Achondroplasia - Pipeline Review, H1 2020

March 2020 | 60 pages | ID: A96241206E6EN
Global Markets Direct

Abstracts

Achondroplasia - Pipeline Review, H1 2020

SUMMARY

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Achondroplasia - Pipeline Review, H1 2020, 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, H1 2020, 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, IND/CTA Filed, Preclinical and Discovery stages are 1, 1, 1, 1, 2 and 1 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.

Contents

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
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
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
Jan 08, 2020: Wacker Biotech provides active ingredient to treat achondroplasia in children for Ascendis Pharma global phase-2 trial
Dec 16, 2019: BioMarin announces positive final results from placebo-controlled phase 3 data in children with achondroplasia treated with Vosoritide
Nov 14, 2019: BioMarin announces cumulative additional height gain of 9.0 cm over 54 months versus natural 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 International Skeletal Dysplasia Society Meeting
Jul 18, 2019: Ascendis Pharma announces filing of Investigational 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 Journal 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 designation 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 preliminary 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 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
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List Of Tables

LIST OF TABLES

Number of Products under Development for Achondroplasia, H1 2020
Number of Products under Development by Companies, H1 2020
Products under Development by Companies, H1 2020
Number of Products by Stage and Target, H1 2020
Number of Products by Stage and Mechanism of Action, H1 2020
Number of Products by Stage and Route of Administration, H1 2020
Number of Products by Stage and Molecule Type, H1 2020
Achondroplasia - Pipeline by Ascendis Pharma A/S, H1 2020
Achondroplasia - Pipeline by BioMarin Pharmaceutical Inc, H1 2020
Achondroplasia - Pipeline by Pfizer Inc, H1 2020
Achondroplasia - Pipeline by PhaseBio Pharmaceuticals Inc, H1 2020
Achondroplasia - Pipeline by QED Therapeutics Inc, H1 2020
Achondroplasia - Pipeline by Ribomic Inc, H1 2020
Achondroplasia - Dormant Projects, H1 2020

List Of Figures

LIST OF FIGURES

Number of Products under Development for Achondroplasia, H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products by Targets, H1 2020
Number of Products by Stage and Targets, H1 2020
Number of Products by Mechanism of Actions, H1 2020
Number of Products by Stage and Mechanism of Actions, H1 2020
Number of Products by Routes of Administration, H1 2020
Number of Products by Stage and Routes of Administration, H1 2020
Number of Products by Molecule Types, H1 2020
Number of Products by Stage and Molecule Types, H1 2020

COMPANIES MENTIONED

Ascendis Pharma A/S
BioMarin Pharmaceutical Inc
Pfizer Inc
PhaseBio Pharmaceuticals Inc
QED Therapeutics Inc
Ribomic Inc