Achondroplasia means loss of cartilage formation in individuals, cartilage is a flexible tissue which leads to formation of the skeleton during the early bone development in human beings. Achondroplasia is a condition were cartilage is not converted into bones. Individuals with achondroplasia have short stature. The average height in males is 4 feet 4 inches, and average height in females is 4 feet 1 inches. Achondroplasia is also called short limb dwarfism which can be treated with growth hormone therapy. Other treatments include limb lengthening.

Global Achondroplasia Treatment Market: Drivers

Achondroplasia is a rare disease which can be treated in various ways such as surgical treatment, hormone therapy, ongoing health care and limb lengthening. Therefore, pharmaceutical companies are focusing on product development in treatment of achondroplasia. For instance, in July 2020, BioMarin Pharmaceutical Inc. submitted a marketing authorization application to the European Medical Agency for vosoritide, an injection of c-type natriuretic peptide for treatment of achondroplasia. These factors are expected to drive growth of the global achondroplasia treatment market. Furthermore, pharmaceutical companies are focusing on research and development of treatments for achondroplasia. For instance, in June 2020, Biomarin Pharmaceuticals entered phase II clinical trials of evaluating safety, tolerability and efficacy of BMN III (Vosoritide)  in treating children with achondroplasia.

Global Achondroplasia Treatment Market: COVID-19 Impact Analysis

Since the outbreak of the COVID-19 pandemic, several markets across the globe have been affected negatively. Pharmaceutical companies operating in this market are facing challenges in carrying out clinical trials due to concerns regarding safety and wellbeing of participants. Furthermore, safety precautions which have been imposed to curb spread of the COVID-19 infection have created complications in carrying out surgical treatments and hormone therapy for achondroplasia.

Global Achondroplasia Treatment Market: Restraints

Cost of the growth hormone therapy ranges from US$ 10,000 to US$ 60,000 depending upon the amount of hormone used for the treatment; therefore, growth hormone therapy is expensive for individuals in low- and mid-income countries. Moreover, growth hormone therapy is characterised by side effects such as carpal tunnel syndrome, higher insulin resistance and heightened risk of developing cancer. These factors are expected to restrain global achondroplasia treatment market growth.

Global Achondroplasia Treatment Market Regional Analysis

On the basis of regions, the global achondroplasia treatment market is segmented into North America, Latin America, Europe, Asia Pacific, Middle East, and Africa. According to National Library of Medicine 2018 report, prevalence of achondroplasia in North America is 0.3 to 0.6 out of 10,000 individuals. Moreover, North America has various social associations such as Association of Little People of Alberto (ALPA) and Dwarf Athletic Association of Canada (DAAC) which offer health support and emotional support to patients with achondroplasia. Furthermore, pharmaceutical companies are focusing on strategies such mergers and acquisitions to innovate potential treatments for achondroplasia. For instance, in 2019, Pfizer Inc., a leading pharmaceutical company, announced completion of its acquisition of Therachon Holding AG, a private biotechnology company. Under this acquisition, Pfizer will financially support Therachon for development and commercialization of TA-46, a novel drug for treatment of achondroplasia.

In Europe, prevalence of achondroplasia is significantly more than other regions. According to the European Commission, in 2019, prevalence of achondroplasia was 3.67 per 10,000 births in the region. Therefore, pharmaceutical companies are focusing on research and development of treatments for achondroplasia to strengthen their share in the global anchondroplasia treatment market. For instance, in August 2020, Acendic Pharma A/S, a pharmaceutical company, received an orphan drug designation for Transcon, a c-type natriuretic peptide for treatment of achondroplasia from the European Commission.

Global Achondroplasia Treatment Market Key Players

Key operating players in global achondroplasia treatment market are Ascendis Pharma A/S, BridgeBio Pharma, Inc., QED Therapeutics, BioMarin Pharmaceutical Inc., Pfizer Inc., Therachon Holding AG and RIBOMIC Inc.

Market Taxonomy:

On the basis of drug class, the global achondroplasia treatment market is segmented into:

  • Lipase Inhibitor
  • Appetite Suppresants
  • Human Growth Hormone

On the basis of distribution channel, the global achondroplasia treatment market is segmented into:

  • Hospital Pharmacies
  • Online Pharmacy
  • Retail Pharmacy

On the basis of patient type, the global achondroplasia treatment market is segmented into:

  • Adult
  • Paediatric

On the basis of region, the global achondroplasia treatment market is segmented into:

  • North America
  • Latin America
  • Europe
  • Asia Pacific
  • Middle East
  • Africa
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