Achondroplasia is a form of short-limbed dwarfism. Achondroplasia is characterized by an average-sized trunk, short arms and legs, notably short upper arms and thighs, a limited range of motion at the elbows, and an enlarged head. Spinal stenosis, a narrowing of the spinal canal that can compress the upper section of the spinal cord, is a potentially serious complication of achondroplasia. Spinal stenosis is characterized by leg pain, tingling, and weakness, which can make walking difficult. Achondroplasia is caused by mutations in the FGFR3 gene. The FGFR3 gene is responsible for giving instructions when making a protein that is involved in the development and maintenance of bone and brain tissue.

Market Dynamics

Increasing approvals of achondroplasia treatment drugs by regulatory bodies are expected to drive the market growth over the forecast period. For instance, in  November 2021, BioMarin, a global biotechnology company, announced that the U.S. Food and Drug Administration (FDA) had granted accelerated approval to VOXZOGO (vosoritide) for injection, indicated to increase linear growth in pediatric patients with achondroplasia of five years of age and older with open epiphyses (growth plates). Moreover, in August 2021, QED Therapeutics, a subsidiary of BridgeBio Pharma, Inc., announced that the European Commission (EC) had granted marketing authorization for VOXZOGO (vosoritide), a once-daily injection to treat achondroplasia in children from the age of 2 until growth plates are closed, which occurs after puberty when children reach final adult height.

Also, increasing research and development by key players for the treatment of achondroplasia is expected to drive the treatment segment growth over the forecast period. For instance, in November 2021, RIBOMIC, Inc., a clinical stage pharmaceutical company specialized in aptamer therapeutics, announced the results from its Phase 1, healthy volunteer clinical study using RBM-007 for the planned treatment of achondroplasia, which was completed in May 2021.

Key features of the study:

• This report provides an in-depth analysis of the global achondroplasia treatment market and provides market size (US$ Million) and compound annual growth rate (CAGR) for the forecast period (2023–2030), considering 2022 as the base year
• It elucidates potential revenue opportunities across different segments and explains attractive investment proposition matrices for this market.
• This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, market trends, regional outlook, and competitive strategies adopted by key players
• It profiles key players in the global Achondroplasia Treatment market based on the following parameters– Company highlights, products portfolio, key highlights, financial performance, and strategies
• Key companies covered as a part of this study include BioMarin, RIBOMIC, Ascendis Pharma A/S, BridgeBio Pharma, Inc., Pfizer Inc., PhaseBio Pharmaceuticals, Inc., SiSaf, Novo Nordisk A/S, F. Hoffmann-La Roche Ltd, LG Chem, Ferring B.V., JCR Pharmaceuticals Co., Ltd, KVK TECH, INC., VIVUS LLC., ProLynx Inc., Teva Pharmaceutical Industries Ltd., Eli Lilly and Company, Ipsen Pharma, Novartis AG, and Xiamen Amoytop Biotech Co., Ltd.
• Insights from this report would allow marketers and the management authorities of the companies to make informed decisions regarding their future product launches, type up-gradation, market expansion, and marketing tactics
• The global achondroplasia treatment market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
• Stakeholders would have ease in decision-making through various strategy matrices used in analyzing the global achondroplasia treatment market.

Detailed Segmentation:

• Global Achondroplasia Treatment Market, By Treatment Type:
  • Vosoritide
  • Human Growth Hormone
  • Others
• Global Achondroplasia Treatment Market, By Route of Administration:
  • Oral
  • Parenteral
• Global Achondroplasia Treatment Market, By Distribution Channel:
  • Hospital Pharmacies
  • Retail Pharmacies
  • Online Pharmacies
• Global Achondroplasia Treatment, By Region:
  • North America
    
    • U.S.
    • Canada
  • Latin America
    
    • Brazil
    • Mexico
    • Argentina
    • Rest of Latin America
  • Europe
    
    • Germany
    • U.K.
    • France
    • Italy
    • Spain
    • Russia
    • Rest of Europe
  • Asia Pacific
    
    • China
    • India
    • Japan
    • Australia
    • South Korea
    • ASEAN
    • Rest of Asia Pacific
  • Middle East
    
    • GCC
    • Israel
    • Rest of Middle East
  • Africa
    
    • South Africa
    • Central Africa
    • North Africa
• Company Profiles
  • BioMarin
  • RIBOMIC
  • Ascendis Pharma A/S
  • BridgeBio Pharma, Inc.
  • Pfizer Inc.
  • PhaseBio Pharmaceuticals, Inc.
  • SiSaf
  • Novo Nordisk A/S
  • Hoffmann-La Roche Ltd
  • LG Chem
  • Ferring B.V.
  • JCR Pharmaceuticals Co., Ltd
  • KVK TECH, INC.
  • VIVUS LLC.
  • ProLynx Inc.
  • Teva Pharmaceutical Industries Ltd.
  • Eli Lilly and Company
  • Ipsen Pharma
  • Novartis AG
  • Xiamen Amoytop Biotech Co., Ltd.