Achondroplasia - Pipeline Review, H1 2018

  • ID: 4479895
  • Drug Pipelines
  • 43 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • Ascendis Pharma A/S
  • Ribomic Inc
  • MORE
Achondroplasia - Pipeline Review, H1 2018

Summary:

This latest Pharmaceutical and Healthcare disease pipeline guide Achondroplasia - Pipeline Review, H1 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:

This Pharmaceutical and Healthcare latest pipeline guide Achondroplasia - Pipeline Review, H1 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, 1 and 3 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 the publisher'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 for Purchase:
  • 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.
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Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • Ascendis Pharma A/S
  • Ribomic Inc
  • MORE
Introduction

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 2

Feb 28, 2017: Ascendis Pharma Announces Data on TransCon CNP at ENDO 2

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

Secondary 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 2018

List 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
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  • Ascendis Pharma A/S
  • BioMarin Pharmaceutical Inc
  • Ribomic Inc
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