Achondroplasia Market

• According to DelveInsight's’ estimates, Achondroplasia market in 7MM is expected to show a positive growth, during the forecast period (2023–2032), mainly attributed to new product launches, a decent pipeline of emerging therapies, as well as the ongoing clinical development.
• BridgeBio, QED Therapeutics, Ribomic Inc, Sanofi, and others are some of the companies working in the Achondroplasia market.
• Infigratinib, RBM-007, SAR-442501, and others are some of the drugs in the Achondroplasia treatment market.

Request for Sample Report @ 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.

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.

Geography Covered

• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan

Study Period: 2019-2032

Achondroplasia Disease Understanding and Treatment Algorithm

The DelveInsight’s Achondroplasia market report gives a thorough understanding of the Achondroplasia by including details such as disease definition, symptoms, causes, pathophysiology, diagnosis, and treatment.

Achondroplasia is a rare genetic disorder that causes a specific type of skeletal dysplasia characterized by short limbs. It is caused by a mutation in the fibroblast growth factor receptor 3 (FGFR3) gene. In about 80% of cases, this mutation occurs spontaneously, while in the remaining 20%, it is inherited from a parent.

The main features of this genetic disorder include a disproportionately large head (macrocephaly), short upper arms (rhizomelic dwarfism), and short stature, with adult height typically reaching around 4 feet. FGFR genetic alterations are responsible for more than 99% of all Achondroplasia cases. Some infants may also experience symptoms such as excessive fluid buildup in the brain (hydrocephalus) and low muscle tone (hypotonia), which are common in Achondroplasia. Additionally, children with this condition may experience delays in achieving motor milestones during development.

The current treatment options for Achondroplasia include medications like growth hormone therapy, surgical interventions, and supportive therapy. The first drug approved specifically for treating Achondroplasia is called VOXZOGO (Vosoritide), developed by BioMarin Pharmaceuticals. 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 US 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.

Achondroplasia Diagnosis

This segment of the report covers the detailed diagnostic methods or tests for Achondroplasia.

Achondroplasia Treatment

It covers the details of conventional and current medical therapies available in the Achondroplasia market for the treatment of the condition. It also provides Achondroplasia treatment algorithms and guidelines in the United States, Europe, and Japan.

Achondroplasia Epidemiology

The Achondroplasia epidemiology section provides insights about the historical and current Achondroplasia patient pool and forecasted trends for individual seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the Achondroplasia market report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

Key Findings

The disease epidemiology covered in the report provides historical as well as forecasted Achondroplasia epidemiology scenario in the 7MM covering the United States, EU5 countries (Germany, Spain, Italy, France, and the United Kingdom), and Japan from 2019 to 2032.

Country Wise- Achondroplasia Epidemiology

The epidemiology segment also provides the Achondroplasia epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

Achondroplasia Drug Chapters

The drug chapter segment of the Achondroplasia report encloses the detailed analysis of Achondroplasia marketed drugs and late-stage (Phase-III and Phase-II) Achondroplasia pipeline drugs. It also helps to understand the Achondroplasia clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

Achondroplasia Marketed Drugs

The report provides the details of the marketed products/off-label treatments available for Achondroplasia treatment.

Achondroplasia Emerging Drugs

The report provides the details of the emerging therapies under the late and mid-stage of development for Achondroplasia treatment.

Infigratinib: QED Therapeutics/ BridgeBio Pharma

Infigratinib is an orally administered, ATP-competitive, tyrosine kinase inhibitor that is designed to inhibit FGFR, and being investigated for treatment of individuals with FGFR-driven conditions. Recently, positive results from PROPEL2, a Phase II trial of the investigational therapy Infigratinib in children with achondroplasia, demonstrating potential best-in-class efficacy and clean safety profile. Infigratinib is an oral small molecule designed to inhibit FGFR3 and target achondroplasia at its source. Under the terms of the Agreement, BridgeBio will retain all rights to Infigratinib in skeletal dysplasia, including achondroplasia. Infigratinib is not currently approved for the treatment of achondroplasia by the US FDA or any other health authority.

RBM-007: RIBOMIC Inc.

RBM-007 is a novel nucleic acid medicine (oligonucleotide-based aptamer) developed in-house at RIBOMIC’s research facilities in Tokyo. RBM-007 has been shown to have potent effects in limiting excessive interactions between fibroblast growth factors, which are known to cause achondroplasia. Along with these potential candidates some other early stage developmental products like SAR-442501 (Sanofi) and others are expected to eventually add in the potential treatment league overall strengthening the treatment landscape of achondroplasia.

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.

Key Findings

This section includes a glimpse of the Achondroplasia market in 7MM.

The United States: Achondroplasia Market Outlook

This section provides the total Achondroplasia market size and market size by therapies in the United States.

EU-5 Countries: Achondroplasia Market Outlook

The total Achondroplasia market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom is provided in this section.

Japan: Achondroplasia Market Outlook

The total Achondroplasia market size and market size by therapies in Japan is also mentioned.

Achondroplasia Drugs Uptake

This section focuses on the rate of uptake of the potential Achondroplasia drugs recently launched in the Achondroplasia market or expected to get launched in the market during the study period 2019-2032. The analysis covers Achondroplasia market uptake by drugs; patient uptake by therapies; and sales of each drug.

