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

Publisher Global Markets Direct
Published May 31, 2022
Length 61 Pages
SKU # GMD17226467

Description

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

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Achondroplasia - Drugs In Development, 2022, 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, 2022, 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, 3 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 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.

Table of Contents

61 Pages
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
Lixte Biotechnology Holdings Inc
Pfizer Inc
PhaseBio Pharmaceuticals Inc
Ribomic Inc
Sanofi
Sisaf Ltd
TagCyx Biotechnologies
Tyra Biosciences Inc
Achondroplasia – Drug Profiles
ACP-015 – Drug Profile
Aptamer for Achondroplasia – Drug Profile
ASP-5878 – Drug Profile
CNP-ELP – Drug Profile
FGFR3 (ACH) – Drug Profile
infigratinib phosphate – Drug Profile
LB-100 – Drug Profile
RBM-007 – Drug Profile
recifercept – Drug Profile
SAR-442501 – Drug Profile
SIS-102-ACH – Drug Profile
vosoritide – Drug Profile
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
Table 1: Number of Products under Development for Achondroplasia, 2022
Table 2: Number of Products under Development by Companies, 2022
Table 3: Products under Development by Companies, 2022
Table 4: Number of Products by Stage and Target, 2022
Table 5: Number of Products by Stage and Mechanism of Action, 2022
Table 6: Number of Products by Stage and Route of Administration, 2022
Table 7: Number of Products by Stage and Molecule Type, 2022
Table 8: Achondroplasia – Pipeline by Ascendis Pharma AS, 2022
Table 9: Achondroplasia – Pipeline by Astellas Pharma Inc, 2022
Table 10: Achondroplasia – Pipeline by BioMarin Pharmaceutical Inc, 2022
Table 11: Achondroplasia – Pipeline by BridgeBio Pharma Inc, 2022
Table 12: Achondroplasia – Pipeline by Lixte Biotechnology Holdings Inc, 2022
Table 13: Achondroplasia – Pipeline by Pfizer Inc, 2022
Table 14: Achondroplasia – Pipeline by PhaseBio Pharmaceuticals Inc, 2022
Table 15: Achondroplasia – Pipeline by Ribomic Inc, 2022
Table 16: Achondroplasia – Pipeline by Sanofi, 2022
Table 17: Achondroplasia – Pipeline by Sisaf Ltd, 2022
Table 18: Achondroplasia – Pipeline by TagCyx Biotechnologies, 2022
Table 19: Achondroplasia – Pipeline by Tyra Biosciences Inc, 2022
Table 20: Achondroplasia – Dormant Projects, 2022
List of Figures
Figure 1: Number of Products under Development for Achondroplasia, 2022
Figure 2: Number of Products under Development by Companies, 2022
Figure 3: Number of Products by Targets, 2022
Figure 4: Number of Products by Stage and Targets, 2022
Figure 5: Number of Products by Mechanism of Actions, 2022
Figure 6: Number of Products by Stage and Mechanism of Actions, 2022
Figure 7: Number of Products by Routes of Administration, 2022
Figure 8: Number of Products by Stage and Top 10 Routes of Administration, 2022
Figure 9: Number of Products by Molecule Types, 2022
Figure 10: Number of Products by Stage and Molecule Types, 2022

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