Press release
Achondroplasia Treatment Market Size in 7MM is expected to grow at a decent CAGR by 2034, estimates DelveInsight | Ascendis Pharma, QED Therapeutics (BridgeBio), Novartis, Sanofi, RIBOMIC
Achondroplasia Market
Discover which therapies are expected to grab the Achondroplasia Market Share @ Achondroplasia Market Outlook- https://www.delveinsight.com/sample-request/achondroplasia-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
Key Takeaways from the Achondroplasia Market Report
• In March 2025, Tyra Biosciences Inc. announced a phase 2 study to determine the safety, tolerability, and identify potentially effective dose(s) of TYRA-300, a fibroblast growth factor receptor (FGFR)-3 selective tyrosine kinase inhibitor, in children 3 to 10 years of age with achondroplasia with open growth plates that will examine three cohorts of children: the Sentinel Safety Cohort, Cohort 1, and Cohort 2.
• In March 2025, Ascendis Pharma A/S announced a phase 2 study, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg CNP/kg of Navepegritide (TransCon CNP) administered SC once-weekly for 52 weeks in infants with genetically verified heterozygous ACH, aged 0 to < 2 years at the time of randomization.
• The total diagnosed prevalent cases of Achondroplasia in Japan and the US were ~18,000 in 2023, and they are expected to rise in the future.
• In the US, achondroplasia affects a slightly higher percentage of females (51.2%) compared to males (48.8%), possibly due to genetic or biological factors contributing to the marginally more significant occurrence among females.
• Japan had nearly 3000 total diagnosed prevalent cases of Achondroplasia in 2023.
• According to the epidemiology model, the age-specific cases are divided into nine groups: 0-4, 5-10, 11-15, 16-20, 21-30, 31-40, 41-50, 51-60, and >60. Based on DelveInsight's estimates, the people in the age group 5-10 years of age were affected the most in the US, accounting for approximately 4000 cases in 2023.
• Achondroplasia prevalence is estimated at 1 in 15,000 to 40,000 live births, making it one of the most common skeletal dysplasias.
• The leading Achondroplasia Companies such as Ascendis Pharma, QED Therapeutics (BridgeBio), Novartis, Sanofi, RIBOMIC, and others.
• Promising Achondroplasia Pipeline Therapies such as VOXZOGO (vosoritide), Infigratinib, RBM-007, SAR-442501, Recombinant human growth hormone, Infigratinib, TransCon CNP, Infigratinib, vosoritide, BMN 111, and others.
Stay ahead in the Achondroplasia Therapeutics Market with DelveInsight's Strategic Report @ Achondroplasia Market Outlook- https://www.delveinsight.com/sample-request/achondroplasia-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
Epidemiology Segmentation in the 7MM
• Total Achondroplasia Prevalence Cases
• Achondroplasia Prevalent Cases by severity
• Achondroplasia Gender-specific Prevalence
• Achondroplasia Diagnosed Cases of Episodic and Chronic
Download the report to understand which factors are driving Achondroplasia Epidemiology Trends @ Achondroplasia Prevalence- https://www.delveinsight.com/sample-request/achondroplasia-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
Marketed Achondroplasia Drugs
• VOXZOGO (vosoritide): BioMarin Pharmaceuticals
VOXZOGO (vosoritide) by BioMarin Pharmaceuticals is a groundbreaking treatment for achondroplasia. It works by mimicking C-type natriuretic peptide (CNP), which counteracts the overactive FGFR3 pathway that impairs bone growth. Administered via daily subcutaneous injections, Voxzogo enhances growth velocity in children with achondroplasia.
Emerging Achondroplasia Drugs
• RBM-007: RIBOMIC Inc.
RBM-007, developed by RIBOMIC Inc., is an RNA aptamer targeting fibroblast growth factor 2 (FGF2) for the treatment of achondroplasia. Its mechanism of action involves inhibiting FGF2, thereby promoting bone growth and reducing the effects of the FGFR3 mutation responsible for achondroplasia. RBM-007 is administered via subcutaneous injection. It is currently in Phase IIa clinical trials, assessing its safety and preliminary efficacy in affected individuals.
