Achondroplasia treatment market

Achondroplasia Treatment Market, By Treatment Type (Vosoritide, Human Growth Hormone, Others), By Route of Administration (Oral, Parenteral), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies , Online Pharmacies), by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa)

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Jul 2023
CMI4161
207 Pages
Excel & Pdf
Pharmaceutical

Summary

The global 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% during the forecast period (2023-2030).

Analysts’ Views on Global Achondroplasia Treatment Market:

The global market for achondroplasia treatments is anticipated to grow over the forecast period due to increasing awareness of the importance of diagnosis of congenital illnesses. In addition, an before increase in achondroplasia cases prompts key players and research institutions to conduct more clinical trials for the development of novel treatments. The global market growth is anticipated to be driven by an increase in investments in R&D for the management and treatment of achondroplasia during the forecast period. Moreover, the global achondroplasia treatment market is anticipated to grow as a result of the approval of new treatments for this condition.

Figure 1. Global Achondroplasia Treatment Market Share (%),By Treatment Type, 2023

Global Achondroplasia Treatment Market– Driver

Increased Incidence of Achondroplasia: The growing prevalence of achondroplasia is projected to drive the market growth during the forecast period. For instance, as per the data published by the European Medicines Agency in June 2021, it is projected that 350 children are born with achondroplasia each year in Europe. This boosts the market growth.
Increasing Drug Approvals and Launches: The increasing number of drug approvals and launches by major market players is expected to drive the market growth. For instance, In January 2022, Ascendis Pharma A/S manufactures pharmaceutical products. The Company produces products for the treatments of growth hormone deficiency, endocrinology announced ,that the European Commission (EC) had approved TransCon hGH as a once-weekly subcutaneous injection to treat pediatric patients experiencing growth failure due to insufficient endogenous growth hormone secretion or growth hormone deficiency. TransCon hGH contains Somatropin, which delivers unmodified Somatropin (hGH) to the body in therapeutic quantities.

Figure 2. Global Achondroplasia Treatment Market Share(%) by Region, 2023

Global Achondroplasia Treatment Market- Regional Analysis

Among regions, North America is estimated to hold a dominant position in the global achondroplasia treatment market over the forecast period. North America is estimated to hold 34.1% of the market share in 2023. The global achondroplasia treatment market is expected to witness significant growth in the coming years, driven by the high prevalence of achondroplasia, favorable health reimbursement, and increased awareness. North America is expected to emerge as the leading region for the achondroplasia treatment market. For instance, in April 2022, the University of Arkansas sponsored a clinical trial to characterize skeletal muscle amino acid kinetics to an Extrinsic allergic alveolitis EAA challenge, i.e., an oral amino acid tolerance test (OATT), to determine the state of muscle health. For that purpose, trial subjects were given 10 g of a commercially available nutritional supplement for achondroplasia. Hence, an increase in such clinical studies demonstrating the clinical outcome of supplements to treat achondroplasia may result in the launch of new supplements and thus contribute to the growth of the studied market in the region. These kinds of developments in the healthcare system and rising health expenditures, as well as increased knowledge of advanced cancer treatments, are fueling the achondroplasia treatment market growth in North America.

Global Achondroplasia Treatment 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 declared it a public health emergency on January 30, 2020.

COVID-19 affected the economy in three main ways: by directly affecting the production and demand of drugs, 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, the U.A.E., Egypt, and others, were facing problems with the transportation of drugs from one place to another.

However, the COVID-19 pandemic had a positive impact on the global achondroplasia treatment market. The COVID-19 outbreak affected the market's growth adversely in its preliminary phase; however, the number of cancer patients increased in Pandemic, and so has the demand for the achondroplasia market. which is expected to hamper the global achondroplasia treatment market over the forecast period, owing to delayed clinical trials of products. For instance, the annual report of Ascendis Pharma A/S 2020, a biopharmaceutical company, stated that due to the pandemic, there was a potential impact on the conduct of clinical trials of investigational drug candidates.

Achondroplasia Market Report Coverage

Report Coverage	Details
Base Year:	2022	Market Size in 2023:	US$ 127.8 Mn
Historical Data for:	2018 to 2021	Forecast Period:	2023 to 2030
Forecast Period 2023 to 2030 CAGR:	36.5%	2030 Value Projection:	US$ 1,126.0 Mn
Geographies covered:	North America: U.S. and Canada Latin America: Brazil, Argentina, Mexico, and Rest of Latin America Europe: Germany, U.K., Spain, France, Italy, Russia, and Rest of Europe Asia Pacific: China, India, Japan, Australia, South Korea, ASEAN, and Rest of Asia Pacific Middle East: GCC Countries, Israel, and Rest of Middle East Africa: South Africa, North Africa, and Central Africa
Segments covered:	By Treatment Type: Vosoritide, Human Growth Hormone, Others By Route of Administration: Oral, Parenteral By Distribution Channel: Hospital Pharmacies, Retail Pharmacies , Online Pharmacies
Companies covered:	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.
Growth Drivers:	Increasing incidence of achondroplasia Increasing drug approvals and launches Increasing drug approvals and launches
Restraints & Challenges:	High cost of drugs

Global Achondroplasia Treatment Market Segmentation:

The global achondroplasia treatment market report is segmented into Treatment Type, Route of Administration, Distribution Channel, and Region.

