보솔리티드(VOXZOGO) : 판매 예측 및 시장 규모(2034년)

VOXZOGO Sales Forecast, and Market Size Analysis - 2034

US $ 2,750 ￦ 4,098,000

PDF (Single User License)

PDF 보고서를 1명만 이용할 수 있는 라이선스입니다. 인쇄 가능하며 인쇄물의 이용 범위는 PDF 이용 범위와 동일합니다.

US $ 4,125 ￦ 6,147,000

PDF (2-3 User License)

PDF 보고서를 동일 사업장에서 3명까지 이용할 수 있는 라이선스입니다. 인쇄 가능하며 인쇄물의 이용 범위는 PDF 이용 범위와 동일합니다.

US $ 5,500 ￦ 8,196,000

PDF (Site License)

PDF 보고서를 동일 사업장의 모든 분이 이용할 수 있는 라이선스입니다. 인쇄 가능하며 인쇄물의 이용 범위는 PDF 이용 범위와 동일합니다.

US $ 8,250 ￦ 12,294,000

PDF (Global License)

PDF 보고서를 동일 기업의 모든 분이 이용할 수 있는 라이선스입니다. 인쇄 가능하며 인쇄물의 이용 범위는 PDF 이용 범위와 동일합니다.

한글목차

VOXZOGO의 성장을 견인하는 주요 요인

1. 시장 점유율 확대 및 신규 환자 수 증가

• VOXZOGO는 연골무형성증 치료 영역에서 급속히 시장 점유율을 확대하고, 이 희소 유전성 질환에 대한 최초의 질환 수식 요법으로서의 지위를 확립하고 있습니다.
• 진단률의 상승 및 조기 유전 검사의 실시는 특히 소아 환자층에서 신규 환자수의 현저한 증가를 보여줍니다.
• BioMarin의 세계 의사 교육, 환자 지원 활동, 신생아 스크리닝 노력이 인지도와 채용을 가속화하고 있습니다.

2. 주요 적응증에서의 확대

• 소아의 연골 무형성증 : 2세 이상의 유아에서 채용률이 높고 지속적인 성장 속도 개선과 장기 안전성 데이터가 이를 지원합니다.
• 저연령 소아 환자층 : 조기 치료가 표준 치료가 되는 가운데 저 연령아에서의 사용이 증가하고 있습니다.
• 적응 확대 가능성 : 진행 중인 임상 프로그램은 보다 조기 개입과 폭넓은 연령층을 대상으로 한 연구를 진행하고 있으며, 이로 인해 대상 시장이 더욱 확대될 수 있습니다.
• 플랫폼 확장 : VOXZOGO의 성공은 BioMarin의 CNP 아날로그 플랫폼을 강화하고 다른 골 형성 부전에 대한 응용을 가능하게 합니다.

3. 지리적 확대

• VOXZOGO는 미국, EU, 일본 및 여러 국제 시장에서 승인되었습니다.
• 아시아태평양은 희귀질환의 진단 정밀도 향상, 정부 상환 프로그램, 인지도 증가를 배경으로 높은 성장이 예상되는 지역입니다.
• BioMarin은 신흥 시장에서 조기 액세스 프로그램과 상환 경로의 확충을 계속하고 있습니다.

4. 신규 적응증 승인

• 규제 당국은 VOXZOGO의 적응증을 '출생시부터 골단 폐쇄기까지의 소아'로 확대하여 대상 환자층을 크게 확대했습니다.
• 추가 국가의 규제 당국에 대한 신청이 진행 중이며, 세계의 접근 확대가 계속되고 있습니다.
• 이러한 승인으로 BioMarin은 유전성 골성장 장애 부위의 리더십을 강화하고 있습니다.

5. 희소질환에서의 견조한 판매량의 기세

• 연골무형성증은 치료 보급률의 향상 및 높은 지속율에 의해 계속해서 주요한 촉진요인이 되고 있습니다.
• BioMarin의 최근 기업 정보에 따르면 실제 임상 데이터를 기반으로 한 신규 환자 수의 성장률은 두 자릿수로 추이하고 있습니다.
• 장기연장시험에 의해 VOXZOGO(R)의 질환 수식 효과가 계속 뒷받침되고 있습니다.

