“Achondroplasia Pipeline Insight, 2025" report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Achondroplasia Market.
The Achondroplasia Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.
Some of the key takeaways from the Achondroplasia Pipeline Report:
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Companies across the globe are diligently working toward developing novel Achondroplasia treatment therapies with a considerable amount of success over the years.
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Achondroplasia companies working in the treatment market are BioMarin Pharmaceuticals, BridgeBio/ QED Therapeutics, Ribomic Inc, Sanofi, Changchun GeneScience Pharma, Ascendis Pharma A/S, QED Therapeutics, Inc, BioMarin Pharmaceuticals, BioMarin Pharmaceutical, and others, are developing therapies for the Achondroplasia treatment
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Emerging Achondroplasia therapies in the different phases of clinical trials are- VOXZOGO (vosoritide), Infigratinib, RBM-007, SAR-442501, Recombinant human growth hormone, Infigratinib, TransCon CNP, Infigratinib, vosoritide, BMN 111, and others are expected to have a significant impact on the Achondroplasia market in the coming years.
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In June 2025, Ascendis Pharma reported the week 26 interim results from its ongoing Phase II COACH Trial, which is assessing the combination of TransCon CNP (navepegritide) and TransCon hGH (lonapegsomatropin) in children with achondroplasia, a rare genetic disorder. The data indicate that TransCon hGH enhances the effects of TransCon CNP, leading to greater growth and improved body proportions after 26 weeks of treatment. The combination's safety and tolerability were consistent with what has been observed for each therapy individually.
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In March 2025, Ascendis Pharma A/S (Nasdaq: ASND) submitted a New Drug Application (NDA) to the U.S. FDA for TransCon CNP (navepegritide), aimed at treating children with achondroplasia. This investigational, once-weekly prodrug of C-type natriuretic peptide (CNP) is designed to provide sustained CNP exposure to target receptors throughout the body—especially in growth plates and skeletal muscles—to address the root cause of achondroplasia.
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In January 2025, The FDA cleared the Investigational New Drug (IND) application for TYRA-300, an oral FGFR3-selective inhibitor, for treating low-grade, intermediate-risk non-muscle invasive bladder cancer (NMIBC). This approval paves the way for a Phase 2 clinical trial in this patient group. TYRA-300 is an investigational oral therapy under development for cancers and skeletal disorders, including achondroplasia and hypochondroplasia.
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In November 2024, BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced positive and consistent results from multiple real-world evidence studies of VOXZOGO® (vosoritide) in children with achondroplasia. The company also shared data from ongoing investigational studies evaluating VOXZOGO for hypochondroplasia. These findings were presented at the 62nd Annual Meeting of the European Society for Paediatric Endocrinology (ESPE) in Liverpool.
Achondroplasia Overview
Achondroplasia is a rare genetic disorder that affects bone growth, leading to disproportionate short stature. It is the most common cause of dwarfism and is caused by mutations in the FGFR3 gene, which slows the development of cartilage into bone, especially in the long bones of the arms and legs. People with achondroplasia typically have a normal-sized torso, short limbs, an enlarged head with a prominent forehead, and specific facial features, but intelligence and lifespan are usually normal.
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Emerging Achondroplasia Drugs Under Different Phases of Clinical Development Include:
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VOXZOGO (vosoritide): BioMarin Pharmaceuticals
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Infigratinib: BridgeBio/ QED Therapeutics
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RBM-007: Ribomic Inc
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Sanofi: SAR-442501
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Recombinant human growth hormone: Changchun GeneScience Pharma
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Infigratinib: QED Therapeutics, Inc.
