DelveInsight’s “Achondroplasia Market Insights, Epidemiology, and Market Forecast-2032″ report offers an in-depth understanding of the Achondroplasia, historical and forecasted epidemiology as well as the Achondroplasia market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.
To Know in detail about the Achondroplasia market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Achondroplasia Market Forecast
Some of the key facts of the Achondroplasia Market Report:
The Achondroplasia market size is anticipated to grow with a significant CAGR during the study period (2019-2032)
In January 2022, Ascendis Pharma A/S announced that the European Commission (EC) granted marketing authorization for TransCon hGH. This approval allows for the once-weekly subcutaneous injection of TransCon hGH as a treatment for children and adolescents aged 3 to 18 years experiencing growth failure due to inadequate secretion of endogenous growth hormone, also known as growth hormone deficiency (GHD). TransCon hGH is a prodrug of somatropin, offering sustained release of unmodified somatropin (hGH) at predictable therapeutic levels in the body. The EC’s decision is grounded in clinical findings presented in the Marketing Authorization Application (MAA), incorporating data from Ascendis Pharma’s Phase III heiGHt, fliGHt, and enliGHten Trials. These trials collectively involved over 300 pediatric patients diagnosed with GHD, and the authorization also considered information from a non-clinical safety program.
In April 2022, RIBOMIC, Inc. submitted an Investigational New Drug Application (IND) for an observational study involving the drug RBM-007. This drug has demonstrated potent effects in restricting excessive interactions between fibroblast growth factors, recognized contributors to achondroplasia. The submission was made to the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan. Concurrently, TransCon hGH is undergoing development for pediatric Growth Hormone Deficiency (GHD) in Japan, and BridgeBio has reported positive Phase II results for Infigratinib. These advancements are anticipated to enhance the long-term prospects in the treatment landscape for achondroplasia. Consequently, the global medical community is adjusting its Research and Development (R&D) priorities, placing a significant emphasis on exploring the future development of therapeutic drug candidates for achondroplasia. This shift aims to cultivate a potential revenue ecosystem that will significantly drive research in the future.
As stated by Benjamin et al., achondroplasia is an autosomal dominant genetic disorder, with the majority of frequency estimates ranging between 1 in 25,000 and 1 in 35,000 live births. However, it is suggested that the actual frequency might be slightly elevated.
As per information from NORD, Achondroplasia arises due to an independent genetic mutation in approximately 80% of individuals, while the remaining 20% inherit it from a parent.
Key Achondroplasia Companies: BridgeBio/ QED Therapeutics , Ribomic Inc, Sanofi, Changchun GeneScience Pharma, Ascendis Pharma A/S, QED Therapeutics, Inc, BioMarin Pharmaceuticals, BioMarin Pharmaceutical, and others
Key Achondroplasia Therapies: Infigratinib, RBM-007, SAR-442501, Recombinant human growth hormone, Infigratinib, TransCon CNP, Infigratinib, vosoritide, BMN 111, and others
The Achondroplasia market is expected to surge due to the disease’s increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Achondroplasia pipeline products will significantly revolutionize the Achondroplasia market dynamics.
Achondroplasia Overview
Achondroplasia is a genetic disorder that affects bone growth, leading to dwarfism. It is the most common cause of short stature with disproportionately short limbs. The condition results from a mutation in the FGFR3 gene, which provides instructions for making a protein involved in the development and maintenance of bone and brain tissue.
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Achondroplasia Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.
Achondroplasia Epidemiology Segmentation:
The Achondroplasia market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into:
Total Prevalence of Achondroplasia
Prevalent Cases of Achondroplasia by severity
Gender-specific Prevalence of Achondroplasia
Diagnosed Cases of Episodic and Chronic Achondroplasia
Download the report to understand which factors are driving Achondroplasia epidemiology trends @ Achondroplasia Epidemiology Forecast
Achondroplasia Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Achondroplasia market or expected to get launched during the study period. The analysis covers Achondroplasia market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Achondroplasia Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
Achondroplasia Therapies and Key Companies
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
Discover more about therapies set to grab major Achondroplasia market share @ Achondroplasia Treatment Landscape
Scope of the Achondroplasia Market Report
Study Period: 2019–2032
Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
Key Achondroplasia Companies: 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
Key Achondroplasia Therapies: 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
To know more about Achondroplasia companies working in the treatment market, visit @ Achondroplasia Clinical Trials and Therapeutic Assessment
Table of Contents
1. Achondroplasia Market Report Introduction
2. Executive Summary for Achondroplasia
3. SWOT analysis of Achondroplasia
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. Achondroplasia Unmet Needs
11. Achondroplasia Emerging Therapies
12. Achondroplasia Market Outlook
13. Country-Wise Achondroplasia Market Analysis (2019–2032)
14. Achondroplasia Market Access and Reimbursement of Therapies
15. Achondroplasia Market Drivers
16. Achondroplasia Market Barriers
17. Achondroplasia Appendix
18. Achondroplasia Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight
About DelveInsight
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It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.
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