The achondroplasia market was valued at USD 174.45 Million in 2024, driven by the increasing understanding and awareness of genetic disorders, across the 8 major markets. It is expected to grow at a CAGR of 36.50% during the forecast period of 2025-2034, with the values likely to reach USD 3917.40 Million by 2034.
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The market is witnessing growth driven by increasing research into gene therapies and targeted treatments, offering the potential for improved management of the condition and better patient outcomes.
The rising awareness of rare genetic disorders, along with advancements in diagnostic technologies, is leading to earlier diagnosis of achondroplasia, enabling timely interventions and driving demand for treatment options.
The growing focus on personalised medicine and genetic research is paving the way for tailored treatments for achondroplasia, offering more effective and specific therapeutic options catering to the unique needs of patients, thus supporting market growth.
Achondroplasia is a genetic disorder causing short stature and disproportionately short limbs, due to abnormal bone growth. It is the most common form of dwarfism, caused by a mutation in the FGFR3 gene. Symptoms include a larger head, limited joint mobility, and potential complications affecting the spine and respiratory system.
FDA Approvals Drive Market Growth
Increasing investment in genetic disorder therapies and rising demand for targeted treatments are key drivers in the achondroplasia market. In September 2024, BridgeBio Pharma announced that its oral drug infigratinib, being developed for children with achondroplasia, received a Breakthrough Therapy Designation from the FDA. This designation aims to fast-track the drug’s development due to its potential to substantially improve clinical outcomes over existing therapies. This development is poised to significantly accelerate the market’s growth by fostering innovation and expediting product availability, expanding treatment options for achondroplasia patients.
Surge in Clinical Trial Initiatives to Meet Rising Achondroplasia Market Demand
The growing focus on rare genetic diseases and advancements in targeted therapies are major drivers for the achondroplasia market. In October 2024, Tyra Biosciences announced that the FDA cleared its Investigational New Drug (IND) application for TYRA-300, an oral FGFR3-selective inhibitor for children with achondroplasia. The clearance allows the company to proceed with a Phase 2 trial. This promising first-in-class treatment could provide a safer and more effective option for achondroplasia patients, potentially transforming the treatment landscape and fostering market expansion in the forecast period.
The market is witnessing several trends and developments to improve the current scenario. Some of the notable trends are as follows:
The market report offers a detailed analysis of the market based on the following segments:
Market Breakup by Treatment
Market Breakup by Route of Administration
Market Breakup by End User
Market Breakup by Region
The growth hormone therapy is likely to hold a significant market share based on treatment. This is primarily due to its widespread use in children with achondroplasia to promote growth and improve height. Growth hormone therapy is considered the most effective non-invasive treatment to stimulate bone growth, especially when administered early in a child’s development. As per the analysis by Expert Market Research, the global human growth hormone market is anticipated to grow at a CAGR of 8.5% during the forecast period of 2025-2034. The therapy’s ability to provide significant improvements in stature, alongside relatively well-established treatment protocols, drives its dominance. Additionally, ongoing research into optimising growth hormone formulations and their long-term benefits continues to fuel its market leadership.
The United States holds the largest market share in the market, driven by its strong healthcare infrastructure and advanced research capabilities. The region leads in genetic research and clinical trials for rare diseases, with a high concentration of pharmaceutical companies focusing on rare genetic disorders like achondroplasia. Moreover, the United States benefits from robust reimbursement systems and significant government funding for rare disease therapies, enabling faster access to new treatments and therapies. The country's focus on personalised medicine and gene therapy innovation further fuels its market dominance.
The key features of the market report comprise patent analysis, grants analysis, clinical trials analysis, funding and investment analysis, and strategic initiatives by the leading players. The major companies in the market are as follows:
BioMarin Pharmaceutical Inc., founded in 1997 and headquartered in San Rafael, California, USA, is a global biopharmaceutical company specialising in treatments for rare genetic disorders. In the achondroplasia market, BioMarin is developing vosoritide, a first-in-class peptide therapy aimed at stimulating bone growth in children with achondroplasia. For instance, in March 2023, the FDA accepted the supplemental New Drug Application (sNDA) for VOXZOGO (vosoritide) to expand its use in the U.S. to treat children under 5 with achondroplasia, a common form of short stature.
QED Therapeutics, established in 2016 and headquartered in San Francisco, California, USA, is a biopharmaceutical company focused on developing therapies for rare genetic disorders. In the achondroplasia market, QED is focused on the development of small molecule inhibitors targeting the FGFR3 pathway, which is crucial in the development of achondroplasia. Their research aims to develop a treatment that can address the underlying cause of the condition, potentially offering a disease-modifying therapy for affected individuals.
Ascendis Pharma A/S, founded in 2006 and headquartered in Hellerup, Denmark, is a biotechnology company developing therapies for rare diseases. Ascendis Pharma is advancing TransCon CNP, a novel treatment for achondroplasia. This innovative therapy is designed to provide continuous activity of C-type natriuretic peptide (CNP), aiming to improve bone growth in children with achondroplasia. Ascendis Pharma’s approach offers a promising treatment option that could enhance growth rates and quality of life for affected patients.
Pfizer Inc., founded in 1849 and headquartered in New York City, USA, is a global leader in pharmaceuticals and vaccines. In the achondroplasia market, Pfizer is collaborating with other biopharma companies on the development of therapies for rare diseases. Pfizer’s research is focused on developing novel treatments that target the genetic and molecular pathways involved in conditions like achondroplasia, with a focus on advancing innovative therapies to address unmet needs in pediatric and adult populations affected by growth disorders. For instance, in May 2019, Pfizer announced its acquisition of Therachon Holding AG for USD 340 million upfront, with up to USD 470 million in milestone payments. Therachon’s assets include treatments for achondroplasia and short bowel syndrome, conditions with significant unmet medical needs.
*Please note that this is only a partial list; the complete list of key players is available in the full report. Additionally, the list of key players can be customized to better suit your needs.*
Other key players in the market include Ribomic Inc., Astellas Pharma Inc., Sanofi S.A., and Novo Nordisk A/S.
*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*
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| REPORT FEATURES | DETAILS |
| Base Year | 2024 |
| Historical Period | 2018-2024 |
| Forecast Period | 2025-2034 |
| Scope of the Report |
Historical and Forecast Trends, Industry Drivers and Constraints, Historical and Forecast Market Analysis by Segment:
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| Breakup by Treatment |
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| Breakup by Route of Administration |
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| Breakup by End User |
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| Breakup by Region |
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| Market Dynamics |
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| Supplier Landscape |
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| Companies Covered |
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United States (Head Office)
30 North Gould Street, Sheridan, WY 82801
+1-415-325-5166
Australia
63 Fiona Drive, Tamworth, NSW
+61-448-061-727
India
C130 Sector 2 Noida, Uttar Pradesh 201301
+91-723-689-1189
Philippines
40th Floor, PBCom Tower, 6795 Ayala Avenue Cor V.A Rufino St. Makati City, 1226.
+63-287-899-028, +63-967-048-3306
United Kingdom
6 Gardner Place, Becketts Close, Feltham TW14 0BX, Greater London
+44-753-713-2163
Vietnam
193/26/4 St.no.6, Ward Binh Hung Hoa, Binh Tan District, Ho Chi Minh City
+84-865-399-124
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