Achondroplasia Market: Epidemiology, Therapies, Companies, DelveInsight | Ascendis Pharma, QED Therapeutics (BridgeBio), Novartis, Sanofi, RIBOMIC

Achondroplasia therapies, such as VOXZOGO (vosoritide), RBM-007, SAR-442501, Low dose infigratinib, and others, are expected to boost the Achondroplasia Market in the upcoming years.

DelveInsight has launched a new report on "Achondroplasia - Market Insights, Epidemiology, and Market Forecast-2034" that delivers an in-depth understanding of the Achondroplasia, historical and forecasted epidemiology as well as the Achondroplasia market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

Discover about the Achondroplasia market report @ https://www.delveinsight.com/report-store/achondroplasia-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Some of the key facts of the Achondroplasia Market Report:

The achondroplasia market is expected to grow steadily, driven by advancements in genetic therapies, better diagnosis rates, and increased R&D investments.
In March 2025, Ascendis Pharma submitted a New Drug Application (NDA) to the FDA for TransCon CNP (navepegritide), a once-weekly injectable prodrug designed to provide continuous exposure to C-type natriuretic peptide (CNP) . This investigational therapy aims to support bone growth and muscle function in children with achondroplasia. The NDA is supported by data from three randomized, double-blind, placebo-controlled clinical trials, including up to three years of open-label extension data.
In September 2024, the FDA granted Breakthrough Therapy Designation to oral infigratinib, developed by BridgeBio Pharma, for treating children with achondroplasia . This designation was based on Phase 2 PROPEL 2 trial results, which showed a sustained increase in annualized height velocity and improved body proportionality over 18 months. If approved, infigratinib would be the first oral therapy for achondroplasia.
In 2023, the U.S. reported around 14,500 diagnosed prevalent cases. Market dynamics are shifting with the emergence of novel treatments and increased healthcare spending.
BioMarin's VOXZOGO, the first FDA-approved therapy (approved in 2021), marked a key milestone. However, unmet needs remain, including targeted treatments, early diagnosis, and comprehensive patient support.
DelveInsight's report provides in-depth market insights, epidemiology, and forecasts through 2034 across the U.S., EU4, UK, and Japan, highlighting ongoing and future opportunities in this rare skeletal disorder market.
In 2023, the combined diagnosed prevalent cases of achondroplasia in the US and Japan totaled approximately 18,000, with numbers projected to increase over time. In the US, females made up a slightly larger share of cases (51.2%) than males (48.8%), potentially due to underlying genetic or biological influences. Japan accounted for around 3,000 diagnosed cases that same year.
Key players in the treatment and prevention landscape are BioMarin Pharmaceuticals, Tyra Biosciences, Inc., RIBOMIC Inc., Bridge Bio Inc., Sanofi, and others.
Promising Achondroplasia therapies include VOXZOGO (vosoritide), RBM-007, SAR-442501, Low dose infigratinib, and others.

Achondroplasia Overview

Achondroplasia is a rare genetic disorder caused by a mutation in the FGFR3 gene, which disrupts bone growth and leads to dwarfism. The mutation slows bone formation at growth plates, affecting nearly all bones. Although inherited in an autosomal dominant manner, over 80% of cases arise from new mutations in families with no history of the condition. Key physical traits include short limbs, a large head with a prominent forehead, a flat nasal bridge, spinal curvature, and "trident"-shaped fingers. Average adult height is about 4'2" for females and 4'4" for males.

Achondroplasia Market Outlook

Achondroplasia, a genetic condition and leading cause of dwarfism, has traditionally been managed with supportive care and surgeries to address complications like bone deformities and spinal issues-without impacting the core growth issue. Recent medical advances, however, are changing the treatment landscape. Vosoritide, a daily injectable targeting the FGFR3 pathway, has shown success in improving growth rates in children. A newer therapy under development, TransCon CNP, aims to offer longer-lasting effects with less frequent dosing. Additionally, gene therapy research is underway, aiming to correct the root FGFR3 mutation and potentially provide a lasting cure. These emerging therapies represent a major shift from symptom management to addressing the underlying cause of the disorder.

Discover how the Achondroplasia market is rising in the coming years @ https://www.delveinsight.com/sample-request/achondroplasia-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Achondroplasia Marketed Drugs

VOXZOGO (vosoritide): BioMarin Pharmaceuticals

Achondroplasia Emerging Drugs
RBM-007: RIBOMIC Inc.
SAR-442501: Sanofi
Low dose infigratinib: BridgeBio Inc.
Scope of the Achondroplasia Market Report
Study Period: 2020-2034
Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
Key Achondroplasia Companies: Ascendis Pharma, QED Therapeutics (BridgeBio), Novartis, Sanofi, RIBOMIC, and others
Key Achondroplasia Therapies: VOXZOGO (vosoritide), RBM-007, SAR-442501, Low dose infigratinib, and others
Achondroplasia Therapeutic Assessment: Achondroplasia current marketed and Achondroplasia emerging therapies
Achondroplasia Market Dynamics: Achondroplasia market drivers and Achondroplasia market barriers
Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
Achondroplasia Unmet Needs, KOL's views, Analyst's views, Achondroplasia Market Access and Reimbursement

To know what's more in our Achondroplasia report, visit https://www.delveinsight.com/report-store/achondroplasia-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Key benefits of the Achondroplasia Market Report:
Achondroplasia market report covers a descriptive overview and comprehensive insight of the Achondroplasia Epidemiology and Achondroplasia market in the 7MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan).
The Achondroplasia market report provides insights into the current and emerging therapies.
The Achondroplasia market report provides a global historical and forecasted market covering drug outreach in 7MM.
The Achondroplasia market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Achondroplasia market.

Got queries? Click here to know more about the Achondroplasia market Landscape https://www.delveinsight.com/sample-request/achondroplasia-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Table of Contents

1. Report Introduction
2. Executive Summary
3. SWOT analysis
4. Achondroplasia Patient Share (%) Overview at a Glance
5. Achondroplasia Market Overview at a Glance
6. Achondroplasia Disease Background and Overview
7. Achondroplasia Epidemiology and Patient Population
8. Country-Specific Patient Population of Achondroplasia
9. Achondroplasia Current Treatment and Medical Practices
10. Unmet Needs
11. Achondroplasia Emerging Therapies
12. Achondroplasia Market Outlook
13. Country-Wise Achondroplasia Market Analysis (2020-2034)
14. Market Access and Reimbursement of Therapies
15. Market drivers
16. Market barriers
17. Appendix
18. Achondroplasia Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight

Click here to read more about Achondroplasia Market Outlook 2034 https://www.delveinsight.com/report-store/achondroplasia-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Related Reports:

Achondroplasia Pipeline Insights, DelveInsight

"Achondroplasia Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Achondroplasia market. A detailed picture of the Achondroplasia pipeline landscape is provided, which includes the disease overview and Achondroplasia treatment guidelines.

Contact Info:

Kritika Rehani, Team Leader Marketing & Branding
krehani@delveinsight.com
info@delveinsight.com
www.delveinsight.com
+14699457679

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

This release was published on openPR.