The Achondroplasia Treatment Market is estimated to be valued at US$ 127.8 million in 2023 and is expected to exhibit a CAGR of 36.5% over the forecast period 2023-2030, as highlighted in a new report published by Coherent Market Insights.

Market Overview:

Achondroplasia is the most common form of disproportionate short stature in humans caused by mutations in the fibroblast growth factor receptor 3 gene. Currently available treatment options focus on managing complications and improving quality of life.

Market Dynamics:
The market is primarily driven by rising prevalence of achondroplasia globally and robust pipeline candidates under clinical trials. As per recent estimates, achondroplasia occurs in approximately 1 in 25,000 live births worldwide. Several companies are developing novel therapeutics such as vosoritide by BioMarin and elafofloxacin by Ascendis Pharma A/S that directly target the underlying cause of disease by stimulating bone growth. Vosoritide received regulatory approval in the European Union in 2021 and if approved, similar products will significantly boost market revenues during the forecast period.

Market key trends:
The Achondroplasia Treatment market has been witnessing rising demand for targeted therapies to address causes at the genetic level. One key trend gaining traction is the focus on developing recombinant human growth hormone (rhGH) therapeutics. rhGH treatments help stimulate bone growth and increase height in children with dwarfism caused by achondroplasia. Companies are conducting clinical trials for potential rhGH therapies to treat symptoms and halt or slow the progression of the condition. Additionally, recent years have seen a surge in research exploring gene therapy techniques using viral vectors to permanently alter the functioning of the FGFR3 gene which is responsible for achondroplasia. With success, such therapies can help reduce the dependence on regular medication intake.

SWOT Analysis
Strength:
- Growing research & development investment by companies in gene therapy and targeted treatment options.
- Rising healthcare expenditure and demand for advanced treatment options.
Weakness:
- High costs associated with developing and commercializing gene therapies.
- Clinical efficacy and safety challenges associated with new treatment modalities.
Opportunity:
- Large underserved patient population indicative of market potential.
- Collaborations between pharma companies and research institutes to fast track drug development.
Threats:
- Stringent regulatory norms for approval of new drug candidates.
- Low treatment adherence for symptomatic therapies requiring regular medical supervision.

Key Takeaways
The Global Achondroplasia Treatment Market is expected to witness high growth, exhibiting CAGR of 36.5% over the forecast period 2023-2030, due to increasing demand for curative therapies. The market size is estimated to reach US$ 127.8 million in 2023.

Regional analysis:
North America is expected to dominate the global market over the forecast period. This is attributed to the growing patient population in the US, high healthcare expenditure, and presence of major market players in the region conducting clinical trials. Asia Pacific is likely to exhibit the highest growth rate owing to rising healthcare infrastructure, increasing diagnosis rates, and growing research collaborations.

Key players:
Key players operating in the Achondroplasia Treatment market are 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. Companies are involved in developing novel therapeutics such as gene therapies and recombinant protein.

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