Achondroplasia Market

Market Overview:

The 7 major achondroplasia markets reached a value of USD 136.8 Million in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 655.5 Million by 2035, exhibiting a growth rate (CAGR) of 15.31% during 2025-2035.

The achondroplasia market has been comprehensively analyzed in IMARC's new report titled "Achondroplasia Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2025-2035". Achondroplasia is a disease of bone development that results in disproportionate dwarfism. It develops due to the mutation of the FGFR3 gene, with the consequence that cartilage becomes unable to grow into bone with normal effectiveness and specificity, especially the long limbs of the legs and arms. Achondroplasia produces a short growth, shortened legs, an oversize head, and a usually normal torso size. Some other frequent complications involve restricted elbow extension, chronic ear infections, spinal stenosis, and sleep apnea due to obstruction. Achondroplasia results from spontaneous mutations but can also be inherited as autosomal dominant. Clinical examination, radiological imaging, and molecular testing for the confirmation of FGFR3 mutations are normally the basis of diagnosis. Fetal diagnosis may be achieved via ultrasound or genetic screening. Although there is no cure, new developments in targeted therapies, such as vosoritide, provide encouraging therapeutic alternatives with the potential to alter disease progression, enhance height results, and relieve complications of the disorder.

The achondroplasia market is underpinned primarily by its increasing prevalence and the ongoing development of targeted therapies to alter disease course. The most notable treatment advancement is vosoritide, a C-type natriuretic peptide (CNP) analog that inhibits FGFR3 overactivity, promoting normal bone growth. Vosoritide has demonstrated encouraging outcomes in clinical trials, resulting in regulatory approvals across major markets, thus increasing the availability of treatment. In addition, gene-editing therapy and small-molecule inhibitor development against FGFR3 mutations is picking up pace, with some candidates in preclinical and early-phase trials. Apart from the pharmacological developments, heightened awareness regarding early diagnosis and management is also leading to improved patient outcomes. The utilization of multidisciplinary care strategies, incorporating orthopedic, neurological, and respiratory professionals, is also enhancing the quality of life in patients. Additionally, regulatory rewards and orphan drug labeling are spurring pharma investment, hastening the arrival of new therapies. As increasingly numerous new treatment paradigms present themselves, achondroplasia's marketplace is on target for continued growth, holding potential for better control of the disease and better-quality life for affected patients.

IMARC Group's new report provides an exhaustive analysis of the achondroplasia market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the achondroplasia market in any manner.

Recent Developments:

• In September 2024, Ascendis Pharma's TransCon CNP showed a 5.89 cm/year growth increase in Phase II/III trials, positioning it as a competitor to Voxzogo.
• In September 2024, BridgeBio's infigratinib received FDA Breakthrough Therapy Designation after Phase II success, with Phase III enrolment expected to be completed by late 2024.
• In August 2024, RIBOMIC announced the completion of patient enrolment in an early Phase II study of umedaptanib pegol for achondroplasia and the initiation of dosing in Cohort 2 of the study.

Key Highlights:

• Achondroplasia affects 1 in 15,000 to 1 in 40,000 births, making it the most common short-limb dwarfism condition worldwide.
• A mutation in the FGFR3 gene disrupts cartilage-to-bone conversion, impairing bone growth, particularly in the arms and legs.
• Key features include short limbs (rhizomelic shortening), a large head with a prominent forehead, and distinct facial traits.
• It follows an autosomal dominant pattern, with 80% of cases from new mutations; affected individuals have a 50% chance of passing it on.
• Complications include apnea, ear infections, obesity, and spinal stenosis, requiring regular medical monitoring and early intervention for effective management.

Drugs:

Voxzogo (vosoritide), developed by BioMarin Pharmaceutical, is a subcutaneous injection designed to promote bone growth in children with achondroplasia who have open growth plates. It works by targeting the genetic pathway that regulates bone development, aiming to improve height outcomes in patients with this common form of dwarfism.

TYRA-300, developed by Tyra Biosciences, is an oral FGFR3-selective inhibitor in Phase 2 trials for achondroplasia. It aims to promote bone growth by targeting FGFR3 mutations. Recognized with FDA Orphan Drug and Rare Pediatric Disease designations, TYRA-300 represents a promising therapeutic approach for improving outcomes in achondroplasia patients.

Navepegritide (TransCon CNP) by Ascendis Pharma is an investigational prodrug designed for achondroplasia treatment. With a sustained-release mechanism, it delivers active C-type natriuretic peptide (CNP) weekly, supporting bone growth. Clinical studies indicate notable improvements in growth velocity and quality of life for children with achondroplasia.

Time Period of the Study

• Base Year: 2024
• Historical Period: 2019-2024
• Market Forecast: 2025-2035

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the achondroplasia market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the achondroplasia market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current achondroplasia marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the achondroplasia market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2024 and how are they expected to perform till 2035?
• What was the country-wise size of the achondroplasia market across the seven major markets in 2024 and what will it look like in 2035?
• What is the growth rate of the achondroplasia market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?

Epidemiology Insights

• What is the number of prevalent cases (​2019-2035) of achondroplasia across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of achondroplasia by age across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of achondroplasia by gender across the seven major markets?
• How many patients are diagnosed (​2019-2035) with achondroplasia across the seven major markets?
• What is the size of the achondroplasia patient pool (2019-2024) across the seven major markets?
• What would be the forecasted patient pool (2025-2035) across the seven major markets?
• What are the key factors driving the epidemiological trend of achondroplasia?
• What will be the growth rate of patients across the seven major markets?

Achondroplasia: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for achondroplasia drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the achondroplasia market?
• What are the key regulatory events related to the achondroplasia market?
• What is the structure of clinical trial landscape by status related to the achondroplasia market?
• What is the structure of clinical trial landscape by phase related to the achondroplasia market?
• What is the structure of clinical trial landscape by route of administration related to the achondroplasia market?