Achondroplasia Market Overview: Epidemiology, Treatment Landscape, Key Players | Ascendis Pharma, QED Therapeutics (BridgeBio), Novartis, Sanofi, RIBOMIC

DelveInsight's Achondroplasia Market Insights, Epidemiology, and Market Forecast 2034 report provides a detailed outlook of the current and future Achondroplasia treatment market, segmented within countries, by therapies, and by classes. Further, the market of each region is then segmented by each therapy to provide a detailed view of the current and future Achondroplasia market share of all therapies.

Achondroplasia treatments, including VOXZOGO (vosoritide), RBM-007, SAR-442501, low-dose infigratinib, and others, are anticipated to drive growth in the Achondroplasia market in the coming years.

DelveInsight has released a new report titled "Achondroplasia - Market Insights, Epidemiology, and Market Forecast 2034", offering comprehensive insights into the disorder, along with historical and projected epidemiology trends and market dynamics across the United States, the EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

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Some of the key facts of the Achondroplasia Market Report:

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The achondroplasia market is projected to witness steady growth, fueled by progress in genetic therapies, improved diagnostic practices, and rising R&D investments.

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In March 2025, Ascendis Pharma submitted a New Drug Application (NDA) to the FDA for TransCon CNP (navepegritide), a once-weekly injectable prodrug designed to deliver sustained exposure to C-type natriuretic peptide (CNP). This investigational therapy aims to promote bone growth and muscle function in children with achondroplasia. The application is backed by data from three randomized, double-blind, placebo-controlled trials, including up to three years of open-label extension results.

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Earlier, in September 2024, the FDA granted Breakthrough Therapy Designation to oral infigratinib (BridgeBio Pharma) for pediatric achondroplasia, following Phase 2 PROPEL 2 trial data that demonstrated durable improvements in annualized height velocity and body proportionality over 18 months. If approved, infigratinib would represent the first oral treatment option for achondroplasia.

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In 2023, the U.S. recorded approximately 14,500 diagnosed prevalent cases, with market dynamics evolving due to the introduction of innovative therapies and rising healthcare expenditures. A major milestone was the FDA's 2021 approval of BioMarin's VOXZOGO (vosoritide), the first targeted therapy for achondroplasia. However, significant unmet needs remain in areas such as early diagnosis, targeted interventions, and holistic patient management.

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DelveInsight's latest report, "Achondroplasia - Market Insights, Epidemiology, and Forecast 2034", provides detailed analyses of market trends, disease epidemiology, and growth opportunities across the U.S., EU4, the UK, and Japan.

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By 2023, combined diagnosed prevalent cases in the U.S. and Japan reached nearly 18,000, with females slightly outnumbering males in the U.S. (51.2% vs. 48.8%), potentially due to underlying biological factors. Japan reported around 3,000 diagnosed cases in the same year.

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Key companies active in this space include BioMarin Pharmaceuticals, Tyra Biosciences, RIBOMIC Inc., BridgeBio Pharma, Sanofi, and others. Promising therapies currently in development include VOXZOGO (vosoritide), RBM-007, SAR-442501, low-dose infigratinib, among additional pipeline candidates.

Achondroplasia Overview

Achondroplasia is a rare genetic disorder triggered by mutations in the FGFR3 gene, which impair bone development and result in dwarfism. The mutation slows growth plate activity, impacting nearly all bones. While the condition follows an autosomal dominant inheritance pattern, more than 80% of cases stem from new (de novo) mutations in families with no prior history.

Distinctive physical characteristics include shortened limbs, enlarged head with a prominent forehead, flat nasal bridge, spinal curvature, and trident-shaped fingers. On average, adult height is approximately 4 feet 2 inches in females and 4 feet 4 inches in males.

Achondroplasia Market Outlook

Achondroplasia, the most common genetic cause of dwarfism, has historically been managed through supportive care and surgical interventions to treat complications such as bone deformities and spinal abnormalities-without addressing the fundamental growth defect.

Recent advances are reshaping this approach. Vosoritide, a daily injectable that targets the FGFR3 pathway, has demonstrated effectiveness in improving growth velocity in children. A next-generation therapy in development, TransCon CNP, is designed to deliver sustained benefits with less frequent dosing. Meanwhile, ongoing gene therapy research seeks to directly correct the FGFR3 mutation, offering the potential for a long-term cure.

Together, these innovations mark a transformative shift from merely managing symptoms to treating the root cause of achondroplasia.

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Achondroplasia Marketed Drugs

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VOXZOGO (vosoritide): BioMarin Pharmaceuticals

Achondroplasia Emerging Drugs

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RBM-007: RIBOMIC Inc.

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SAR-442501: Sanofi

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Low dose infigratinib: BridgeBio Inc.

Scope of the Achondroplasia Market Report

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Study Period: 2020-2034

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Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

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Key Achondroplasia Companies: Ascendis Pharma, QED Therapeutics (BridgeBio), Novartis, Sanofi, RIBOMIC, and others

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Key Achondroplasia Therapies: VOXZOGO (vosoritide), RBM-007, SAR-442501, Low dose infigratinib, and others

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Achondroplasia Therapeutic Assessment: Achondroplasia current marketed and Achondroplasia emerging therapies

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Achondroplasia Market Dynamics: Achondroplasia market drivers and Achondroplasia market barriers

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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies

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Achondroplasia Unmet Needs, KOL's views, Analyst's views, Achondroplasia Market Access and Reimbursement

To know what's more in our Achondroplasia report, visit https://www.delveinsight.com/report-store/achondroplasia-market?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr

Key benefits of the Achondroplasia Market Report:

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Achondroplasia market report covers a descriptive overview and comprehensive insight of the Achondroplasia Epidemiology and Achondroplasia market in the 7MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan).

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The Achondroplasia market report provides insights into the current and emerging therapies.

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The Achondroplasia market report provides a global historical and forecasted market covering drug outreach in 7MM.

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The Achondroplasia market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Achondroplasia market.

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Table of Contents

1. Report Introduction

2. Executive Summary

3. SWOT analysis

4. Achondroplasia Patient Share (%) Overview at a Glance

5. Achondroplasia Market Overview at a Glance

6. Achondroplasia Disease Background and Overview

7. Achondroplasia Epidemiology and Patient Population

8. Country-Specific Patient Population of Achondroplasia

9. Achondroplasia Current Treatment and Medical Practices

10. Unmet Needs

11. Achondroplasia Emerging Therapies

12. Achondroplasia Market Outlook

13. Country-Wise Achondroplasia Market Analysis (2020-2034)

14. Market Access and Reimbursement of Therapies

15. Market drivers

16. Market barriers

17. Appendix

18. Achondroplasia Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

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Related Reports:

Achondroplasia Pipeline Insights, DelveInsight

"Achondroplasia Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Achondroplasia market. A detailed picture of the Achondroplasia pipeline landscape is provided, which includes the disease overview and Achondroplasia treatment guidelines.

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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