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TransCon CNP: Ascendis Pharma A/S
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Infigratinib: QED Therapeutics, Inc
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vosoritide: BioMarin Pharmaceuticals
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BMN 111: BioMarin Pharmaceutical
Achondroplasia Route of Administration
Achondroplasia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as
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Oral
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Parenteral
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Intravenous
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Subcutaneous
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Topical
Achondroplasia Molecule Type
Achondroplasia Products have been categorized under various Molecule types, such as
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Monoclonal Antibody
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Peptides
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Polymer
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Small molecule
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Gene therapy
Achondroplasia Pipeline Therapeutics Assessment
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Achondroplasia Assessment by Product Type
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Achondroplasia By Stage and Product Type
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Achondroplasia Assessment by Route of Administration
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Achondroplasia By Stage and Route of Administration
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Achondroplasia Assessment by Molecule Type
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Achondroplasia by Stage and Molecule Type
DelveInsight's Achondroplasia Report covers around 5+ products under different phases of clinical development like
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Late-stage products (Phase III)
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Mid-stage products (Phase II)
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Early-stage product (Phase I)
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Pre-clinical and Discovery stage candidates
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Discontinued & Inactive candidates
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Route of Administration
Further Achondroplasia product details are provided in the report. Download the Achondroplasia pipeline report to learn more about the emerging Achondroplasia therapies
Some of the key companies in the Achondroplasia Therapeutics Market include:
Key companies developing therapies for Achondroplasia are - Novartis, Sanofi, RIBOMIC, Ascendis Pharma, QED Therapeutics (BridgeBio), BioMarin Pharmaceutical, Pfizer, PhaseBio Pharmaceuticals, and others.
Achondroplasia Pipeline Analysis:
The Achondroplasia pipeline report provides insights into
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The report provides detailed insights about companies that are developing therapies for the treatment of Achondroplasia with aggregate therapies developed by each company for the same.
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It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Achondroplasia Treatment.
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Achondroplasia key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
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Achondroplasia Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
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Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Achondroplasia market.
The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.
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Achondroplasia Pipeline Market Drivers
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Increasing prevalence of achondroplasia worldwide, creating demand for novel therapies, Advancements in genetic research enabling targeted drug development, Rising awareness and early diagnosis of rare genetic disorders, are some of the important factors that are fueling the Achondroplasia Market.
Achondroplasia Pipeline Market Barriers
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However, Limited number of therapeutic candidates in late-stage clinical trials, High costs associated with rare disease drug development, Challenges in recruiting sufficient participants for clinical trials, and other factors are creating obstacles in the Achondroplasia Market growth.
Scope of Achondroplasia Pipeline Drug Insight
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Coverage: Global
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Key Achondroplasia Companies: BioMarin Pharmaceuticals, BridgeBio/ QED Therapeutics, Ribomic Inc, Sanofi, Changchun GeneScience Pharma, Ascendis Pharma A/S, QED Therapeutics, Inc, BioMarin Pharmaceuticals, BioMarin Pharmaceutical, and others
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Key Achondroplasia Therapies: VOXZOGO (vosoritide), Infigratinib, RBM-007, SAR-442501, Recombinant human growth hormone, Infigratinib, TransCon CNP, Infigratinib, vosoritide, BMN 111, and others
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Achondroplasia Therapeutic Assessment: Achondroplasia current marketed and Achondroplasia emerging therapies
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Achondroplasia Market Dynamics: Achondroplasia market drivers and Achondroplasia market barriers
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Table of Contents
1. Achondroplasia Report Introduction
2. Achondroplasia Executive Summary
3. Achondroplasia Overview
4. Achondroplasia- Analytical Perspective In-depth Commercial Assessment
5. Achondroplasia Pipeline Therapeutics
6. Achondroplasia Late Stage Products (Phase II/III)
7. Achondroplasia Mid Stage Products (Phase II)
8. Achondroplasia Early Stage Products (Phase I)
9. Achondroplasia Preclinical Stage Products
10. Achondroplasia Therapeutics Assessment
11. Achondroplasia Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Achondroplasia Key Companies
14. Achondroplasia Key Products
15. Achondroplasia Unmet Needs
16 . Achondroplasia Market Drivers and Barriers
17. Achondroplasia Future Perspectives and Conclusion
18. Achondroplasia Analyst Views
19. Appendix
20. About DelveInsight
About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.
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