Achondroplasia (Musculoskeletal) – Drugs In Development, 2021

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Achondroplasia – Drugs In Development, 2021, 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 – Drugs In Development, 2021, 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 Pre-Registration, Phase II, Phase I, Preclinical and Discovery stages are 1, 3, 2, 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.

Companies mentioned

Ascendis Pharma AS

Astellas Pharma Inc

BioMarin Pharmaceutical Inc

BridgeBio Pharma Inc

Pfizer Inc

PhaseBio Pharmaceuticals Inc

Ribomic Inc

Sanofi

TagCyx Biotechnologies

Table of Contents

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 AS

Astellas Pharma Inc

BioMarin Pharmaceutical Inc

BridgeBio Pharma Inc

Pfizer Inc

PhaseBio Pharmaceuticals Inc

Ribomic Inc

Sanofi

TagCyx Biotechnologies

Achondroplasia - Drug Profiles

ACP-015 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Aptamer for Achondroplasia - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ASP-5878 - 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

SAR-442501 - 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 25, 2021: BioMarin receives positive CHMP opinion in Europe for Vosoritide for the treatment of children with Achondroplasia from age 2 until growth plates close

Apr 15, 2021: BioMarin announces new and updated data at 2021 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting demonstrating commitment to understanding achondroplasia and potential treatment choice

Mar 20, 2021: BioMarin announces oral presentation at ENDO2021, the Endocrine Society's annual meeting, with data demonstrating 2 years of treatment benefit in children with achondroplasia treated with Vosoritide

Mar 03, 2021: BioMarin completes full enrollment in phase 2 study of vosoritide for treatment of infants and young children with achondroplasia

Jan 07, 2021: VISEN Pharmaceuticals receives IND approval to initiate phase 2 clinical trial of TransCon CNP in achondroplasia (ACH) in China

Dec 21, 2020: BioMarin announces benefit maintained for over two years in children with achondroplasia treated with vosoritide in phase 3 extension study

Dec 15, 2020: Pfizer doses first participants as part of global achondroplasia phase 2 development program

Nov 09, 2020: BioMarin expands Vosoritide clinical program

Nov 02, 2020: Food and Drug Administration accepts BioMarin's new drug application for Vosoritide to treat children with achondroplasia

Oct 12, 2020: Novel drug may increase bone growth in children with achondroplasia

Sep 11, 2020: BioMarin announces presentation of Vosoritide phase 3 data in children with Achondroplasia at the American Society for Bone and Mineral Research 2020 Annual Meeting

Sep 08, 2020: BioMarin announces The Lancet publishes detailed vosoritide phase 3 data demonstrating statistically significant increase in annualized growth velocity (AGV) over 52 weeks in children with achondroplasia

Aug 20, 2020: BioMarin submits new drug application to U.S. Food and Drug Administration for Vosoritide to treat children with achondroplasia

Aug 13, 2020: European Medicines Agency validates BioMarin's Marketing Authorization Application for vosoritide to treat children with achondroplasia

Aug 12, 2020: Ascendis Pharma receives Orphan Designation for TransCon CNP for the treatment of achondroplasia in Europe

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, 2021

Number of Products under Development by Companies, 2021

Products under Development by Companies, 2021

Number of Products by Stage and Target, 2021

Number of Products by Stage and Mechanism of Action, 2021

Number of Products by Stage and Route of Administration, 2021

Number of Products by Stage and Molecule Type, 2021

Achondroplasia – Pipeline by Ascendis Pharma AS, 2021

Achondroplasia – Pipeline by Astellas Pharma Inc, 2021

Achondroplasia – Pipeline by BioMarin Pharmaceutical Inc, 2021

Achondroplasia – Pipeline by BridgeBio Pharma Inc, 2021

Achondroplasia – Pipeline by Pfizer Inc, 2021

Achondroplasia – Pipeline by PhaseBio Pharmaceuticals Inc, 2021

Achondroplasia – Pipeline by Ribomic Inc, 2021

Achondroplasia – Pipeline by Sanofi, 2021

Achondroplasia – Pipeline by TagCyx Biotechnologies, 2021

Achondroplasia – Dormant Projects, 2021

List of Figures

List of Figures

Number of Products under Development for Achondroplasia, 2021

Number of Products under Development by Companies, 2021

Number of Products by Targets, 2021

Number of Products by Stage and Targets, 2021

Number of Products by Mechanism of Actions, 2021

Number of Products by Stage and Mechanism of Actions, 2021

Number of Products by Routes of Administration, 2021

Number of Products by Stage and Routes of Administration, 2021

Number of Products by Molecule Types, 2021

Number of Products by Stage and Molecule Types, 2021

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