Global Cystic Fibrosis (CF) Market Study 2017-2030 with Analysis of Emerging Drugs MRT5005 (Translate Bio) and VX-121 (Vertex Pharmaceuticals) - ResearchAndMarkets.com

The "Cystic Fibrosis (CF) - Market Insights, Epidemiology, and Market Forecast - 2030" report has been added to ResearchAndMarkets.com's offering.

The report delivers an in-depth understanding of the Cystic Fibrosis (CF), historical and forecasted epidemiology as well as the market trends in the United States, and EU5 (Germany, Spain, Italy, France, and United Kingdom).

The report provides current treatment practices, emerging drugs and their market share of the individual therapies, current and forecasted Cystic Fibrosis (CF) symptoms market size from 2017 to 2030 segmented by six major markets. The report also covers current Cystic Fibrosis (CF) symptoms 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 market.

Epidemiology

The Cystic Fibrosis (CF) symptoms epidemiology division provides insights about the historical and current patient pool along with the forecasted trend for every six 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 report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

Key Findings

The diagnosed prevalent cases of Cystic Fibrosis (CF) is increasing in 6MM during the study period, i.e. 2017-2030.

The disease epidemiology covered in the report provides historical as well as forecasted Cystic Fibrosis (CF) symptoms epidemiology segmented as the Total Prevalent cases of Cystic Fibrosis (CF), Gender-specific cases of Cystic Fibrosis (CF), Age-specific cases of Cystic Fibrosis (CF), Type-specific cases of Cystic Fibrosis (CF). The report includes the prevalent scenario of Cystic Fibrosis (CF) symptoms in 6MM covering the United States, and EU5 countries (Germany, France, Italy, Spain, and the United Kingdom) from 2017 to 2030.

The total prevalent cases of Cystic Fibrosis (CF) associated in 6MM countries was 61,306 in 2017.

Drug Chapters

The drug chapter segment of the Cystic Fibrosis (CF) report encloses the detailed analysis of Cystic Fibrosis (CF) marketed drugs and early-stage (Phase-I, II, III) pipeline drugs. It also helps to understand the Cystic Fibrosis (CF) 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.

Emerging Drugs

MRT5005: Translate Bio Inc.

MRT5005, a lead program for the lung, is the first clinical-stage mRNA product candidate designed to address the underlying cause of CF by delivering mRNA encoding fully functional CFTR protein to the lung epithelial cells through nebulization. MRT5005 is being developed to treat all patients with CF, regardless of the underlying genetic mutation, including those with limited or no CFTR protein.

VX-121: Vertex Pharmaceuticals

VX-121 is an orally administered CFTR corrector that helps fix and restore the function of defective CFTR protein. The corrected CFTR protein can then translocate to the cell surface where it performs the normal function and helps maintain the right balance of fluid in the airways. Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) is a transmembrane protein in epithelial cells that plays a crucial role in ion transport across the cell, especially the transport of chloride and sodium ions.

Market Outlook

The Cystic Fibrosis (CF) market outlook of the report helps to build the detailed comprehension of the historic, current and forecasted Cystic Fibrosis (CF) market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology.

This segment gives a thorough detail of Cystic Fibrosis (CF) market trend of each marketed drug and early-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 market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

Cloning of CF Transmembrane conductance Regulator (CFTR) gene has provided a breakthrough in the understanding of the molecular basis of this disease. There are over 1,800 mutations of CFTR identified thus far, wherein 23 variants account for the majority of CF-causing mutations. The CF-causing mutations of CFTR can be grouped into classes depending on the physiological effect. Some mutations cause little or no protein to be made (Class I and IV), others create a defective protein that does not make it to the cell membrane (Class II), and others cannot be effectively regulated or conduct chloride (Class III and IV).

Classification of the mutations allows the development of specialized drugs to overcome the specific gene mutation. The most common CF mutation, F508del, is primarily considered to be a processing mutation. The F508del mutation removes a single amino acid from the CFTR protein. Without this building block, the CFTR protein cannot stay in the correct 3-D shape. The cell recognizes that the protein is not the right shape and disposes of it.

