Achondroplasia Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Pages: 61 Published: May 31, 2022 Report Code: GDGMDHC22177IDB

Achondroplasia pipeline market research report provides comprehensive information on the therapeutics under development for Achondroplasia, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Achondroplasia and features dormant and discontinued projects.

Key Targets in the Achondroplasia Pipeline Market

The key targets in the Achondroplasia pipeline market are Fibroblast Growth Factor Receptor 3, Fibroblast Growth Factor Receptor 1, Fibroblast Growth Factor Receptor 2, Fibroblast Growth Factor Receptor 4, Atrial Natriuretic Peptide Receptor 2, Fibroblast Growth Factor), Fibroblast Growth Factor 2, and Protein Phosphatase 2A. Fibroblast Growth Factor Receptor 3 has the highest number of pipeline products.

Achondroplasia Pipeline Market, by Targets

Achondroplasia Pipeline Market, by Targets

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Key Types of MoA in the Achondroplasia Pipeline Market

The key types of MoA in the Achondroplasia pipeline market are Fibroblast Growth Factor Receptor 3 Inhibitor, Fibroblast Growth Factor Receptor 1 Inhibitor, Fibroblast Growth Factor Receptor 2 Inhibitor, Fibroblast Growth Factor Receptor 4 Inhibitor, Atrial Natriuretic Peptide Receptor 2 Agonist, Fibroblast Growth Factor Inhibitor, Fibroblast Growth Factor Receptor 3 Antagonist, Fibroblast Growth Factor 2 Inhibitor, and Protein Phosphatase 2A Inhibitor. Fibroblast Growth Factor Receptor 3 Inhibitor has the maximum number of pipeline products.

Achondroplasia Pipeline Market, by Types of MoA

Achondroplasia Pipeline Market, by Types of MoA

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Key Types of RoA in the Achondroplasia Pipeline Market

The key types of RoA in the Achondroplasia pipeline market are Subcutaneous, Oral, Intravenous, and Intravitreal. Subcutaneous has the highest share in the Achondroplasia pipeline market.

Achondroplasia Pipeline Market, by Types of RoA

Achondroplasia Pipeline Market, by Types of RoA

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Key Molecule Types in the Achondroplasia Pipeline Market

The key molecule types in the Achondroplasia pipeline market are small molecule, aptamer, recombinant protein, synthetic peptide, antisense rnai oligonucleotide, fusion protein, monoclonal antibody, and recombinant peptide. Small molecule has the highest number of pipeline products.

Achondroplasia Pipeline Market, by Molecule Types

Achondroplasia Pipeline Market, by Molecule Types

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Key Companies in the Achondroplasia Pipeline Market

The key companies in the Achondroplasia pipeline market are Ascendis Pharma AS, BridgeBio Pharma Inc, Pfizer Inc, Astellas Pharma Inc, BioMarin Pharmaceutical Inc, Lixte Biotechnology Holdings Inc, PhaseBio Pharmaceuticals Inc, Ribomic Inc, Sanofi, Sisaf Ltd, TagCyx Biotechnologies, and Tyra Biosciences Inc. Ascendis Pharma AS has the highest pipeline products.

Achondroplasia Pipeline Market, by Companies

Achondroplasia Pipeline Market, by Companies

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Market Report Overview

Key Targets Fibroblast Growth Factor Receptor 3, Fibroblast Growth Factor Receptor 1, Fibroblast Growth Factor Receptor 2, Fibroblast Growth Factor Receptor 4, Atrial Natriuretic Peptide Receptor 2, Fibroblast Growth Factor), Fibroblast Growth Factor 2, and Protein Phosphatase 2A
Key MoA Types Fibroblast Growth Factor Receptor 3 Inhibitor, Fibroblast Growth Factor Receptor 1 Inhibitor, Fibroblast Growth Factor Receptor 2 Inhibitor, Fibroblast Growth Factor Receptor 4 Inhibitor, Atrial Natriuretic Peptide Receptor 2 Agonist, Fibroblast Growth Factor Inhibitor, Fibroblast Growth Factor Receptor 3 Antagonist, Fibroblast Growth Factor 2 Inhibitor, and Protein Phosphatase 2A Inhibitor
Key RoA Types Subcutaneous, Oral, Intravenous, and Intravitreal
Key Molecule Types Small Molecule, Aptamer, Recombinant Protein, Synthetic Peptide, Antisense RNAi Oligonucleotide, Fusion Protein, Monoclonal Antibody, and Recombinant Peptide
Key Companies Ascendis Pharma AS, BridgeBio Pharma Inc, Pfizer Inc, Astellas Pharma Inc, BioMarin Pharmaceutical Inc, Lixte Biotechnology Holdings Inc, PhaseBio Pharmaceuticals Inc, Ribomic Inc, Sanofi, Sisaf Ltd, TagCyx Biotechnologies, and Tyra Biosciences Inc

