DelveInsight’s Achondroplasia pipeline report covers the emerging drugs profiles, including clinical and nonclinical products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type.
Key Highlights from the Achondroplasia Pipeline Report
Get a detailed analysis of the pipeline landscape @ Achondroplasia Pipeline Outlook
Achondroplasia Overview
Achondroplasia is a rare genetic disorder that causes short-limb skeletal dysplasia in about 1 in 20,000-30,000 live births. This genetic disorder is caused by a mutation in the fibroblast growth factor receptor 3 (FGFR3) gene, which occurs as a result of either a spontaneous genetic mutation (in 80% of patients) or an inherited genetic mutation (in 20% of patients).
Achondroplasia Symptoms
Achondroplasia patients experience the following symptoms:
Achondroplasia Pipeline Analysis: Drug Profiles
Recifercept (TA 46): Pfizer
Product Description
Recifercept (TA 46) is a soluble recombinant human fibroblast growth factor receptor 3 (FGFR3) decoy with an MOA are Achondroplasia treatmentsexpected to normalize the overactive FGFR3 signaling pathways that underpin achondroplasia-related bone development abnormalities.
Phase II
NCT04638153: Pfizer launched a Phase II trial titled “A Phase II multiple dose, randomized study to assess the safety, tolerability, pharmacokinetics, and efficacy of Refercept in children with Achondroplasia” in December 2020.
Achondroplasia Pipeline Therapies and Key Companies
Find out the emerging therapies and companies @ Achondroplasia Clinical Trials
Achondroplasia Therapeutics Assessment
Scope of the Report
Coverage: Global
Achondroplasia Key Players: BioMarin, Pfizer, Ribomic Inc., among others
Achondroplasia Pipeline Therapies: Vosoritide, Recifercept, RBM-007, and others
Table of Contents
1.
Introduction
2.
Executive Summary
3.
Achondroplasia: Overview
4.
Achondroplasia – Analytical Perspective In-depth Commercial Assessment
5.
Pipeline Therapeutics
6.
Late Stage Products (Phase III)
7.
Mid Stage Products (Phase II)
8.
Early Stage Products (Phase I)
9.
Pre-clinical and Discovery Stage Products
10.
Therapeutic Assessment
11.
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
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Key questions answered in the Achondroplasia Pipeline Report
Related Reports
Achondroplasia Market
DelveInsight’s “Achondroplasia – Market Insights, Epidemiology, and Market Forecast-2030” report delivers an in-depth understanding of the Achondroplasia, historical and forecasted epidemiology, and the Achondroplasia market trends in the 7MM.
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.
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