Market Overview:
Achondroplasia is the most common cause of dwarfism. It is caused by a mutation or change in the fibroblast growth factor receptor 3 (FGFR3) gene. People with achondroplasia have proportionate short stature with short arms and legs, a large head and mid-face restructure. Market approval for gene and other therapies for achondroplasia aims to treat the underlying molecular defect. This will help reduce complications associated with the condition and improve patients’ quality of life.
Market key trends:
One of the major trends in the achondroplasia treatment market is growing adoption of gene therapies. Voxzogo by BioMarin became the first FDA approved drug to treat achondroplasia in November 2020. Studies evaluating gene therapy administered by intramuscular or intravenous methods are ongoing. If approved, these have potential to provide a permanent cure. Growing research collaborations to develop combinational gene editing approaches is another trend. Researchers are exploring targeted correction of causal mutations using CRISPR/Cas9 and mRNA therapies. Advancing technologies to maximize safety and efficacy will aid researchers. Increasing diagnosis rates supported by newborn screening programs is expanding the potential patient pool for these therapies.
The global Achondroplasia Treatment Market Demand is estimated to be valued at US$ 127.8 Mn in 2023 and is expected to exhibit a CAGR of 36. % over the forecast period 2023 to 2030, as highlighted in a new report published by Coherent Market Insights.
Porter’s Analysis
Threat of new entrants: The Achondroplasia Treatment Market requires high R&D investment and deals with patent protected drugs which makes the threat of new entrants low.
Bargaining power of buyers: The bargaining power of buyers is moderate as the treatment options for achondroplasia are limited.
Bargaining power of suppliers: The suppliers have less bargaining power due to presence of many companies investing in R&D for achondroplasia treatment.
Threat of new substitutes: There is no substitute available for achondroplasia treatment currently.
Competitive rivalry: The Achondroplasia Treatment Market witnesses high competitive rivalry due to presence of global players.
Key Takeaways
The global Achondroplasia Treatment market is expected to witness high growth, exhibiting CAGR of 36.% over the forecast period, due to increasing investments in research and development of novel therapeutics.
Regional analysis
North America is expected to dominate the Achondroplasia Treatment market owing to presence of major players and availability of advanced healthcare facilities in the region. Asia Pacific is anticipated to exhibit the fastest growth during the forecast period due to rising healthcare expenditures and an increasing patient population.
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.
*Note:
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it