Achondroplasia Drugs Uptake helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs, and allow the comparison of the drugs on the basis of Achondroplasia market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

Achondroplasia Pipeline Development Activities

The Achondroplasia report provides insights into different therapeutic candidates in Phase II, and Phase III stage. It also analyses Achondroplasia key players involved in developing targeted therapeutics.

Pipeline Development Activities

The Achondroplasia report covers the detailed information of collaborations, acquisition, and merger, licensing, patent details, and other information for Achondroplasia emerging therapies.

Reimbursement Scenario in Achondroplasia

Approaching reimbursement proactively can have a positive impact both during the late stages of product development and well after product launch. In a report, we take reimbursement into consideration to identify economically attractive indications and market opportunities. When working with finite resources, the ability to select the markets with the fewest reimbursement barriers can be a critical business and price strategy.

KOL- Views

To keep up with current Achondroplasia market trends, we take KOLs and SMEs ' opinion working in the Achondroplasia domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or Achondroplasia market trends. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs.

Competitive Intelligence Analysis

We perform Competitive and Market Intelligence analysis of the Achondroplasia Market by using various Competitive Intelligence tools that include - SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies etc. The inclusion of the analysis entirely depends upon the data availability.

Scope 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
• The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Achondroplasia market

Achondroplasia Market Report Highlights

• In the coming years, the Achondroplasia market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market
• The companies and academics are working to assess challenges and seek opportunities that could influence Achondroplasia R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition
• Major players are involved in developing therapies for Achondroplasia. The launch of emerging therapies will significantly impact the Achondroplasia market
• A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for Achondroplasia
• Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities

Achondroplasia Report Insights

• Achondroplasia Patient Population
• Achondroplasia Therapeutic Approaches
• Achondroplasia Pipeline Analysis
• Achondroplasia Market Size and Trends
• Achondroplasia Market Opportunities
• Impact of upcoming Achondroplasia Therapies

Achondroplasia Report Key Strengths

• 11 Years Forecast
• 7MM Coverage
• Achondroplasia Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Achondroplasia Market
• Achondroplasia Drugs Uptake

Achondroplasia Market Report Assessment

• Current Treatment Practices
• Achondroplasia Unmet Needs
• Achondroplasia Pipeline Product Profiles
• Achondroplasia Market Attractiveness
• Achondroplasia Market Drivers and Barriers

Key Questions

Achondroplasia Market Insights:

• What was the Achondroplasia drug class share (%) distribution in 2019 and how it would look like in 2032?
• What would be the Achondroplasia total market size as well as market size by therapies across the 7MM during the forecast period (2019-2032)?
• What are the key findings pertaining to the market across 7MM and which country will have the largest Achondroplasia market size during the forecast period (2019-2032)?
• At what CAGR, the Achondroplasia market is expected to grow by 7MM during the forecast period (2019-2032)?
• What would be the Achondroplasia market outlook across the 7MM during the forecast period (2019-2032)?
• What would be the Achondroplasia market growth till 2032, and what will be the resultant market Size in the year 2032?
• How would the unmet needs affect the Achondroplasia market dynamics and subsequent analysis of the associated trends?

Achondroplasia Epidemiology Insights:

• What are the disease risk, burden, and regional/ethnic differences of the Achondroplasia?
• What are the key factors driving the Achondroplasia epidemiology trend for seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
• What is the historical Achondroplasia patient pool in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
• What would be the forecasted patient pool of Achondroplasia in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
• Where will be the growth opportunities in the 7MM with respect to the patient population pertaining to Achondroplasia?
• Out of all 7MM countries, which country would have the highest prevalent population of Achondroplasia during the forecast period (2019-2032)?
• At what CAGR the patient population is expected to grow in 7MM during the forecast period (2019-2032)?

Current Treatment Scenario, Marketed Drugs and Emerging Therapies:

• What are the current options for the Achondroplasia treatment in addition to the approved therapies?
• What are the current treatment guidelines for the treatment of Achondroplasia in the USA, Europe, and Japan?
• What are the Achondroplasia marketed drugs and their respective MOA, regulatory milestones, product development activities, advantages, disadvantages, safety and efficacy, etc.?
• How many companies are developing therapies for the treatment of Achondroplasia?
• How many therapies are in-development by each company for Achondroplasia treatment?
• How many are emerging therapies in mid-stage, and late stage of development for Achondroplasia treatment?
• What are the key collaborations (Industry - Industry, Industry - Academia), Mergers and acquisitions, licensing activities related to the Achondroplasia therapies?
• What are the recent novel therapies, targets, mechanisms of action and technologies being developed to overcome the limitation of existing therapies?
• What are the ongoing Achondroplasia clinical studies and their status?
• What are the current challenges faced in Achondroplasia drug development?
• What are the key designations that have been granted for the emerging therapies for Achondroplasia?
• What are the global historical and forecasted Achondroplasia market trends?

Reasons to buy

• The report will help in developing business strategies by understanding trends shaping and driving the Achondroplasia market
• To understand the future market competition in the Achondroplasia market and Insightful review of the key market drivers and barriers
• Organize sales and marketing efforts by identifying the best opportunities for Achondroplasia in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom) and Japan
• Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors
• Organize sales and marketing efforts by identifying the best opportunities for Achondroplasia market
• To understand the future market competition in the Achondroplasia market