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

Achondroplasia Market analysis of the epidemiology, pipeline therapies, and major companies operating in the market

“Achondroplasia Market”

DelveInsight’s Achondroplasia Market Insights, Epidemiology, and Market Forecast-2032 report provides an in-depth understanding of Achondroplasia, historical and forecast epidemiology, and Achondroplasia market trends in United States, EU5 (Germany, Spain, Italy, France). , and United Kingdom) and Japan.

DelveInsights “Achondroplasia Market Insights, Epidemiology and Market Forecast 2032The report provides an in-depth understanding of Achondroplasia, historical and projected epidemiology, and Achondroplasia market trends in United States, EU5 (Germany, Spain, Italy, France and UK) and Japan.

The Achondroplasia Market Report includes Current Treatment Practices, New Drugs, Achondroplasia Market Share of each Therapy, Current and Forecast Achondroplasia Market Size from 2019 to 2032, segmented by seven major markets. The report also covers current Achondroplasia treatment practices/algorithms, market drivers, market barriers, and unmet medical needs to curate the best opportunities and evaluates 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 de novo achondroplasia and the remaining cases had a family history (genetically inherited).
  • According to Pauli & Legare, achondroplasia is an autosomal dominant disease, but about 80% of cases are sporadic, due to de novo mutation in the offspring of unaffected parents.
  • According to Benjamin et al. is achondroplasia an autosomal dominant disease; 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 Achondroplasia Market Report:

  • The report includes a descriptive overview of achondroplasia, explaining its causes, signs and symptoms, pathophysiology, diagnosis, and currently available therapies
  • A comprehensive insight into achondroplasia epidemiology and treatment was provided at the 7MM
  • In addition, a comprehensive report on current and emerging therapies for achondroplasia is provided, along with the assessment of emerging therapies that will impact the current treatment landscape
  • A Detailed Review of the Achondroplasia Market; historical and projected data are included in the report covering drug use in the 7MM
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Overview of achondroplasia

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

This genetic disorder is characterized by an abnormally large head (macrocephaly), short upper arms (rhizomelic dwarfism), and short stature (adult height of about 1.20 m). FGFR gene changes can cause more than 99% of achondroplasia cases. A small proportion of infants may experience symptoms such as excess fluid around the brain (hydrocephalus) and low muscle tone (hypotension) that are typical of achondroplasia. Acquisition of motor developmental milestones may also be delayed.

The current treatment landscape includes drugs such as growth hormone therapy, surgery, and supportive therapy for achondroplasia. The first drug approved for the treatment of achondroplasia is (Vosoritide) from BioMarin Pharmaceuticals. The European Commission granted marketing authorization for VOXZOGO in August 2021 as a once-daily injection for the treatment of achondroplasia in children from the age of 2 years until the growth plates close, which occurs after puberty when children reach final adult height. In November 2021, US FDA approved VOXOGO under the Accelerated Review and Priority Review Voucher in Rare Pediatric Conditions, establishing the regulatory timeline. Marketing authorization reviews are underway in Japan, Brazil and Australia, with possible approvals in these countries in 2022.

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Achondroplasia Epidemiological Findings:

  • According to the National Organization for Rare Disorders (NORD), the estimated incidence of achondroplasia worldwide is between approximately 1 in 15,000 and 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 a live birth prevalence of 3.05 per 100,000 in 28 European countries.
  • According to the American Journal of Medical Genetics, an estimated 20.2% of familial cases and 10% of cases with achondroplasia had major congenital abnormalities.
  • According to a European population-based epidemiological study provided by the European Surveillance of Congenital Anomalies (EUROCAT) research network, the prevalence of achondroplasia is 3.7 per birth per 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 in the absence of hydrocephalus or other central nervous system complications.
  • According to NORD, achondroplasia occurs in ~80% of patients as a result of spontaneous genetic mutation, and in the remaining 20% ​​it is inherited from a parent.

Achondroplasia Epidemiological segmentation

  • Achondroplasia overall widespread cases
  • Achondroplasia age-specific cases

Achondroplasia Market Outlook

The report’s Achondroplasia Market Outlook helps in building a detailed understanding of the historical, current, and forecast Achondroplasia Market trends by analyzing the impact of current Achondroplasia Therapies on the market, unmet needs, drivers, and barriers, and demands for better technology.

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This segment provides a detailed overview of the Achondroplasia market trend for each marketed drug and late-stage pipeline therapy, by analyzing their impact based on annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, and growing need of the market are evaluated. growing patient pool, patient segment covered, expected launch year, competition from other therapies, brand equity, its impact on the market and key opinion leaders assessment. The calculated Achondroplasia market data is presented with relevant tables and graphs to present a clear overview of the market at first glance.

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

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Key companies for achondroplasia

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

Achondroplasia Therapies

  • TransCon CNP
  • secukinumab
  • Recifercept
  • And many others

Table of Contents

  • Important Findings
  • Report introduction
  • Summary of Achondroplasia
  • Disease background and overview
  • Epidemiology and patient population
  • The United States
  • EU5
  • New therapies for achondroplasia
  • Achondroplasia Market Outlook
  • Market access and reimbursement of therapies
  • Achondroplasia Report Methodology
  • DelveInsight features
  • Disclaimer
  • About DelveInsight

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