Achondroplasia Treatment Market, By Product Type (RBM-007, TA-46, B-701, and Others), By End User (Hospital, Clinic, and Others), and By Region (North America, Europe, Asia Pacific, Latin America, and Middle East & Africa) - Trends, Analysis and Forecast till 2034

Achondroplasia Treatment Market was valued at US$ 120.4 million in 2024 and is projected to grow at a CAGR of 38.8 % to reach US$ 3208.6 million by 2034.

Achondroplasia refers to a genetic disorder characterized by dwarfism and specific physical features. Achondroplasia is known as short-limbed dwarfism where by the process called ossification cartilage is converted into bones which are observed more in the ligaments like long bones of legs and arms. No cure or prevention remedies for the rising prevalence of achondroplasia dwarfism are major factor to fuel the growth of the global market of achondroplasia treatment. It is caused by mutation in FGFR3 gene. Key features include short stature, disproportionate limbs, bowed legs, spinal issues. People have normal lifespan, intelligence. Regular medical monitoring and management by healthcare professionals, including orthopedic specialists, are important for addressing any potential complications.

Achondroplasia is a autosomal dominant genetic disorder. More than 300 different conditions can lead to dwarfism wherein achondroplasia is the most common type of dwarfism. It is associated with increased mortality in childhood, obesity and morbidity due to lumbar stenosis, and other nonspecific joint problems .Other genetic conditions, kidney diseases and problems with metabolism might also lead to dwarfism.

Management of achondroplasia is symptomatic and requires a multidisciplinary approach involving pharmacological and nonpharmacological interventions. The current treatment landscape has only one approved therapy in the US and Europe, VOXZOGO (vosoritide), which is indicated to increase linear growth in pediatric patients with achondroplasia with open epiphyses. It is a C-type natriuretic peptide (CNP) analog, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.

Disclaimer: This data is only a representation. Actual data may vary and will be available in the report.

DRIVERS:

• Increased incidences of Achondroplasia

Achondroplasia is most commonly occurring abnormality of bone growth and development, 1 in 20,000-30,000 live births. Exploring new ways to restore bone growth, spinal problems boosts the Achondroplasia treatment market for better quality of life for children with Achondroplasia.

Growing government initiative support for R&D, increasing acceptance among people regarding various disorder are also contributing factors toward the expansion of achondroplasia treatment market.

• Rising drug approval and launches

Achondroplasia treatment market is anticipated to increase because of the growing number of drug approvals and launches by significant market participants. The European Commission (EC) approved TransCon hGH as a once-weekly subcutaneous injection to treat pediatric patients experiencing growth failure due to insufficient endogenous growth hormone secretion or growth hormone deficiency. The company, endocrinology, produces products for the treatment of growth hormone deficiency. Somatropin is an ingredient of TransCon hGH, which provides the body with therapeutic doses of unaltered somatropin (hGH).

RESTRAINS:

Poor, insufficient, unfavorable reimbursement policies, lack of awareness hampers the achondroplasia treatment market growth. Financial constraints lead to less access to the treatment and recent advancements. Lack of awareness leads to delayed diagnosis and reduces the demand for treatment. Harmone therapy being effective for short duration and high cost hinders the Achondroplasia treatment market over the forecast period.

Major factor hindering the achondroplasia treatment market includes the side effects and high cost of treatment. Treatment cost may range from US$10-000-60,000 a year for hormone treatment alone. Limited pharmacological therapies, and surgeries having the risk of morbidity accounts for an urgent need for an effective therapy that can restore skeletal growth and improve patients’ quality of life.

Recent Development:

The New Launched Product News,

• In March 2023, BioMarin Pharmaceutical Inc.,a global biotechnology company dedicated to transforming lives through genetic discovery, announced acceptance of supplemental New Drug Application (sNDA) for VOXZOGO® (vosoritide) for injection to expand treatment in the United States to include children with achondroplasia under the age of 5 by U.S. Food and Drug Administration (FDA) .Achondroplasia is the most common form of disproportionate short stature. VOXZOGO is approved in the U.S. and indicated to increase linear growth in pediatric patients with achondroplasia who are 5 years of age and older with open epiphyses. This indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history.
• In May 2023, The Australian Government listed Voxzogo® (vosoritide) on the PBS(Pharmaceutical Benefits Scheme )for treatment of patients with achondroplasia whose growth plates are not yet closed. Voxzogo works directly on the growth points of the bones to promote new bone growth in patients whose bones are still growing. Without subsidy, Voxzogo could cost more than $330,000 per year. Through the PBS, Australian families with eligible children will pay a maximum of $30 per script or just $7.30 with a concession card.

Achondroplasia treatment market is segmented based on Product Type, End-User and Region.

Product Type Insights

On the basis of Product Type, the target market is segmented into RBM-007, TA-46, B-701, and Others. These are drugs used for treating achondroplasia. RBM-007 is a small compound which works by modulating the activity of FGF2 specifically, one of signaling molecule for FGFR3. gene specifically TA-46 is another experimental compound functioning in same way preventing FGF binding to mutant FGFR3RBM-007 is under development for treatment of achondroplasia whereas TA-46 has completed phase 1 trials and has received Orphan Drug Designation from the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA). B-701 is currently in clinical trials for urothelial cell carcinoma. No preclinical studies on achondroplasia have been published till date tends to boost the achondroplasia treatment market for development of various drug over the forecast period.

End-User Insights

On the basis of End User, the target market is segmented into Hospital, Clinic, and Others. Hospital segment account for the major share in the segment of achondroplasia treatment market due to specialized expertise, immediate medication, emergency care capabilities, insurance coverage, connections, and support groups. This is mostly in infants and thus hospitals accounts for major segment in the targeted market due to immediate lookup and care. Good focus on pediatric wards in hospitals also allows people to choose hospitals over other option boosting the hospital segment for achondroplasia treatment.