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.

Key Players

  • Ascendis Pharma AS

    Astellas Pharma Inc

    BioMarin Pharmaceutical Inc

    BridgeBio Pharma Inc

    Lixte Biotechnology Holdings Inc

    Pfizer Inc

    PhaseBio Pharmaceuticals Inc

    Ribomic Inc

    Sanofi

    Sisaf Ltd

    TagCyx Biotechnologies

    Tyra Biosciences Inc

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

Achondroplasia – Drug Profiles

Achondroplasia – Dormant Projects

Achondroplasia – Product Development Milestones

Featured News & Press Releases

Apr 19, 2022: RIBOMIC announces completion of IND submission for an observational study for continuous phase 2 trial of RBM-007 for treatment of Achondroplasia

Nov 19, 2021: BioMarin receives FDA approval for VOXZOGO (vosoritide) for injection, indicated to increase linear growth in children with achondroplasia aged 5 and up with open growth plates

Nov 19, 2021: FDA approves first drug to improve growth in children with most common form of dwarfism

Nov 15, 2021: RIBOMIC announces RBM-007 phase 1 clinical trial results for achondroplasia

Aug 30, 2021: BioMarin’s Voxzogo receives EC approval for achondroplasia in children

Jul 22, 2021: Lixte Biotechnology comments on report that its PP2A inhibitor LB-100 combined with another investigational agent stimulates bone growth in models of dwarfism

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

May 12, 2021: RIBOMIC announces completion of its phase I trial of RBM-007 for the treatment of achondroplasia

May 06, 2021: RIBOMIC announces publication of research paper on aptamer drug discovery for achondroplasia

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

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, 2022

Number of Products under Development by Companies, 2022

Products under Development by Companies, 2022

Number of Products by Stage and Target, 2022

Number of Products by Stage and Mechanism of Action, 2022

Number of Products by Stage and Route of Administration, 2022

Number of Products by Stage and Molecule Type, 2022

Achondroplasia – Pipeline by Ascendis Pharma AS, 2022

Achondroplasia – Pipeline by Astellas Pharma Inc, 2022

Achondroplasia – Pipeline by BioMarin Pharmaceutical Inc, 2022

Achondroplasia – Pipeline by BridgeBio Pharma Inc, 2022

Achondroplasia – Pipeline by Lixte Biotechnology Holdings Inc, 2022

Achondroplasia – Pipeline by Pfizer Inc, 2022

Achondroplasia – Pipeline by PhaseBio Pharmaceuticals Inc, 2022

Achondroplasia – Pipeline by Ribomic Inc, 2022

Achondroplasia – Pipeline by Sanofi, 2022

Achondroplasia – Pipeline by Sisaf Ltd, 2022

Achondroplasia – Pipeline by TagCyx Biotechnologies, 2022

Achondroplasia – Pipeline by Tyra Biosciences Inc, 2022

Achondroplasia – Dormant Projects, 2022

List of Figures

List of Figures

Number of Products under Development for Achondroplasia, 2022

Number of Products under Development by Companies, 2022

Number of Products by Targets, 2022

Number of Products by Stage and Targets, 2022

Number of Products by Mechanism of Actions, 2022

Number of Products by Stage and Mechanism of Actions, 2022

Number of Products by Routes of Administration, 2022

Number of Products by Stage and Top 10 Routes of Administration, 2022

Number of Products by Molecule Types, 2022

Number of Products by Stage and Molecule Types, 2022

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