Achondroplasia
Achondroplasia is a rare genetic bone growth disorder that results in marked short stature (dwarfism) due to a mutation in the ﬁbroblast growth factor receptor 3 (FGFR3) gene. The mutation leads to a gain-of-function of the FGFR3 gene, which slows down the formation of bone in the cartilage of the growth plate and impairs growth in almost all bones in the body.
Achondroplasia Diagnosis
<ul>
<li>Molecular genetic testing</li>
<li>Prenatal diagnosis</li>
<li>Preimplantation genetic diagnosis</li>
<li>Skeletal surveys</li>
</ul>
&nbsp;
Achondroplasia Epidemiology Insights (2022)
The total diagnosed prevalent cases in the 7MM were approximately 28.6K in 2022
<ul>
<li>The United States accounted for the 51% of the total prevalent cases in the 7MM</li>
<li>The EU4 and the UK&nbsp; accounted for the 38% of the total prevalent cases in the 7MM</li>
<li>The Japan accounted for the 11% of the total prevalent cases in the 7MM</li>
</ul>
Around 51% of males and 49% of females were diagnosed with achondroplasia in the US
Current Achondroplasia Treatment Options
<ul>
<li>Pharmacological therapy</li>
<li>Non Pharmacological therapy</li>
<li>Psychological support</li>
<li>Surgery</li>
</ul>
Achondroplasia Market Insight (2022)
The Achondroplasia Market size in the 7MM was valued approximately USD 198 million in 2022
<ul>
<li>The United States accounted for the 56% of the total market size in the 7MM</li>
<li>The EU4 and the UK accounted for the 36% of the total market size in the 7MM&nbsp;</li>
<li>The Japan accounted for the 8% of the total market size in the 7MM</li>
</ul>
A total of ~USD 87 million of revenue was accounted by BioMarin Pharmaceutical and Chugai Pharmaceutical&rsquo;s VOXZOGO
Achondroplasia Market Strengths
<ul>
<li>Treatment options for achondroplasia have historically been limited; however, evidence new therapies are emerging.</li>
</ul>
Achondroplasia Market Opportunities
<ul>
<li>Developing affordable yet personalized growth hormone therapies tailored to improve height and overall physical health for individuals with achondroplasia can further boost their confidence.</li>
</ul>
Achondroplasia Emerging Drugs
<ul>
<li>TransCon CNP: Ascendis Pharma&nbsp;</li>
<li>Infigratinib: QED Therapeutics (BridgeBio)/ Novartis</li>
<li>SAR442501: Sanofi</li>
<li>RBM-007: RIBOMIC, and others</li>
</ul>

Achondroplasia Market Infographics

Achondroplasia Infograpahics

Achondroplasia Infographics

Achondroplasia Symptoms, Treatment, epidemiology, companies, drugs, infographic is a short guide to understand the problem and market landscape