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DelveInsight’s “Achondroplasia Market Insights, Epidemiology, and Market Forecast-2034” report delivers an in-depth understanding of the Achondroplasia, historical and forecasted epidemiology as well as the Achondroplasia market trends in the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom) and Japan.

 

Key Takeaways from the Achondroplasia Market Research Report

• The increase in Achondroplasia Market Size is a direct consequence of the increasing patient population and anticipated launch of emerging therapies in the 7MM.
• According to DelveInsight’s estimates, in 2022, there were nearly 28,613 diagnosed prevalent cases of Achondroplasia in the 7MM. These cases will change during the study period (2020–2034).
• The leading Achondroplasia Companies working in the market include Ascendis Pharma, QED Therapeutics (BridgeBio), Novartis, Sanofi, RIBOMIC, and others.
• Promising Achondroplasia Pipeline Therapies in the various stages of development include TransCon CNP, Infigratinib 0.25 mg/kg/day, Navepegritide, BMN 111, vosoritide, Somatropin, and others.
• April 2024: QED Therapeutics Inc. announced a study of Phase 3 clinical trials for Infigratinib 0.25 mg/kg/day. This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of infigratinib in children and adolescents with achondroplasia (ACH) who have completed at least 26 weeks of participation in the QED-sponsored study PROPEL (QBGJ398-001).
• April 2024: Ascendis Pharma Growth Disorders A/S announced a study of Phase 2 clinical trials for Navepegritide. This trial is a Phase 2, 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.
• March 2024: Sanofi announced a study of Phase 2 clinical trials for SAR442501. This is a Phase 2, open-label, multicenter, study to evaluate safety, tolerability and efficacy of SAR442501 in children from birth up to 12 years of age with Achondroplasia.

 

Discover which therapies are expected to grab the Achondroplasia Market Share @ Achondroplasia Market Outlook

 

Achondroplasia Overview

Achondroplasia is a rare genetic bone growth disorder that results in marked short stature (dwarfism) due to a mutation in the broblast 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 Insights

The epidemiology section of Achondroplasia offers insights into both historical and current patient populations, as well as forecasted trends across seven major countries. This section aids in understanding the factors behind present and projected trends through analysis of various studies and input from key opinion leaders. Additionally, this portion of the market report provides information on the diagnosed patient pool, trends, and underlying assumptions. Achondroplasia Epidemiology segmentation is given below-

• Total Achondroplasia Diagnosed Prevalent Cases
• Achondroplasia Gender-specific Diagnosed Prevalent Cases

 

Download the report to understand which factors are driving Achondroplasia Epidemiology trends @ Achondroplasia Epidemiological Insights

 

Achondroplasia Market Insights

Achondroplasia is a genetic disorder that primarily affects bone growth, leading to short stature and various physical characteristics. It is a common form of dwarfism, characterized by a significantly shorter stature than the average for a person’s age and ethnic background. Achondroplasia is caused by a mutation in the FGFR3 gene, which encodes a protein involved in regulating bone growth. The disease is mainly characterized by an unusually large head (macrocephaly), short upper arms (Rhizomelic dwarfism), and short stature (adult height of approximately 4 ft. Achondroplasia is associated with neurological compilations and does not typically cause impairment or deficiencies in mental abilities. Further, recurrent ear infections, hearing loss, and other complications also occur in people with Achondroplasia. 

 

Achondroplasia Treatment Market Landscape

The current treatment landscape of Achondroplasia lacks curative therapies and involves both pharmacological and nonpharmacological options. Different treatment is directed against the specific issues encountered in Achondroplasia. Pharmacological intervention includes treatment with CNP analogs and other symptomatic medications for managing complications. Further, growth hormone therapy may be considered as a treatment option for Achondroplasia; however, its long-term benefits are controversial and are only authorized in Japan.

