansion Across Key Indications

• Achondroplasia in Children: Strong uptake in children aged >=2 years, supported by consistent growth velocity improvements and long-term safety data.
• Younger Pediatric Populations: Growing use in younger children as early treatment becomes standard of care.
• Potential Label Expansion: Ongoing clinical programs are exploring earlier intervention and broader age ranges, which could further expand the addressable market.
• Platform Expansion: Success with VOXZOGO strengthens BioMarin's CNP analog platform for other skeletal dysplasias.

3. Geographic Expansion

• VOXZOGO is approved across the US, EU, Japan, and multiple international markets.
• Asia-Pacific is expected to be a high-growth region, driven by improved rare disease diagnosis, government reimbursement programs, and rising awareness.
• BioMarin continues to expand early access programs and reimbursement pathways in emerging markets.

4. New Indication Approvals

• Regulatory agencies have expanded VOXZOGO's label to include children from birth to epiphyseal closure, significantly increasing the eligible patient population.
• Ongoing regulatory filings in additional countries continue to broaden global access.
• These approvals strengthen BioMarin's leadership in genetic bone growth disorders.

5. Strong Rare Disease Volume Momentum

• Achondroplasia remains the primary growth driver, with increasing treatment penetration and high persistence rates.
• BioMarin's recent corporate updates highlight double-digit volume growth in new patient starts, supported by real-world growth velocity data.
• Long-term extension studies continue to reinforce VOXZOGO's disease-modifying benefit.

6. Competitive Differentiation and Market Trends

• First-in-class CNP analog therapy that directly addresses the underlying FGFR3 pathway abnormality.
• Daily subcutaneous administration provides a systemic, disease-modifying alternative to surgical limb-lengthening procedures.
• Broader market trends such as earlier genetic diagnosis, newborn screening, and rare disease reimbursement frameworks support sustained growth.
• Growing reliance on real-world evidence (RWE) is strengthening payer confidence and long-term adoption.

VOXZOGO Recent Developments

In May 2025, BioMarin Pharmaceutical announced new data from studies of VOXZOGO (vosoritide), demonstrating meaningful impact on tibial bowing in children with achondroplasia and investigational early efficacy results in other skeletal conditions. The data were presented at the 2025 Joint Congress of the European Society for Paediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE) in Copenhagen, Denmark and will be shared at the Pediatric Endocrine Society Annual Meeting (PES).

"VOXZOGO Sales Forecast, and Market Size Analysis - 2034" report provides comprehensive insights of VOXZOGO for approved indication like Achondroplasia; as well as potential indications like Hypochondroplasia, Noonan syndrome, Short stature, and Turner's syndrome in the 7MM. A detailed picture of VOXZOGO's existing usage in approved and anticipated entry and performance in potential indications in the 7MM, i.e., the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan for the study period 2020 -2034 is provided in this report along with a detailed description of the VOXZOGO for approved and potential indications. The VOXZOGO market report provides insights about VOXZOGO's sales forecast, mechanism of action (MoA), dosage and administration, as well as research and development including regulatory milestones, along with other developmental activities. Further, it also consists of historical and current VOXZOGO performance, future market assessments inclusive of the VOXZOGO market forecast analysis for approved and potential indications in the 7MM, SWOT, analysts' views, comprehensive overview of market competitors, and brief about other emerging therapies in respective indications. It also provides analysis of VOXZOGO sales forecasts, along with factors driving its market.

VOXZOGO Drug Summary

VOXZOGO (Vosoritide) is a modified synthetic analog of C-type natriuretic peptide (CNP) indicated to increase linear growth in pediatric patients with achondroplasia aged 4 months and older with open epiphyses (growth plates). It binds to the natriuretic peptide receptor B (NPR-B) on chondrocytes in the growth plate, elevating intracellular cyclic guanosine monophosphate (cGMP) levels to antagonize overactive fibroblast growth factor receptor 3 (FGFR3) signaling caused by the GNAS3 gain-of-function mutation, thereby promoting chondrocyte proliferation and hypertrophy, widening the hypertrophic zone, and accelerating endochondral ossification to improve annualized growth velocity (~1.5-2 cm/year gain in trials). Administered daily via subcutaneous injection at 15 μg/kg (using weight-based kits with lyophilized powder reconstituted in sterile water), vosoritide requires blood pressure monitoring due to natriuretic effects and injection site reactions, representing the first targeted therapy for this most common skeletal dysplasia. The report provides VOXZOGO's sales, growth barriers and drivers, post usage and approvals in multiple indications.