• SAR-442501: Sanofi
SAR-442501, developed by Sanofi, is a novel treatment for achondroplasia. It functions as a selective antagonist of the fibroblast growth factor receptor 3 (FGFR3), which is implicated in inhibiting bone growth in achondroplasia. By blocking this receptor, SAR-442501 aims to enhance skeletal growth and improve height outcomes. The drug is administered via subcutaneous injection. The upreACH Phase II study of SAR442501 in pediatric patients with achondroplasia will remain ongoing but is not expected to advance further.
• Low dose infigratinib: BridgeBio Inc.
Infigratinib is an oral small molecule in development for the treatment of FGFR-driven conditions, including achondroplasia, a bone growth disorder in children. Over-activating FGFR3 mutations drive downstream MAPK and STAT1 signaling that aberrates growth plate development, thereby causing disproportionate short stature and severe health complications stemming from cranial and spinal issues. Low-dose infigratinib has the potential to help children due to its direct inhibition of the mutant FGFR3 receptor. BridgeBio initiated PROPEL 3, a Phase III clinical trial studying the efficacy and safety of infigratinib in children with achondroplasia. Both the US FDA and the EU EMA indicated the trial design for PROPEL 3 would be acceptable as a registrational study to support a marketing application for the treatment of children with achondroplasia.
To learn more about Achondroplasia treatment guidelines, visit @ Achondroplasia Treatment Market Landscape- https://www.delveinsight.com/sample-request/achondroplasia-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
Achondroplasia Therapies and Companies
• VOXZOGO (vosoritide): BioMarin Pharmaceuticals
• Infigratinib: BridgeBio/ QED Therapeutics
• RBM-007: Ribomic Inc
• Sanofi: SAR-442501
• Recombinant human growth hormone: Changchun GeneScience Pharma
• Infigratinib: QED Therapeutics, Inc.
• TransCon CNP: Ascendis Pharma A/S
• Infigratinib: QED Therapeutics, Inc
• vosoritide: BioMarin Pharmaceuticals
• BMN 111: BioMarin Pharmaceutical
Scope of the Achondroplasia Market Report
• Study Period: 2020-2034
• Coverage: 7MM
• Achondroplasia Companies: BioMarin Pharmaceuticals, BridgeBio/ QED Therapeutics , Ribomic Inc, Sanofi, Changchun GeneScience Pharma, QED Therapeutics, Inc., Ascendis Pharma A/S, QED Therapeutics, Inc, BioMarin Pharmaceuticals, BioMarin Pharmaceutical, and others
• Achondroplasia Therapies: VOXZOGO (vosoritide), Infigratinib, RBM-007, SAR-442501, Recombinant human growth hormone, Infigratinib, TransCon CNP, Infigratinib, vosoritide, BMN 111, and others
• Achondroplasia Therapeutic Assessment: Achondroplasia current marketed and Achondroplasia emerging therapies
• Achondroplasia Market Dynamics: Achondroplasia market drivers and Achondroplasia market barriers
• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
• Achondroplasia Unmet Needs, KOL's views, Analyst's views, Achondroplasia Market Access and Reimbursement
Discover more about Achondroplasia Drugs in development @ Achondroplasia Clinical Trials Assessment- https://www.delveinsight.com/sample-request/achondroplasia-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
Table of Content
1. Key Insights
2. Report Introduction
3. Achondroplasia Market Overview
4. Methodology of Achondroplasia Epidemiology and Market
5. Executive Summary of Achondroplasia
6. Key Events
7. Disease Background and Overview of Achondroplasia
8. Epidemiology and Patient Population
9. Patient Journey
10. Marketed Therapies
11. Emerging Therapies
12. Achondroplasia: Market Analysis
13. Key Opinion Leaders' Views
14. SWOT Analysis
15. Unmet needs
16. Market Access and Reimbursement
17. Appendix
18. Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
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About Us
DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.
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