6. 경쟁상의 차별화 및 시장 동향

• FGFR3 경로의 근본적인 이상을 직접적으로 개선하는 일등급 CNP 아날로그 요법입니다.
• 매일 피하 투여는 외과적 사지 연장 수술을 대체하는 전신성 질환 변형 치료 옵션을 제공합니다.
• 조기 유전자 진단, 신생아 스크리닝, 희귀질환의 상환 틀 등 광범위한 시장 동향이 지속적인 성장을 지원하고 있습니다.
• 실제 임상 데이터(RWE)에 대한 의존도가 증가함에 따라 지불자의 신뢰 및 장기적인 채택이 강화되었습니다.

본 보고서는 연골무형성증과 같은 승인된 적응증뿐만 아니라 저형성증, 누낭 증후군, 저신장, 터너 증후군 등의 잠재적 적응증에 대해 주요 7개국(미국, EU4(독일, 프랑스, 이탈리아, 스페인), 영국)에서 VOXZOGO에 대한 종합적인 인사이트를 제공합니다. 2020-2034년 주요 7개국에서 VOXZOGO의 승인 적응증 및 잠재적응증에서의 사용상황, 진입 전망, 실적에 대한 상세한 전망을 제공함과 동시에 승인 및 잠재적응증에 대한 VOXZOGO의 상세한 설명을 제시합니다. 또한 이 약제의 매출 예측, 작용기전(MoA), 투여량 및 투여 방법, 규제상의 이정표를 포함한 연구개발 및 기타 활동에 관한 지견을 제공합니다. 또한 과거 및 현재 VOXZOGO의 실적, 미래 시장 평가, SWOT 분석, 분석가의 견해, 경쟁업체의 종합적 개요, 각 적응증에 대한 기타 신흥 치료법의 개요, 매출 예측 분석, 시장 견인 요인 등도 정리했습니다.

목차

제1장 보고서 개요

제2장 VOXZOGO 개요 : 골무형성증 등의 승인 적응증. 연골 저형성증, 누낭 증후군, 저신장, 터너 증후군 등의 잠재적 적응증

• 제품 상세
• 임상 개발
  • 임상 연구
  • 임상시험 정보
  • 안전성 및 유효성
• 기타 개발 활동
• 제품 프로파일

제3장 VOXZOGO : 경쟁 구도(출시 끝난 치료제)

제4장 VOXZOGO : 경쟁 구도(개발 후기 치료제)

제5장 VOXZOGO: 시장 평가

• 승인 적응증 및 잠재적 적응증에 대한 시장 전망
• 주요 7개국 분석
• 국가별 시장 분석
  • 미국
  • 독일
  • 영국

제6장 VOXZOGO : SWOT 분석

제7장 애널리스트의 견해

제8장 부록

제9장 DelveInsight의 서비스 내용

제10장 면책사항

제11장 DelveInsight 정보

제12장 보고서 구매 옵션

AJY

영문 목차

영문목차

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?

Table of Contents

1. Report Introduction

2. VOXZOGO Overview in approved indications like Achondroplasia; as well as potential indications like Hypochondroplasia, Noonan syndrome, Short stature, and Turner's syndrome

• 2.1. Product Detail
• 2.2. VOXZOGO Clinical Development
  • 2.2.1. VOXZOGO Clinical studies
  • 2.2.2. VOXZOGO Clinical trials information
  • 2.2.3. Safety and efficacy
• 2.3. Other Developmental Activities
• 2.4. Product Profile

3. VOXZOGO Competitive Landscape (Marketed Therapies)

4. Competitive Landscape (Late-stage Emerging VOXZOGO Therapies)

5. VOXZOGO Market Assessment

• 5.1. VOXZOGO Market Outlook in approved and potential indications
• 5.2. 7MM Analysis
  • 5.2.1. VOXZOGO Market Size in the 7MM for approved and potential indications
• 5.3. Country-wise Market Analysis
  • 5.3.1. VOXZOGO Market Size in the United States for approved and potential indications
  • 5.3.2. VOXZOGO Market Size in Germany for approved and potential indications
  • 5.3.3. VOXZOGO Market Size in France for approved and potential indications
  • 5.3.4. VOXZOGO Market Size in Italy for approved and potential indications
  • 5.3.5. VOXZOGO Market Size in Spain for approved and potential indications
  • 5.3.6. VOXZOGO Market Size in the United Kingdom for approved and potential indications
  • 5.3.7. VOXZOGO Market Size in Japan for approved and potential indications

6. VOXZOGO SWOT Analysis

7. Analysts' Views

8. Appendix

• 8.1. Bibliography
• 8.2. Report Methodology

9. DelveInsight Capabilities

10. Disclaimer

11. About DelveInsight

12. Report Purchase Options