 

To know more about Achondroplasia treatment guidelines, visit @ Achondroplasia Treatment Landscape

 

Achondroplasia Market Outlook

The report’s outlook on the Achondroplasia market aids in developing a comprehensive understanding of historical, current, and projected trends. This is achieved by examining the influence of existing Achondroplasia therapies, unmet needs, as well as drivers, barriers, and the demand for advanced technology. This section provides detailed insights into the trends of each marketed Achondroplasia drug and late-stage pipeline therapy. It assesses their impact based on various factors such as annual therapy costs, inclusion/exclusion criteria, mechanism of action, compliance rates, market demand, patient population growth, covered patient segments, anticipated launch year, competition with other therapies, brand value, and input from key opinion leaders. The analyzed Achondroplasia market data are presented concisely through relevant tables and graphs to offer a clear overview of the market dynamics.

 

Achondroplasia Drugs Uptake

• TransCon CNP (navepegritide) is a novel long-acting CNP prodrug that comes in a convenient once-weekly SC dose and is intended to offer therapeutic amounts of continuous CNP exposure. It is being developed for the treatment of children with achondroplasia. TransCon CNP is made to effectively shield CNP from neutral endopeptidase degradation in the blood compartment and SC tissue. It also reduces CNP binding to the natriuretic peptide receptor (NPR-B) receptor in the cardiovascular system, preventing hypotension, and minimizes CNP binding to the NPR-C receptor to decreaseclearance. 

• Infigratinib (BBP-831/BGJ398) is an orally administered, ATP-competitive, selective FGFR1-3 TKI used to treat FGFR-driven conditions, including achondroplasia in children. It blocks the abnormal signaling pathway (MAPK and STAT-1) associated with FGFR3 mutations. 

• SAR442501 is an anti-FGFR3 monoclonal antibody (mAb) that targets overactive FGFR3 in achondroplasia and Osteochondrodysplasia patients. It is administered through the SC route. 

• RBM-007 is a novel nucleic acid medicine (oligonucleotide-based aptamer) developed in-house at RIBOMIC’s research facilities in Tokyo, Japan. It comprises 36 nucleotides and binds stably and specifically to FGF2 but not other FGFs. RBM-007 acts as an anti-FGF2 aptamer and has shown potent effects in limiting excessive interactions between FGF2 and FGF receptor 3 activating variants (known to cause achondroplasia). Thus, when delivered by SC injection, RBM-007 restored defective skeletal growth in a mouse model of achondroplasia. 

 

Major Achondroplasia Companies

Several companies in the market include Ascendis Pharma, QED Therapeutics (BridgeBio), Novartis, Sanofi, RIBOMIC, and others.

 

Learn more about the FDA-approved drugs for Achondroplasia @ Drugs for Achondroplasia Treatment

 

Scope of the Achondroplasia Market Research Report

• Coverage- 7MM
• Study Period- 20190-2032
• Achondroplasia Companies- Ascendis Pharma, QED Therapeutics (BridgeBio), Novartis, Sanofi, RIBOMIC, and others.
• Achondroplasia Pipeline Therapies- TransCon CNP, Infigratinib 0.25 mg/kg/day, Navepegritide, BMN 111, vosoritide, Somatropin, and others
• Achondroplasia Market Dynamics: Achondroplasia Market Drivers and Barriers
• Achondroplasia Market Access and Reimbursement, Unmet Needs and Future Perspectives

 

Discover more about Achondroplasia Drugs in development @ Achondroplasia Clinical Trials Assessment

 

Table of Content

1. Key Insights

2. Report Introduction

3. Achondroplasia Market Overview at a Glance

4. Methodology of Achondroplasia Epidemiology and Market

5. Executive Summary of Achondroplasia

6. Key Events

7. Disease Background and Overview

8. Patient Journey

9. Epidemiology and Patient Population

10. Marketed Drugs

11. Emerging Drugs

12. Achondroplasia: Market Analysis

13. Key Opinion Leaders’ Views

14. SWOT Analysis

15. Unmet Needs

16. Market Access and Reimbursement

17. Appendix

18. DelveInsight Capabilities

19. Disclaimer

20. About DelveInsight

 

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