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Achondroplasia (Musculoskeletal) Drug Pipeline Landscape Report 2021 -

DUBLIN--(BUSINESS WIRE)--The "Achondroplasia (Musculoskeletal) - Drugs In Development, 2021" report has been added to's offering.

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

The publisher'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 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)

Key Topics Covered:



  • 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
  • Aptamer for Achondroplasia - Drug Profile
  • ASP-5878 - Drug Profile
  • Fusion Protein to Agonize NPR2 for Achondroplasia - Drug Profile
  • infigratinib phosphate - Drug Profile
  • RBM-007 - Drug Profile
  • SAR-442501 - Drug Profile
  • TA-46 - Drug Profile
  • vosoritide - Drug Profile

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


For more information about this report visit

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Last Updated: 02-Sep-2021