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Achondroplasia Market Size 2034: EMA, PDMA, FDA Approval, Clinical Trials, Medication, Prevalence, Revenue, Statistics and Companies by DelveInsight
Achondroplasia Market
The Achondroplasia market report provides current treatment practices, emerging drugs, the market share of the individual therapies, and the current and forecasted Achondroplasia market size from 2020 to 2034, segmented by seven major markets. The Report also covers current Achondroplasia treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying potential of the 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=apr
Key Takeaways from the Achondroplasia Market Report
• In May 2025, BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today 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) in National Harbor, Maryland.
• 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.
• In January 2025, Tyra Biosciences, Inc. (Nasdaq: TYRA), a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in Fibroblast Growth Factor Receptor (FGFR) biology, announced today that the U.S. Food and Drug Administration (FDA) cleared its Investigational New Drug (IND) application for TYRA-300 allowing the company to proceed with a Phase 2 clinical trial of TYRA-300 in low-grade, intermediate risk non-muscle invasive bladder cancer (IR NMIBC). This program will be led by newly appointed Dr. Erik Goluboff, SVP, Clinical Development of TYRA, who brings more than thirty years of experience as an academic urologic oncologist, principal investigator, practicing urologist and most recently Principal Medical Lead for GU/GI cancers at Genentech/Roche.
• 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=apr
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=apr
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=apr
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=apr
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 DelveInsight
DelveInsight is a leading Life Science market research and business consulting company recognized for its off-the-shelf syndicated market research reports and customized solutions to firms in the healthcare sector.
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