Regional Insights

• North America market is estimated to witness a significantly high revenue share over the forecast period driven by the high prevalence of achondroplasia, favorable health reimbursement, and increased awareness. In 2022, the US had the largest market size of achondroplasia among the 7MM, accounting for approximately USD 111.85 million, expected to increase further by 2032. Increasing developments in the healthcare system and rising health expenditures, as well as increased knowledge of advanced cancer treatments, are fueling the achondroplasia treatment market growth in region over the forecast period. U.S is one of the top countries in region contributing towards achondroplasia related research and development driving the growth of targeted market in the region.
• Asia Pacific market is estimated to witness a fastest revenue share over the forecast period due to advanced technological advancements in healthcare sectors, supportive guidelines by government and private organizations. Healthcare infrastructure, increasing awareness, increasing population expansion and health insurances boosts the achondroplasia treatment market in the region.

Report Scope:

Attribute	Details
Base year for estimation	2023
Forecast period	2024 – 2034
Market representation	Revenue in USD Billion & CAGR from 2024 to 2034
Market Segmentation	By Product Type – RBM-007, TA-46, B-701, and Others By End-User -  Hospital, Clinic, and Others
Regional scope	North America - U.S., Canada Europe - UK, Germany, Spain, France, Italy, Russia, Rest of Europe Asia Pacific - Japan, India, China, South Korea, Australia, Rest of Asia-Pacific Latin America - Brazil, Mexico, Argentina, Rest of Latin America Middle East & Africa - South Africa, Saudi Arabia, UAE, Rest of Middle East & Africa
Report coverage	Revenue forecast, company share, competitive landscape, growth factors, and trends

Segments Covered in the Report:

This report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends and opportunities in each of the sub-segments from 2024 to 2034.

Achondroplasia Treatment  Market, By Product Type:

• RBM-007
• TA-46, B-701
• Others

Achondroplasia Treatment  Market, By End-User:

• Hospital
• Clinic
• Others

Achondroplasia Treatment  Market, By Region:

• North America
  • U.S.
  • Canada
• Europe
  • Germany
  • UK
  • France
  • Russia
  • Italy
  • Rest of Europe
• Asia Pacific
  • China
  • India
  • Japan
  • South Korea
  • Rest of Asia Pacific
• Latin America
  • Brazil
  • Mexico
  • Rest of Latin America
• Middle East & Africa
  • GCC
  • Israel
  • South Africa
  • Rest of Middle East & Africa

The key players operating the Achondroplasia treatment market includes 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.

Research Objective and Assumption

• Preface
• Research Objectives
• Study Scope
• Years Considered for the study
• Assumptions
• Abbreviations

1. Research Methodology
   • Research data
   • Primary Data
     • Primary Interviews
     • Primary Breakdown
     • Key data from Primary Sources
     • Key Thickness Insights
   • Secondary Data
     • Major Secondary Sources
     • Secondary Sources
   • Market Estimation
   • Top-Down Approach
     • Approach for estimating Market Share by Top-Down Analysis (Supply Side)
   • Bottom-Up Approach
     • Approach for estimating market share by Bottom-up Analysis (Demand Side)
   • Market Breakdown and Data Triangulation
   • Research Assumptions
   • PORTER’s
   • Investment Outlook
2. Market Purview
   • Executive Summary
   • Key Findings—Global Outlook for Achondroplasia Treatment Strategies
     • Key Questions this Study will Answer

     • • By Product Type (RBM-007, TA-46, B-701, and Others)

       • By End User (Hospital, Clinic, and Others)

       • By Region (North America, Europe, Asia Pacific, Latin America, and Middle East & Africa)

   • Opportunity Map Analysis
   • Executive Summary—3 Big Predictions
3. Market Dynamics, Regulations, and Trends Analysis
   • Market Dynamics
     • Drivers
     • Restraints
     • Market Opportunities
     • Market Trends
   • DR Impact Analysis
   • PEST Analysis
   • Porter’s Five Forces Analysis
   • Opportunity Orbit
   • Overview
   • Market Value and Forecast (US$ Mn), and Share Analysis (%), 2020 – 2030
   • Y-o-Y Growth Analysis (%), 2020 – 2030
   • Regional Trends
   • Market Investment Feasibility Index
   • Macroeconomic Factor Analysis
4. Regional Analysis
   • Regional Revenue Analysis
   • Regional By segment Analysis
   • Reasons for regional growth
   • Key Player of particular region
   • Countries Include in the report:

1. • • North America (U.S., Canada)
     • Europe (UK, France, Germany, Russia, Italy, Rest of Europe)
     • Asia Pacific (India, Japan, South Korea, China, Rest of Asia Pacific)
     • Latin America (Brazil, Mexico, Rest of Latin America)
     • Middle East & Africa (GCC, Israel, South Africa, Rest of Middle East)

1. Competitive Landscape
   • Heat Map Analysis
   • Market Presence and Specificity Analysis
   • Competitive Overview
   • Competitive Benchmarking
   • Market Share Analysis
   • Key Developments In The Global Achondroplasia Treatment Market
   • Key Developments: Mergers & Acquisitions
   • Key Developments: Collaboration
   • Key Developments: Contracts & Agreements
   • Key Developments: Expansion
   • Key Developments: Product Developments
2. Company Profiles

 

• Company Overview
• Product Portfolio
• Key Highlights
• Financial Performance
• Business Strategies
• SWOT Analysis

• • Ascendis Pharma A/S
  • BioMarin Pharmaceutical Inc
  • Ribomic Inc.

1. The Last Word
   • Future Impact
   • About Us
   • Contact