Table of Content


List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Achondroplasia - Overview
Achondroplasia - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Products under Development by Companies
Achondroplasia - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Achondroplasia - Companies Involved in Therapeutics Development
Ascendis Pharma A/S
BioMarin Pharmaceutical Inc
Daiichi Sankyo Co Ltd
Pfizer Inc
PhaseBio Pharmaceuticals Inc
QED Therapeutics Inc
Ribomic Inc
Achondroplasia - Drug Profiles
ACP-015 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ASB-20123 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Fusion Protein to Agonize NPR2 for Achondroplasia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
infigratinib phosphate - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RBM-007 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
TA-100 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
TA-46 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
vosoritide - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Achondroplasia - Dormant Projects
Achondroplasia - Product Development Milestones
Featured News & Press Releases
Nov 14, 2019: BioMarin announces cumulative additiol height gain of 9.0 cm over 54 months versus tural history in children with Achondroplasia treated with Vosoritide in phase 2 study
Nov 06, 2019: BioMarin to highlight innovative development pipeline including vosoritide at R&D Day on November 14th
Oct 17, 2019: QED and parent company BridgeBio announce preclinical data supporting tolerability and activity of low-dose infigratinib in treating Achondroplasia
Sep 12, 2019: Ascendis Pharma announces presentations on Achondroplasia and TransCon CNP at Intertiol Skeletal Dysplasia Society Meeting
Jul 18, 2019: Ascendis Pharma announces filing of Investigatiol New Drug (IND) application for initiation of a global phase 2 trial for TransCon CNP in Children with Achondroplasia
Jun 18, 2019: BioMarin announces New England Jourl of medicine publishes vosoritide phase 2 study showing sustained annualized growth up to 42 months in children with Achondroplasia
Feb 28, 2019: Ascendis Pharma announces orphan drug desigtion granted for TransCon CNP as treatment for achondroplasia
Jan 07, 2019: BioMarin highlights Vosoritide at 37th Annual J.P. Morgan Healthcare Conference in San Francisco
Nov 28, 2018: Ascendis Pharma announces positive prelimiry phase 1 data for TransCon CNP
Nov 07, 2018: BioMarin provides update on Vosoritide at R&D day in New York
Nov 01, 2018: BioMarin provides update on vosoritide at R&D Day on November 7th in New York
Jun 21, 2018: Therachon Announces Start of tural History Study in Children with Achondroplasia
Jun 14, 2018: BioMarin Doses First Participant in Phase 2 Study of Vosoritide for Treatment of Infants and Young Children with Achondroplasia
May 08, 2018: Ascendis Pharma Announces Dosing of First Subjects in Phase 1 Trial of TransCon CNP
Feb 14, 2018: Therachon Announces Dosing of First Subject in Phase 1 Clinical Trial Evaluating TA-46, a Novel Investigatiol Therapy for the Potential Treatment of Achondroplasia
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures


Figure 1: Number of Products under Development for Achondroplasia, H2 2019
Figure 2: Number of Products under Development by Companies, H2 2019
Figure 3: Number of Products by Targets, H2 2019
Figure 4: Number of Products by Stage and Top 10 Targets, H2 2019
Figure 5: Number of Products by Mechanism of Actions, H2 2019
Figure 6: Number of Products by Stage and Top 10 Mechanism of Actions, H2 2019
Figure 7: Number of Products by Routes of Administration, H2 2019
Figure 8: Number of Products by Stage and Routes of Administration, H2 2019
Figure 9: Number of Products by Molecule Types, H2 2019
Figure 10: Number of Products by Stage and Molecule Types, H2 2019


List of Tables


Table 1: Number of Products under Development for Achondroplasia, H2 2019
Table 2: Number of Products under Development by Companies, H2 2019
Table 3: Products under Development by Companies, H2 2019
Table 4: Number of Products by Stage and Target, H2 2019
Table 5: Number of Products by Stage and Mechanism of Action, H2 2019
Table 6: Number of Products by Stage and Route of Administration, H2 2019
Table 7: Number of Products by Stage and Molecule Type, H2 2019
Table 8: Achondroplasia - Pipeline by Ascendis Pharma A/S, H2 2019
Table 9: Achondroplasia - Pipeline by BioMarin Pharmaceutical Inc, H2 2019
Table 10: Achondroplasia - Pipeline by Daiichi Sankyo Co Ltd, H2 2019
Table 11: Achondroplasia - Pipeline by Pfizer Inc, H2 2019
Table 12: Achondroplasia - Pipeline by PhaseBio Pharmaceuticals Inc, H2 2019
Table 13: Achondroplasia - Pipeline by QED Therapeutics Inc, H2 2019
Table 14: Achondroplasia - Pipeline by Ribomic Inc, H2 2019
Table 15: Achondroplasia - Dormant Projects, H2 2019