Scope of the VOXZOGO Market Report

The report provides insights into:

• A comprehensive product overview including the VOXZOGO MoA, description, dosage and administration, research and development activities in approved indications like Achondroplasia; as well as potential indications like Hypochondroplasia, Noonan syndrome, Short stature, and Turner's syndrome.
• Elaborated details on VOXZOGO regulatory milestones and other development activities have been provided in VOXZOGO market report.
• The report also highlights VOXZOGO's cost estimates and regional variations, reported and estimated sales performance, research and development activities in approved and potential indications across the United States, Europe, and Japan.
• The VOXZOGO market report also covers the patents information, generic entry and impact on cost cut.
• The VOXZOGO market report contains current and forecasted VOXZOGO sales for approved and potential indications till 2034.
• Comprehensive coverage of the late-stage emerging therapies for respective indications.
• The VOXZOGO market report also features the SWOT analysis with analyst views for VOXZOGO in approved and potential indications.

Methodology:

The VOXZOGO market report is built using data and information sourced primarily from internal databases, primary and secondary research and in-house analysis by DelveInsight's team of industry experts. Information and data from the secondary sources have been obtained from various printable and nonprintable sources like search engines, news websites, global regulatory authorities websites, trade journals, white papers, magazines, books, trade associations, industry associations, industry portals and access to available databases.

VOXZOGO Analytical Perspective by DelveInsight

• In-depth VOXZOGO Market Assessment

This VOXZOGO sales market forecast report provides a detailed market assessment of VOXZOGO for approved indication like Achondroplasia; as well as potential indications like Hypochondroplasia, Noonan syndrome, Short stature, and Turner's syndrome in the seven major markets, i.e., the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan. This segment of the report provides current and forecasted VOXZOGO sales data uptil 2034.

• VOXZOGO Clinical Assessment

The VOXZOGO market report provides the clinical trials information of VOXZOGO for approved and potential indications covering trial interventions, trial conditions, trial status, start and completion dates.

VOXZOGO Competitive Landscape

The report provides Insights on competitors and marketed products within the domain, along with a summary of emerging products and their respective launch dates, posing significant competition in the market.

VOXZOGO Market Potential & Revenue Forecast

• Projected market size for the VOXZOGO and its key indications
• Estimated VOXZOGO sales potential (VOXZOGO peak sales forecasts)
• VOXZOGO Pricing strategies and reimbursement landscape

VOXZOGO Competitive Intelligence

• Number of competing drugs in development (pipeline analysis)
• VOXZOGO Market positioning compared to existing treatments
• VOXZOGO Strengths & weaknesses relative to competitors

VOXZOGO Regulatory & Commercial Milestones

• VOXZOGO Key regulatory approvals & expected launch timelines
• Commercial partnerships, licensing deals, and M&A activity

VOXZOGO Clinical Differentiation

• VOXZOGO Efficacy & safety advantages over existing drugs
• VOXZOGO Unique selling points

VOXZOGO Market Report Highlights

• In the coming years, the VOXZOGO market scenario is set to change due to strong adoption, increased prescriptions and broader uptake in multiple immunological indications; which would expand the size of the market.
• The VOXZOGO companies are developing therapies that focus on novel approaches to treat/improve the disease condition, assess challenges, and seek opportunities that could influence VOXZOGO's dominance.
• Other emerging products for Achondroplasia; as well as potential indications like Hypochondroplasia, Noonan syndrome, Short stature, and Turner's syndrome are expected to give tough market competition to VOXZOGO and launch of late-stage emerging therapies in the near future will significantly impact the market.
• A detailed description of regulatory milestones, and developmental activities, provide the current development scenario of VOXZOGO in approved and potential indications.
• Analyse VOXZOGO cost, pricing trends and market positioning to support strategic decision-making in the immunology landscape.
• Our in-depth analysis of the forecasted VOXZOGO sales data uptil 2034 will support the clients in decision-making process regarding their therapeutic portfolio by identifying the overall scenario of VOXZOGO in approved and potential indications.

Key Questions:

• What is the class of therapy, route of administration and mechanism of action of VOXZOGO? How strong is VOXZOGO's clinical and commercial performance?
• What is VOXZOGO's clinical trial status in each individual indications such as Achondroplasia; as well as potential indications like Hypochondroplasia, Noonan syndrome, Short stature, and Turner's syndrome and study completion date?
• What are the key collaborations, mergers and acquisitions, licensing and other activities related to the VOXZOGO Manufacturers?
• What are the key designations that have been granted to VOXZOGO for approved and potential indications? How are they going to impact VOXZOGO's penetration in various geographies?
• What is the current and forecasted VOXZOGO market scenario for approved and potential indications? What are the key assumptions behind the forecast?
• What are the current and forecasted sales of VOXZOGO in the seven major countries, including the United States, Europe (Germany, France, Italy, Spain) and the United Kingdom, and Japan?
• What are the other emerging products available and how are these giving competition to VOXZOGO for approved and potential indications?
• Which are the late-stage emerging therapies under development for the treatment of approved and potential indications?
• How cost-effective is VOXZOGO? What is the duration of therapy and what are the geographical variations in cost per patient?