Achondroplasia Market Size is Set for Rapid Growth as Innovative Treatments and Rising Awareness Drive Demand by 2034

The Achondroplasia market size is expected to increase during the forecast period with the increasing prevalence, disease awareness, and promising emerging treatment options by leading companies such as RIBOMIC Inc., Sanofi, BridgeBio Inc., Recombinant: Changchun GeneScience Pharmaceutical Co., Ltd., Ascendis Pharma A/S, QED Therapeutics, Inc., BioMarin Pharmaceutical, Sanofi

[Nevada, United States] - DelveInsight's "Achondroplasia Market Insights, Epidemiology, and Market Forecast 2034." report offers a detailed examination of Achondroplasia, covering historical and predicted epidemiology, market trends, and treatment scenarios in the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan.

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Key Findings from the Achondroplasia Market Report:
• The Achondroplasia market size is poised for significant growth, with an anticipated Compound Annual Growth Rate (CAGR) during the study period (2020-2034).
• In October 2024: Sanofi announced a Phase 2, Open-label, Multi-center, 2-stage Sequential Cohort, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Subcutaneous SAR442501 in Pediatric Participants With Achondroplasia
• In October, 2024: QED Therapeutics, Inc. announced that a Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children with Achondroplasia: PROPEL 2
• In October, 2024: Ascendis Pharma Growth Disorders A/S announced a Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial Evaluating Efficacy and Safety of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Children With Achondroplasia Followed by an Open Label Extension Period
• DelveInsight estimates that the US accounted for nearly 14,500 diagnosed prevalent cases of achondroplasia in 2023.
• 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.
• Achondroplasia prevalence is estimated at 1 in 15,000 to 40,000 live births, making it one of the most common skeletal dysplasias.
• Key Achondroplasia Companies are as follows: RIBOMIC Inc., Sanofi, BridgeBio Inc., Recombinant: Changchun GeneScience Pharmaceutical Co., Ltd., Ascendis Pharma A/S, QED Therapeutics, Inc., BioMarin Pharmaceutical, Sanofi
• Key Achondroplasia Therapies are as follow: RBM-007, SAR-442501, Low dose infigratinib, Recombinant, TransCon, Infigratinib, vosoritide, BMN 111, SAR442501
• Launching multiple stage Achondroplasia pipeline products is expected to revolutionize market dynamics, further propelling market growth.

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Achondroplasia Overview:
Achondroplasia is a rare genetic bone growth disorder that results in marked short stature (dwarfism) due to a mutation in the fibroblast growth factor receptor 3 (FGFR3) gene. The mutation leads to a gain-of-function of the FGFR3 gene, which slows down the formation of bone in the cartilage of the growth plate and impairs growth in almost all bones in the body.

Achondroplasia Epidemiology Segmentation:
The Achondroplasia market report proffers epidemiological analysis for the study period 2020-2034 in the 7MM segmented into:
• Achondroplasia Total Diagnosed Prevalent Cases
• Achondroplasia Gender-specific Diagnosed Prevalent Cases

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Achondroplasia Market Insights
Achondroplasia is a rare genetic condition that causes dwarfism due to abnormal cartilage development into bone, primarily affecting the long bones. It is the most common form of disproportionate short stature, and advances in understanding the disease have led to the development of promising therapeutic interventions. This article provides an in-depth analysis of the Achondroplasia market, covering drug uptake, epidemiology, as well as key market drivers and barriers.

Achondroplasia Drugs Uptake
• Vosoritide is a C-type natriuretic peptide (CNP) analog that targets FGFR3 signaling to stimulate bone growth. It was approved by the FDA in 2021 for use in children aged five and above.
• Currently in Phase 3 clinical trials, TransCon CNP is a long-acting CNP analog. Early data suggest a promising safety profile and the potential for weekly dosing, which could improve patient compliance.
• Infigratinib is an FGFR1-3 tyrosine kinase inhibitor (TKI) developed for multiple indications, including achondroplasia. It is undergoing clinical trials with potential use in targeting the overactive FGFR3 pathway. While still under development, the drug's uptake will likely depend on trial outcomes and safety profile.
• In early-stage trials, Recifercept is being studied for its ability to bind FGFR3 and inhibit its negative effects on bone growth. If approved, it may emerge as an alternative or adjunct therapy for children and adolescents with achondroplasia.

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Achondroplasia Therapies and Key Companies:
• RBM-007: RIBOMIC Inc.
• SAR-442501: Sanofi
• Low dose infigratinib: BridgeBio Inc.
• Recombinant: Changchun GeneScience Pharmaceutical Co., Ltd.
• TransCon: Ascendis Pharma A/S
• Infigratinib: QED Therapeutics, Inc.
• vosoritide, BMN 111: BioMarin Pharmaceutical
• SAR442501: Sanofi

Achondroplasia Epidemiology:
Achondroplasia is estimated to occur in 1 in 15,000 to 40,000 live births globally, making it a rare disease. It is caused by a mutation in the FGFR3 gene (Fibroblast Growth Factor Receptor 3), which negatively affects bone growth. This condition is inherited in an autosomal dominant manner, meaning that a single copy of the altered gene is enough to cause the disorder. However, more than 80% of cases result from a spontaneous mutation, with no family history of the condition.
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Achondroplasia Market Drivers:
• Increasing Diagnosis and Awareness
• Growing R&D Investments
• Regulatory Support
• Patient Advocacy and Family Support

Achondroplasia Market Barriers:
• High Cost of Treatment
• Limited Data on Long-Term Efficacy
• Complex Clinical Trial Enrollment
• Skepticism Toward Drug Interventions

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Scope of the Achondroplasia Market Report:
• Study Period: 2020-2034
• Coverage: 7MM (The United States, EU5, and Japan)
• Key Achondroplasia Companies: RIBOMIC Inc., Sanofi, BridgeBio Inc., Recombinant: Changchun GeneScience Pharmaceutical Co., Ltd., Ascendis Pharma A/S, QED Therapeutics, Inc., BioMarin Pharmaceutical, Sanofi
• Key Achondroplasia Therapies: RBM-007, SAR-442501, Low dose infigratinib, Recombinant, TransCon, Infigratinib, vosoritide, BMN 111, SAR442501
• Achondroplasia Therapeutic Assessment: Current marketed and emerging therapies
• Achondroplasia Market Dynamics: Achondroplasia Market drivers and Achondroplasia barriers
• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
• Achondroplasia Unmet Needs: Insights into unmet needs, KOL's views, Analyst's views, Market Access, and Reimbursement.

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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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