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Achondroplasia Market Analysis of Epidemiology, Pipeline Therapies, and Key Companies Working in the market

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Achondroplasia Market Analysis of Epidemiology, Pipeline Therapies, and Key Companies Working in the market

September 26
23:30 2022
Achondroplasia Market Analysis of Epidemiology, Pipeline Therapies, and Key Companies Working in the market
Achondroplasia Market
DelveInsight’s “Achondroplasia Market Insights, Epidemiology, and Market Forecast-2032” report delivers an in-depth understanding of the Achondroplasia, historical and forecasted epidemiology as well as the Achondroplasia market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

DelveInsight’s “Achondroplasia Market Insights, Epidemiology, and Market Forecast-2032” report delivers an in-depth understanding of the Achondroplasia, historical and forecasted epidemiology as well as the Achondroplasia market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

 

The Achondroplasia market report provides current treatment practices, emerging drugs, Achondroplasia market share of the individual therapies, current and forecasted Achondroplasia market Size from 2019 to 2032 segmented by seven major markets. The Report also covers current Achondroplasia treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the Achondroplasia market.

 

Some of the key facts of the Achondroplasia Market Report:

  •  According to population-based research, it was reported that out of the 208 cases of Achondroplasia a total of 166 cases were of De novo Achondroplasia and the remaining were with familial history (Genetically inherited).
  • According to Pauli & Legare, achondroplasia is an autosomal dominant disorder, but about 80% of cases are sporadic, due to a de novo mutation in offspring of unaffected parents.
  •  According to Benjamin et al., achondroplasia is an autosomal dominant disorder; most estimates of its frequency cluster between 1:25,000 and 1:35,000 live births; however, the true frequency may be slightly higher.

 

Key benefits of the Achondroplasia Market report:

 

  • The report covers the descriptive overview of Achondroplasia, explaining its causes, signs and symptoms, pathophysiology, diagnosis and currently available therapies
  • Comprehensive insight has been provided into the Achondroplasia epidemiology and treatment in the 7MM
  • Additionally, an all-inclusive account of both the current and emerging therapies for Achondroplasia is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape
  • A detailed review of the Achondroplasia market; historical and forecasted is included in the report, covering drug outreach in the 7MM

 

 

Got queries? Click here to know more about the Achondroplasia Market Landscape 

Achondroplasia Overview

Achondroplasia is a rare genetic disorder resulting in short-limb skeletal dysplasia occurring in ~1 in 20,000-30,000 live births. This genetic disorder is caused by a mutation in the fibroblast growth factor receptor 3 (FGFR3) gene which occurs as a result of a spontaneous genetic mutation in ~80% of patients and in the remaining 20% is inherited from a parent.

This genetic disorder is characterized by an unusually large head (macrocephaly), short upper arms (rhizomelic dwarfism), and short stature (adult height of ~4 feet). FGFR genetic alterations can cause more than 99% of Achondroplasia cases. In a small proportion of infants, symptoms like excessive accumulation of fluid around the brain (hydrocephalus) and low muscle tone (hypotonia) may happen as typical of achondroplasia. Acquisition of developmental motor milestones may also be delayed.

The current treatment landscape includes medications such as growth hormone therapy, surgery, and supportive therapy for achondroplasia. The first drug to be approved for the treatment of achondroplasia is (Vosoritide) by BioMarin Pharmaceuticals. European Commission granted marketing authorization to VOXZOGO as 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, in August 2021. In November 2021, US FDA approved VOXOGO, under the accelerated approval pathway, and Rare Pediatric Disease Priority Review Voucher, fastening the approval timeline. Marketing authorization reviews are in process in Japan, Brazil, and Australia with potential approvals in these countries in 2022. 

 

Achondroplasia Epidemiological Insights:

 

  • According to the National Organization for Rare Disorders (NORD) the worldwide estimated incidence of achondroplasia ranges from about 1 in 15,000 to 1 in 35,000 births. According to the American Journal of Medical Genetics, achondroplasia has an overall prevalence of 3.72 per 100,000 births and live birth prevalence of 3.05 per 100,000 among 28 European countries.
  • According to the American Journal of Medical Genetics, it was estimated that 20.2% of familial cases and 10% of cases with Achondroplasia had associated major congenital anomalies.
  • According to a European population-based epidemiological study provided by the European Surveillance of Congenital Anomalies (EUROCAT) network research the prevalence of achondroplasia is 3.7 per birth of  100,000 population.
  • According to Wigg et al., the average adult height is 131 ± 5.6 cm for males and 124 ± 5.9 cm for females. Cognitive function is normal unless hydrocephalus or other central nervous system complications occur.
  • According to NORD, Achondroplasia occurs as a result of a spontaneous genetic mutation in ~80% of patients, and in the remaining 20% it is inherited from a parent. 

 

Achondroplasia Epidemiological Segmentation 

  • Achondroplasia total prevalent cases
  • Achondroplasia age-specific cases 

Achondroplasia Market Outlook 

The Achondroplasia market outlook of the report helps to build a detailed comprehension of the historic, current, and forecasted Achondroplasia market trends by analyzing the impact of current Achondroplasia therapies on the market, unmet needs, drivers and barriers, and demand for better technology.

 

This segment gives a thorough detail of Achondroplasia market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria’s, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated Achondroplasia market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

 

According to DelveInsight, the Achondroplasia market in 7MM is expected to witness a major change in the study period 2019-2032.

 

Learn more by requesting for sample @ Achondroplasia Market Landscape

Achondroplasia  Key Companies  

  •  Ascendis Pharma
  •  BridgeBio
  •  QED Therapeutics
  •  Pfizer
  • And many others

Achondroplasia  Therapies 

  • TransCon CNP
  • Secukinumab
  • Recifercept
  • And many others

Table of Contents

  •  Key Insights 
  •  Report Introduction 
  • Executive Summary of Achondroplasia 
  • Disease Background and Overview
  •  Epidemiology and patient population
  •  The United States 
  •  EU 5
  •  Achondroplasia   Emerging Therapies
  •  Achondroplasia  Market Outlook
  •  Market Access and Reimbursement of Therapies
  •  Appendix
  •  Achondroplasia  Report Methodology
  •  DelveInsight Capabilities
  •  Disclaimer
  •  About DelveInsight

Click here to read more about Achondroplasia Market 

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