By Treatment Type, the market is segmented into vosoritide, human growth hormone, and others. Out of which, the vosoritide segment is expected to hold a dominant position in the global achondroplasia treatment market during the forecast period, and this is attributed to prompt treatment can lead to proper recovery.
By Route of Administration, the market is segmented into oral and parenteral. Out of which, the parenteral segement is expected to dominate the market over the forecast period, and this is attributed to on set of action is more than oral route.
By Distribution Channel, the market is segmented into hospital pharmacies, retail pharmacies, and online pharmacies. Out of which, the hospital pharmacies is expected to dominate the market over the forecast period and this is attributed to prevention, diagnosis, multidisciplinary treatment, supportive care, research, and education.

Among all the segmentations, the treatment type segment is expected to dominate the market over the forecast period, and this is attributed to the increased awareness of achondroplasia is expected to boost the growth of achondroplasia treatment market over the forecast period.

Global Achondroplasia Treatment Market Cross Sectional Analysis:

Various government initiatives support research and development of achondroplasia treatments, further contributing to market growth. For instance, according to the article published in February 2022 in PubMed, the U.S. Department of Health and Human Services was one of the leading funding organizations that sponsored 1,604 sarcopenia-related articles. The U.S. is one of the top countries in the North American region that contributes more toward achondroplasia-related research and development. This increased research is anticipated to lead to various innovations in the country, driving the growth of the achondroplasia treatment market in the country.

Global Achondroplasia Treatment Market: Key Developments

On March 7, 2023, BioMarin Pharmaceutical Inc., a biotechnology company dedicated to transforming lives through genetic discovery, announced that the U.S. Food and Drug Administration (FDA) had accepted the company's supplemental New Drug Application (sNDA) for VOXZOGO (vosoritide) for injection to expand treatment in the U.S. to include children with achondroplasia under the age of 5. Achondroplasia is the most common form of disproportionate short stature.

In August 2020, Ascendis Pharma A/S, a biopharmaceutical company that utilizes its innovative TransCon technologies to address unmet medical needs, announced that the European Commission (EC) had granted Orphan Designation to TransCon C-Type Natriuretic Peptide (CNP) for the treatment of achondroplasia, the most common form of dwarfism. TransCon CNP is an investigational long-acting prodrug of CNP designed to provide the continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose. TransCon CNP also received in 2020, orphan designation for the treatment of achondroplasia in the U.S.

On June 1, 2023, Myriad Genetics, Inc., a company of genetic testing and precision medicine, launched new studies and expansion of its Precise Oncology Solutions portfolio at the 2023 American Society of Clinical Oncology (ASCO).

In January 2020, Wacker Biotech is technological leader in the chemical industry and manufactures products for all key global industries. The company Provides Active Ingredient to treat achondroplasia in children global phase-2 Trial for Ascendis Pharma is a biopharmaceutical company that focuses on developing drugs for endocrinology and oncology indications.

Global Achondroplasia Treatment Market: Key Trends

Rise in Awareness and Increased Research Activities: An increase in awareness about timely diagnosis of congenital disorders is anticipated to boost the global achondroplasia treatment market during the forecast period. An increase in cases of achondroplasia results in a rise in R&D by companies and research institutes in clinical trials to manufacture novel therapeutics. Thus, these factors drive the market growth. For instance, in August 2021, the European Commission granted marketing authorization for VOXZOGO as a once-daily injection for children aged 2 and older, until their growth plates close, which typically occurs after puberty when they reach their final adult height. In November 2021, the U.S. FDA approved VOXZOGO under an accelerated approval pathway and Rare Pediatric Disease Priority Review Voucher, expediting the approval process. Currently, VOXZOGO is undergoing marketing authorization reviews in Japan, Brazil, and Australia, with potential approvals in these countries expected in 2022.

Global Achondroplasia Treatment Market: Restraint

High cost of drugs: The major factors that hinder growth of the global achondroplasia treatment market include side effects and the high cost of drugs used in the treatment of achondroplasia. For instance, 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 US$ 837.3 (712€ ) per vial.

Global Achondroplasia Treatment Market- Key Players

Major players operating in the global 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.

Global Achondroplasia Treatment Market– Definition: 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.

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- Frequently Asked Questions -

What is the current market size and compound annual growth rate (CAGR) of the global achondroplasia treatment market during the forecast period (2023-2030)?

The global 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% between 2023 and 2030.

What are the major factors driving the market growth?

Increasing incidence of achondroplasia and Increasing drug approvals and launches are expected to drive the market growth.

Which is the leading by treatment type segment in the market?

Vosoritide is the leading segment in the market.

What are the key factors hampering the growth of the market?

High cost of drugs is expected to hinder the market over the forecast period.

Which are the major players operating in the market?

Major players 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.