Table of Contents 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 Ribomic Inc Achondroplasia - Drug Profiles B-701 - Drug Profile Product Description Mechanism Of Action R&D Progress infigratinib - Drug Profile Product Description Mechanism Of Action R&D Progress RBM-007 - Drug Profile Product Description Mechanism Of Action R&D Progress Recombinant Peptide to Antagonize FGFR3 for Achondroplasia - 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 Feb 14, 2018: Therachon Announces Dosing of First Subject in Phase 1 Clinical Trial Evaluating TA-46, a Novel Investigational Therapy for the Potential Treatment of Achondroplasia Jan 08, 2018: Ascendis Pharma Provides Update on TransCon CNP Sep 05, 2017: Ascendis Pharma Announces Presentations on TransCon CNP at Upcoming Medical Conferences Jun 20, 2017: Therachon Receives EMA and FDA Orphan Drug Designation for TA-46 for Achondroplasia Apr 03, 2017: Ascendis Pharma Presents Data on TransCon CNP at ENDO 2017 Feb 28, 2017: Ascendis Pharma Announces Data on TransCon CNP at ENDO 2017 Dec 12, 2016: BioMarin Enrolls First Participant in Phase 3 Trial of Vosoritide for Treatment of Children with Achondroplasia Oct 19, 2016: BioMarin Presents Vosoritide Data in Achondroplasia at American Society of Human Genetics 2016 Meeting Apr 20, 2016: BioMarin Provides Program Update on Vosoritide in Achondroplasia Oct 12, 2015: BioMarin’s Initial 6-Month Data From Phase 2 Study of Vosoritide in Children With Achondroplasia Presented at the American Society for Bone and Mineral Research Annual 2015 Meeting Jun 17, 2015: BMN 111 Improves Growth Velocity in Children With Achondroplasia in Phase 2 Study Jan 15, 2014: BioMarin Doses First Patient in Phase 2 Trial With BMN 111 for the Treatment of Children With Achondroplasia Appendix Methodology Coverage Secondary Research Primary Research Expert Panel Validation Contact Us Disclaimer

List of Tables Number of Products under Development for Achondroplasia, H1 2018 Number of Products under Development by Companies, H1 2018 Products under Development by Companies, H1 2018 Number of Products by Stage and Target, H1 2018 Number of Products by Stage and Mechanism of Action, H1 2018 Number of Products by Stage and Route of Administration, H1 2018 Number of Products by Stage and Molecule Type, H1 2018 Achondroplasia - Pipeline by Ascendis Pharma A/S, H1 2018 Achondroplasia - Pipeline by BioMarin Pharmaceutical Inc, H1 2018 Achondroplasia - Pipeline by Ribomic Inc, H1 2018 Achondroplasia - Dormant Projects, H1 2018List of Figures Number of Products under Development for Achondroplasia, H1 2018 Number of Products under Development by Companies, H1 2018 Number of Products by Top 10 Targets, H1 2018 Number of Products by Stage and Targets, H1 2018 Number of Products by Mechanism of Actions, H1 2018 Number of Products by Stage and Mechanism of Actions, H1 2018 Number of Products by Routes of Administration, H1 2018 Number of Products by Stage and Routes of Administration, H1 2018 Number of Products by Molecule Types, H1 2018 Number of Products by Stage and Molecule Types, H1 2018