Achondroplasia Treatment Market 2034: EMA, PDMA, FDA Approvals, Prevalence, Medication, Clinical Trials, Therapies, and Companies by DelveInsight

Achondroplasia Treatment Market

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

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.

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Some facts of the Achondroplasia Market Report are:

• According to DelveInsight, Achondroplasia market size is expected to grow at a decent CAGR by 2034.
• In November 2024, RIBOMIC, Inc. (TOKYO:4591), a clinical-stage pharmaceutical company focused on aptamer therapeutics, has been conducting a Phase IIa clinical trial of umedaptanib pegol (anti-FGF2 aptamer) in pediatric patients (ages 5-14) with achondroplasia (ACH). The company announced today that the low-dose (0.3 mg/kg) subcutaneous injection group (cohort 1), administered once a week, has completed treatment, showing a positive effect on the growth rate of the patients.
• In September 2024, BridgeBio Pharma, Inc. (Nasdaq: BBIO), a biopharmaceutical company specializing in genetic diseases, today announced that the FDA has granted Breakthrough Therapy Designation to oral infigratinib, which is being developed for the treatment of children with achondroplasia.
• In Febraury 2024, Tyra Biosciences, Inc. (Nasdaq: TYRA), a clinical-stage biotechnology company dedicated to developing next-generation precision medicines targeting Fibroblast Growth Factor Receptor (FGFR) biology, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation to TYRA-300, an oral FGFR3 selective inhibitor, for the treatment of achondroplasia.
• In the United States, achondroplasia impacts a slightly higher percentage of females (51.2%) compared to males (48.8%). This slight difference may be attributed to genetic or biological factors that contribute to its somewhat greater prevalence in females.
• In 2023, the total number of diagnosed prevalent cases of achondroplasia in Japan and the US was approximately 18,000, with expectations for this number to increase in the coming years.
• In 2023, Japan had around 3,000 diagnosed prevalent cases of achondroplasia.
• The epidemiology model categorizes age-specific cases into nine groups: 0–4, 5–10, 11–15, 16–20, 21–30, 31–40, 41–50, 51–60, and over 60. DelveInsight’s estimates indicate that the 5–10 age group was the most affected in the US, with around 4,000 cases reported in 2023.
• Achondroplasia is estimated to occur in 1 out of every 15,000 to 40,000 live births, making it one of the most prevalent types of skeletal dysplasia.
• 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: VOXZOGO (vosoritide), 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 and development. It is the most common form of dwarfism, accounting for approximately 70% of all cases. People with achondroplasia have short stature, with an average adult height of around four feet. They may also have a larger head size, shorter limbs, and a prominent forehead. Additionally, they may experience spinal stenosis, which can lead to back pain, numbness, and weakness.

Achondroplasia is caused by a mutation in the FGFR3 gene, which provides instructions for making a protein that regulates bone growth. The mutation causes the protein to be overactive, which inhibits bone growth and development.

Achondroplasia is inherited in an autosomal dominant pattern, which means that a person only needs to inherit one copy of the mutated gene from one parent to develop the condition. However, in many cases, achondroplasia occurs sporadically, meaning that there is no family history of the disorder.

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Achondroplasia Market

The Achondroplasia market outlook of the report helps to build a detailed comprehension of the historical, current, and forecasted Achondroplasia market trends by analyzing the impact of current Achondroplasia therapies on the market and unmet needs, and drivers, barriers, and demand for better technology.

This segment gives a thorough detail of the Achondroplasia market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated Achondroplasia market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

According to DelveInsight, the Achondroplasia market in 7MM is expected to witness a major change in the study period 2020-2034.

Achondroplasia Epidemiology

The Achondroplasia epidemiology section provides insights into the historical and current Achondroplasia patient pool and forecasted trends for seven individual major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the Achondroplasia market report also provides the diagnosed patient pool, trends, and assumptions.

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Achondroplasia Drugs Uptake

This section focuses on the uptake rate of the potential Achondroplasia drugs recently launched in the Achondroplasia market or expected to be launched in 2020-2034. The analysis covers the Achondroplasia market uptake by drugs, patient uptake by therapies, and sales of each drug.

Achondroplasia Drugs Uptake helps in understanding the drugs with the most rapid uptake and the reasons behind the maximal use of new drugs and allows the comparison of the drugs based on Achondroplasia market share and size, which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

Achondroplasia Pipeline Development Activities

The Achondroplasia report provides insights into different therapeutic candidates in Phase II, and Phase III stages. It also analyses Achondroplasia key players involved in developing targeted therapeutics.

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Achondroplasia Therapies and Key Companies

• VOXZOGO (vosoritide): BioMarin Pharmaceuticals
• Infigratinib: BridgeBio/ QED Therapeutics
• RBM-007: Ribomic Inc
• 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 [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: 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

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Achondroplasia Report Key Insights

1. Achondroplasia Patient Population

2. Achondroplasia Market Size and Trends

3. Key Cross Competition in the Achondroplasia Market

4. Achondroplasia Market Dynamics (Key Drivers and Barriers)

5. Achondroplasia Market Opportunities

6. Achondroplasia Therapeutic Approaches

7. Achondroplasia Pipeline Analysis

8. Achondroplasia Current Treatment Practices/Algorithm

9. Impact of Emerging Therapies on the Achondroplasia Market

Table of Contents

1. Key Insights

2. Executive Summary

3. Achondroplasia Competitive Intelligence Analysis

4. Achondroplasia Market Overview at a Glance

5. Achondroplasia Disease Background and Overview

6. Achondroplasia Patient Journey

7. Achondroplasia Epidemiology and Patient Population

8. Achondroplasia Treatment Algorithm, Current Treatment, and Medical Practices

9. Achondroplasia Unmet Needs

10. Key Endpoints of Achondroplasia Treatment

11. Achondroplasia Marketed Products

12. Achondroplasia Emerging Therapies

13. Achondroplasia Seven Major Market Analysis

14. Attribute Analysis

15. Achondroplasia Market Outlook (7 major markets)

16. Achondroplasia Access and Reimbursement Overview

17. KOL Views on the Achondroplasia Market

18. Achondroplasia Market Drivers

19. Achondroplasia Market Barriers

20. Appendix

21. DelveInsight Capabilities

22. Disclaimer

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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