Approximately 44% of CF patients are homozygous for the F508del mutation (i.e., F508del/F508del), while around 41% are compound heterozygotes (i.e., F508del/other CF causing CFTR mutation), and about 15% have two non-F508del CF-causing CFTR mutations. Patients who are homozygous for F508del are said to have classic CF. Patients with classic CF tend to be pancreatic insufficient and have more severe lung disease than CF patients with other mutations.

Key Findings

This section includes a glimpse of the Cystic Fibrosis (CF) market in 6MM. The market size of Cystic Fibrosis (CF) in the six major markets was USD 1,940.1 million in 2017.

Drugs Uptake

This section focuses on the rate of uptake of the potential drugs recently launched or expected to get launched in the market during the study period 2017-2030. The analysis covers Cystic Fibrosis (CF) market uptake by drugs; patient uptake by therapies; and sales of each drug.

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

Development Activities

The report provides insights into the therapeutic candidate in Phase I, II and III stage. It also analyses Cystic Fibrosis (CF) key players involved in developing targeted therapeutics. Major players include Translate Bio, Inc., Proteostasis Therapeutics, Vertex Pharmaceuticals, and others.

Pipeline Activities

The report covers the detailed information of collaborations, acquisition, and merger, licensing and patent details for Cystic Fibrosis (CF) emerging therapies.

Reimbursement Scenario

Approaching reimbursement, proactively, can have a positive impact both during the early stages of product development and well after product launch. In the report, we consider reimbursement 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.

Lumacaftor/ivacaftor was approved by the Food and Drug Administration (FDA) as a combination treatment for Cystic Fibrosis (CF) patients who are homozygous for the F508del mutation. Under the base-case, Lumacaftor/ivacaftor resulted in higher QALYs (7.29 vs. 6.84) but at a very high cost (USD 1,778,920.88) compared to usual care (USD 116,155.76) over 10 years. The ICER for base-case and worst-case scenarios were USD 3,655,352/QALY, and USD 8,480,265/QALY gained, respectively.

In the base-case, lumacaftor/ivacaftor was cost-effective at a threshold of USD 150,000/QALY-gained when annual drug costs were lower than USD 4,153; the results were not substantially affected by the sensitivity analyses. The intervention produced substantial QALY gains but at an extremely high cost, resulting in an ICER that would not typically be covered by any insurer. Hence, Lumacaftor/ivacaftor's status as an orphan drug complicates coverage decisions.

Although CF is uncommon, it represents a significant economic burden. In 2013, CF-related hospital costs alone were estimated to exceed USD 1.1 billion. Institute for Clinical and Economic Review (ICER) reviewed the Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) Database for US commercial health plan coverage policies for Trikafta, Kalydeco, Orkambi, Symdeko, developed by the Center for Evaluation of Value and Risk in Health.

KOL Views

To keep up with current market trends, we take KOLs and SME's opinion working in Cystic Fibrosis (CF) 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 Cystic Fibrosis (CF) market trend. 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

The publisher performs Competitive and Market Intelligence analysis of the Cystic Fibrosis (CF) Market by using various Competitive Intelligence tools that includes - 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 Report

• The report covers the descriptive overview of Cystic Fibrosis (CF), explaining its causes, signs and symptoms, physiology and currently available therapies
• Comprehensive insight has been provided into the Cystic Fibrosis (CF) epidemiology and treatment in the 6MM
• Additionally, an all-inclusive account of both the current and emerging therapies for Cystic Fibrosis (CF) is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape
• A detailed review of Cystic Fibrosis (CF) market; historical and forecasted is included in the report, covering drug outreach in the 6MM
• The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Cystic Fibrosis (CF) market

Report Highlights

• In the coming years, Cystic Fibrosis (CF) market is set to change due to the rising awareness of the disease, and Favorable Environment for New Anti-infective Modalities; 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 Cystic Fibrosis (CF) 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 Cystic Fibrosis (CF). Launch of emerging therapies will significantly impact the Cystic Fibrosis (CF) market
• Our in-depth analysis of the pipeline assets across different stages of development (Phase I, II, III), 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

Companies Mentioned

• Vertex Pharmaceuticals
• Translate Bio Inc.
• Proteostasis Therapeutics
• Arrowhead Pharmaceuticals
• ProQR Therapeutics
• Ionis Pharmaceuticals
• Verona Pharmaceuticals
• Eloxx Pharmaceuticals, Inc.

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