Achondroplasia is a genetic disorder that affects the fibroblast growth factor receptor protein in the body. This protein begins to function abnormally in achondroplasia, slowing bone development in the growth plate cartilage. This results in shorter bones, abnormally shaped bones, and a shorter stature; adults with achondroplasia range in height from 42 to 56 inches. The genetic defect can be passed from parent to child. However, in approximately 80% of instances, achondroplasia is caused by a spontaneous mutation (a sudden genetic defect) that originates in the developing embryo.
The global achondroplasia market is estimated to be valued at US$ 93.82 million in 2022 and is expected to exhibit a CAGR of 36.0% during the forecast period (2022-2030)
Figure 1.Global Achondroplasia Market Share (%), by Treatment Type, 2022
Increasing incidence of achondroplasia is expected to drive growth of the achondroplasia market.
The increasing incidence of achondroplasia is expected to drive the market growth over the forecast period. For instance, according to the data published by the European Medicines Agency in June 2021, it is estimated that 350 children are born with achondroplasia every year in Europe.
|Base Year:||2021||Market Size in 2022:||US$ 93.82 Mn|
|Historical Data for:||2017 to 2020||Forecast Period:||2022 to 2030|
|Forecast Period 2020 to 2027 CAGR:||36.0%||2030 Value Projection:||US$ 1,100.97 Mn|
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.
|Restraints & Challenges:||
Figure 2.Global Achondroplasia Market Share (%), by Route of Administration, 2022
Increasing drug approvals and launches are expected to drive the market growth during the forecast period.
The increasing approvals and launches of drugs by the key players in the market are expected to drive the market growth. For instance, on January 13, 2022, Ascendis Pharma A/S, a Denmark-based biopharmaceutical company, announced that the European Commission (EC) approved TransCon hGH as a once-weekly subcutaneous injection for the treatment for pediatric patients who are experiencing growth failure due to insufficient endogenous growth hormone secretion or deficiency of growth hormone. TransCon hGH contains Somatropin that delivers unmodified Somatropin (hGH) at therapeutic quantities in the body.
Global Achondroplasia Market– Impact of Coronavirus (COVID-19) Pandemic
Since the COVID-19 virus outbreak in December 2019, the disease has spread to over 100 countries across the globe and the World Health Organization had declared it a public health emergency on January 30, 2020.
COVID-19 can affect the economy in three main ways: by directly affecting production and demand, by creating disruptions in distribution channels, and through its financial impact on firms and financial markets. Due to nationwide lockdowns, several countries, such as China, India, Saudi Arabia, U.A.E., Egypt, and others are facing problems with regards to the transportation of products from one place to another.
Factors such as investments in research and development of novel growth hormones products targeting achondroplasia as well as funding by various companies operating in the research and development sector were delayed during the pandemic, which are expected to hamper the global achondroplasia market growth during the COVID-19 pandemic.
Moreover, the COVID-19 pandemic had affected the ongoing clinical trials for achondroplasia, which is expected to hamper the global achondroplasia market over the forecast period, owing to delayed clinical trials of products. For instance, the annual report of Ascendis Pharma A/S 2019, a biopharmaceutical company, stated that due to the pandemic there was a potential impact on the conduct of clinical trials of investigational drug candidates
Global Achondroplasia Market: Restraint
The major factors that hinder growth of the global achondroplasia market include side effects and high cost of drugs used in the treatment of achondroplasia. For instance, the growth hormone treatment therapy can be expensive, ranging from US$ 10,000 to US$ 60,000 a year for growth hormone treatment alone. Moreover, the list price of VOXZOGO (vosoritide) in France under the ATU (Temporary Authorization for Use) process is 712€ (US$ 837.3 ) per vial.
Major players operating in the global achondroplasia 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.
Achondroplasia is a form of short-limbed dwarfism. Achondroplasia is characterized by an average-sized trunk, short arms and legs, notably short upper arms and thighs, limited range of motion at the elbows, and an enlarged head. Spinal stenosis, a narrowing of the spinal canal that can compress the upper section of the spinal cord, is a potentially serious complication of achondroplasia. Spinal stenosis is characterized by leg pain, tingling, and weakness, which can make walking difficult. Achondroplasia is caused by mutations in the FGFR3 gene. The FGFR3 gene is responsible for giving instructions during making a protein that is involved in the development and maintenance of bone and brain tissue.
The increasing approvals from the regulatory bodies to the key players in the market for the achondroplasia treatment drugs are expected to drive the market growth over the forecast period. For instance, in November 2021, BioMarin, a global biotechnology company, announced that the U.S. Food and Drug Administration (FDA) had granted accelerated approval to VOXZOGO (vosoritide) for injection, indicated to increase linear growth in pediatric patients with achondroplasia of five years of age and older with open epiphyses (growth plates).
Moreover, in August 2021, QED Therapeutics, a subsidiary of BridgeBio Pharma, Inc., announced that the European Commission (EC) had granted marketing authorization for VOXZOGO (vosoritide), a once daily injection to treat achondroplasia in children from the age of 2 until growth plates are closed, which occurs after puberty when children reach final adult height.
Key features of the study:
“*” marked represents similar segmentation in other categories in the respective section.