Achondroplasia - Pipeline Review, H2 2018

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Date: 11-Sep-2018
No. of pages: 49
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Achondroplasia - Pipeline Review, H2 2018

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Achondroplasia - Pipeline Review, H2 2018, provides an overview of the Achondroplasia (Musculoskeletal Disorders) pipeline landscape.

Achondroplasia is a bone growth disorder that causes disproportionate dwarfism. This is caused by mutations in the FGFR3 gene. Symptoms include decreased muscle tone, apnea, hydrocephalus, short arms and legs, disproportionately large head compared to the body and kyphosis. Treatment includes growth hormones.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Achondroplasia - Pipeline Review, H2 2018, provides comprehensive information on the therapeutics under development for Achondroplasia (Musculoskeletal Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Achondroplasia (Musculoskeletal Disorders) pipeline guide also reviews of key players involved in therapeutic development for Achondroplasia and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I and Preclinical stages are 1, 1, 2 and 2 respectively.

Achondroplasia (Musculoskeletal Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Achondroplasia (Musculoskeletal Disorders).

- The pipeline guide reviews pipeline therapeutics for Achondroplasia (Musculoskeletal Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.

- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.

- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.

- The pipeline guide reviews key companies involved in Achondroplasia (Musculoskeletal Disorders) therapeutics and enlists all their major and minor projects.

- The pipeline guide evaluates Achondroplasia (Musculoskeletal Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.

- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.

- The pipeline guide reviews latest news related to pipeline therapeutics for Achondroplasia (Musculoskeletal Disorders)

Reasons to buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.

- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.

- Find and recognize significant and varied types of therapeutics under development for Achondroplasia (Musculoskeletal Disorders).

- Classify potential new clients or partners in the target demographic.

- Develop tactical initiatives by understanding the focus areas of leading companies.

- Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.

- Formulate corrective measures for pipeline projects by understanding Achondroplasia (Musculoskeletal Disorders) pipeline depth and focus of Indication therapeutics.

- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.

- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Achondroplasia - Pipeline Review, H2 2018

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
BioClin Therapeutics Inc
BioMarin Pharmaceutical Inc
Ribomic Inc
Achondroplasia - Drug Profiles
ACP-015 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
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
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
Jun 21, 2018: Therachon Announces Start of Natural 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 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, H2 2018
Number of Products under Development by Companies, H2 2018
Products under Development by Companies, H2 2018
Number of Products by Stage and Target, H2 2018
Number of Products by Stage and Mechanism of Action, H2 2018
Number of Products by Stage and Route of Administration, H2 2018
Number of Products by Stage and Molecule Type, H2 2018
Achondroplasia - Pipeline by Ascendis Pharma A/S, H2 2018
Achondroplasia - Pipeline by BioClin Therapeutics Inc, H2 2018
Achondroplasia - Pipeline by BioMarin Pharmaceutical Inc, H2 2018
Achondroplasia - Pipeline by Ribomic Inc, H2 2018
Achondroplasia - Dormant Projects, H2 2018

List of Figures
Number of Products under Development for Achondroplasia, H2 2018
Number of Products under Development by Companies, H2 2018
Number of Products by Targets, H2 2018
Number of Products by Stage and Targets, H2 2018
Number of Products by Mechanism of Actions, H2 2018
Number of Products by Stage and Mechanism of Actions, H2 2018
Number of Products by Routes of Administration, H2 2018
Number of Products by Stage and Routes of Administration, H2 2018
Number of Products by Molecule Types, H2 2018
Number of Products by Stage and Molecule Types, H2 2018
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