Achondroplasia is the most common form of short-limbed dwarfism caused by a mutation or defects in fibroblast growth factor receptor 3 (FGFR3) which lead to bone growth failure. Current treatments for achondroplasia focus on treating complications and symptoms. However, recent advancements in understanding the molecular mechanisms and genetic causes of achondroplasia have opened avenues for developing disease-modifying therapies. Gene therapy and advanced drug delivery mechanisms hold promising potential to target the underlying genetic defects and restore normal bone growth in patients.

The global achondroplasia treatment market is estimated to be valued at US$127.8 Mn in 2023 and is expected to exhibit a CAGR of 12% over the forecast period 2023 to 2030, as highlighted in a new report published by Coherent Market Insights.

Market Dynamics:

Rising prevalence of achondroplasia and lack of available cure drives the clinical research and development of novel treatment options. As per the United States based support group for achondroplasia called Little People of America, it is estimated that about 250,000 people worldwide have achondroplasia. Furthermore, recent promising clinical trial results of a gene therapy approach by BioMarin in 2021 has boosted confidence in disease-modifying therapies. This gene therapy works by delivering a normal human gene directly into the body to restore the function of the defective FGFR3 gene. Additionally, a modified mRNA drug called JNJ-57583112 by Janssen Pharmaceutical, is currently under phase 2/3 trials which demonstrated potential to increase annualized height Z-scores in growing children with achondroplasia in phase 1/2 trials. Advancements in vector engineering and targeted delivery mechanisms will aid in developing more efficacious and safer gene therapies with minimized risks. Overall, the treatment landscape is transforming rapidly with advent of new therapeutic modalities.

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 12 % over the forecast period 2023 to 2030, as highlighted in a new report published by Coherent Market Insights.

Segment Analysis

The achondroplasia treatment market can be segmented into drug type and distribution channel. Based on drug type, the market is segmented into somatropin, growth hormone therapy, and others. Among these, somatropin dominates the market as it has been approved for promoting growth in children with achondroplasia. It provides greater increase in growth velocity and height attainment than existing therapies.

PEST Analysis

Political: Regulations regarding drugs and clinical trials for rare disease are supportive which is boosting the market.
Economic: High healthcare expenditure and increasing disposable income are fueling the market growth.
Social: Rising awareness about diseases like achondroplasia and their treatment options is propelling the market.
Technological: Advancements in gene therapy and development of new recombinant human growth hormone are contributing to market growth.

Key Takeaways

The global achondroplasia treatment market is expected to witness high growth, exhibiting 36% CAGR over the forecast period, due to increasing research and development activities.

The North America region dominates the market and is expected to continue its dominance during the forecast period. This is attributed to increasing healthcare expenditure, presence of major players, and growing awareness about achondroplasia treatment in the region.

Regional analysis

The Asia Pacific region is projected to be the fastest growing market for achondroplasia treatment during the analysis period owing to rising healthcare spending, surging disposable income, increasing incidence of achondroplasia, rising medical tourism, and growing focus of international players.

Key players

Key players operating in the achondroplasia treatment market include 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. Major players are focusing on expanding their presence in high growth markets through new product launches, approvals, and strategic collaborations.

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