(Albany, United States) As per DelveInsight’s assessment, globally, the Achondroplasia Pipeline constitutes 5+ key companies continuously working towards developing 5+ Achondroplasia treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analysis DelveInsight.

 

In the Achondroplasia Pipeline Report, a detailed description of the drug is given which includes the mechanism of action of the drug, Achondroplasia clinical trials studies, Achondroplasia NDA approvals (if any), and product development activities comprising the technology, Achondroplasia collaborations, licensing, mergers and acquisition, funding, designations, and other product-related details.

 

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Key Takeaways from the Achondroplasia Pipeline Report

• DelveInsight’s Achondroplasia Pipeline analysis depicts a robust space with 5+ active players working to develop 5+ pipeline treatment therapies.
• The leading Achondroplasia Companies working in the market include BioMarin Pharmaceutical, Ascendis Pharma, QED Therapeutics (BridgeBio), Pfizer, PhaseBio Pharmaceuticals, Ribomic, and others
• Promising Achondroplasia Pipeline Therapies in the various stages of development include Recombinant human growth hormone, TransCon CNP, Infigratinib, BMN 111, and others
• On June 2023, Ascendis Pharma A/S announced a study of phase 2 & 3 clinical trials for TransCon CNP. The purpose of this clinical trial is to evaluate efficacy and safety of once weekly SC doses of 100 µg CNP/kg compared to placebo on Annualized Growth Velocity after a 52-week randomized treatment period in children aged 2 to 11 years with genetically confirmed Achondroplasia. The double-blind, placebo-controlled treatment period is followed by an Open Label Extension (OLE) period of a 52-week duration.
• On June 2023, QED Therapeutics Inc announced a study of phase 2 clinical trials for Infigratinib. This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in subjects with ACH who previously completed a QED-sponsored interventional study, and potentially in additional subjects who are naïve to infigratinib treatment. Quality of Life assessments for this subject population will also be evaluated. Treatment-naïve subjects must have at least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398 001) and will be enrolled in this OLE study only after a dose to be explored further is identified in Phase 2 Study QBGJ398-201.

 

Achondroplasia Overview

Chondroplasia (also called: ACH, Achondroplasia Dwarfism) is the most commonly occurring abnormality of bone growth (skeletal dysplasia) and is a form of short-limbed dwarfism. The word for word translation of ‘Achondroplasia’ is “without cartilage formation.” Cartilage is a robust but flexible tissue that makes up much of the skeleton during early development.

 

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Achondroplasia Emerging Drugs Profile

• BMN-111: BioMarin Pharmaceutical
• Infigratinib: QED Therapeutics (BridgeBio)
• TA-46: Pfizer
• CNP-ELP: PhaseBio Pharmaceuticals

 

Achondroplasia Pipeline Therapeutics Assessment

There are approx. 6+ Achondroplasia companies which are developing the therapies for Achondroplasia. The companies which have their Achondroplasia drug candidates in the most advanced stage, i.e. Phase III include, BioMarin Pharmaceutical.

 

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Achondroplasia Drugs and Companies

• BMN 111: BioMarin Pharmaceutical
• Infigratinib: QED Therapeutics Inc
• TransCon CNP: Ascendis Pharma A/S

 

Achondroplasia Therapeutics Assessment

• Assessment by Stage and Product Type
• Assessment by Route of Administration
• Assessment by Stage and Route of Administration
• Assessment by Molecule Type
• Assessment by Stage and Molecule Type

 

Some of the Companies in the Achondroplasia Therapeutics Market include-

BioMarin Pharmaceutical, Ascendis Pharma, QED Therapeutics (BridgeBio), Pfizer, PhaseBio Pharmaceuticals, Ribomic, and others.

 

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Scope of the Achondroplasia Pipeline Report

• Coverage- Global
• Achondroplasia Companies- BioMarin Pharmaceutical, Ascendis Pharma, QED Therapeutics (BridgeBio), Pfizer, PhaseBio Pharmaceuticals, Ribomic, and others.
• Achondroplasia Pipeline Therapies- Recombinant human growth hormone, TransCon CNP, Infigratinib, BMN 111, and others.
• Achondroplasia Segmentation: Product Type, Molecule Type, Route of Administration

 

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Table of Content

1. Introduction
2. Executive Summary
3. Achondroplasia: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Achondroplasia – DelveInsight’s Analytical Perspective
7. In-depth Commercial Assessment
8. Achondroplasia Collaboration Deals
9. Late Stage Products
10. BMN-111: BioMarin Pharmaceutical
11. Drug profiles in the detailed report…..
12. Mid Stage Products
13. TransCon CNP: Ascendis Pharma
14. Infigratinib: QED Therapeutics (BridgeBio)
15. Drug profiles in the detailed report…..
16. Early Stage Products
17. TA-46: Pfizer
18. Drug profiles in the detailed report…..
19. Pre-clinical and Discovery Stage Products
20. CNP-ELP: PhaseBio Pharmaceuticals
21. Drug profiles in the detailed report…..
22. Inactive Products
23. Achondroplasia Key Companies
24. Achondroplasia Key Products
25. Achondroplasia- Unmet Needs
26. Achondroplasia- Market Drivers and Barriers
27. Achondroplasia- Future Perspectives and Conclusion
28. Achondroplasia Analyst Views
29. Achondroplasia Key Companies
30. Appendix

 

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