Krabbe Disease Market

• The Krabbe Disease Market Size is anticipated to grow with a significant CAGR during the study period (2020-2034).
• In March 2024, Forge Biologics announced that the Medicines and Healthcare Products Regulatory Agency (MHRA), the healthcare regulatory body of the United Kingdom (UK), granted Innovation Passport designation to the Company’s novel AAV gene therapy program, FBX-101, to enter the Innovative Licensing and Access Pathway (ILAP).
• Key Krabbe disease Companies working on treatments and research include Novartis AG, Pfizer, Sanofi-Aventis SA, Shire, GlaxoSmithKline, Johnson & Johnson, Abbott Laboratories, CENETOGENE N.V., Teva Pharmaceutical Industries Ltd., UCB Pharmaceuticals, and others.

DelveInsight's "Krabbe Disease Market Insights, Epidemiology, and Market Forecast-2034" report delivers an in-depth understanding of the Krabbe Disease, historical and forecasted epidemiology as well as the Krabbe Disease therapeutics market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

The Krabbe Disease market report provides current treatment practices, emerging drugs, Krabbe Disease market share of the individual therapies, current and forecasted Krabbe Disease market Size from 2020 to 2034 segmented by seven major markets. The Report also covers current Krabbe Disease 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 Krabbe Disease market.

Krabbe Disease Treatment Market

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

Krabbe Disease Diagnosis

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

Krabbe Disease Treatment

It covers the details of conventional and current medical therapies available in the Krabbe Disease market for the treatment of the condition. It also provides Krabbe Disease treatment algorithms and guidelines in the United States, Europe, and Japan. Krabbe disease clinical trials aim to evaluate emerging gene therapies and enzyme replacement strategies, offering hope for improving outcomes in this rare, often fatal neurodegenerative disorder affecting infants and children.

Krabbe Disease Epidemiology 

The Krabbe Disease epidemiology section provides insights about the historical and current Krabbe Disease 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 Krabbe Disease market report also provides the diagnosed patient pool and their trends along with assumptions undertaken. 

Key Findings

The Krabbe Disease epidemiology covered in the report provides historical as well as forecasted Krabbe Disease epidemiology scenario in the 7MM covering the United States, EU5 countries (Germany, Spain, Italy, France, and the United Kingdom), and Japan from 2020 to 2034.

• As per NORD (2024), an estimated 1 in 100,000 newborn babies have Krabbe disease. The more severe infantile-onset disease occurs in 85–90% of patients. The other 10–15% of patients are diagnosed with late-onset disease, though this number may now be higher based on data from state newborn screening programs.
• By determining numbers of Krabbe patients represented in the KID and PHIS databases, Ghabash et al. (2021), estimated a birth incidence of 1 in 310,000 live births in the United States.
• As per Metabolic Support UK, this disorder is estimated to affect approximately 1/100,000 in the Northern European population.
• Based on an estimated Krabbe incidence of 1 in 310,679 births, it is projected that each year in the United States, approximately 12 births will involve children at risk for symptomatic Krabbe disease.

Country Wise- Krabbe Disease Epidemiology

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

Krabbe Disease Drug Chapters

The drug chapter segment of the Krabbe Disease report encloses the detailed analysis of Krabbe Disease marketed drugs and late-stage (Phase-III and Phase-II) Krabbe Disease pipeline drugs. It also helps to understand the Krabbe Disease 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. The Krabbe Disease drugs market is driven by increasing awareness, improved diagnostic techniques, and ongoing research into gene therapies, with promising developments offering hope for this ultra-rare neurological disorder.

Krabbe Disease Marketed Drugs

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

Krabbe Disease Emerging Drugs

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

FBX-101 : Forge Biologics

FBX-101 was developed to treat children with Krabbe disease. FBX-101 is an adeno-associated viral serotype rh10 (AAVrh10) gene therapy that is delivered intravenously after HSCT infusion. The vector delivers a functional copy of the GALC gene to cells in both the central and peripheral nervous system. FBX-101 has been shown to functionally correct the central and peripheral neuropathy associated with Krabbe, improve myelination and gross motor function, and significantly prolong lifespan in animal models. This is a novel approach that has the potential to overcome some of the immunological safety challenges observed in traditional AAV gene therapies (without transplantation). HSCT has normal GALC and brings an immune system that does not recognize GALC as an antigen.

PLX-300: Polaryx Therapeutics

PLX-300 is a new drug isolated from cinnamon. It is a PPARα agonist. PLX-300 and its bioactive metabolites are also abundantly present in the human diet, including vegetables, fruits, honey, and whole grains.

Krabbe Disease Market Outlook

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

This segment gives a thorough detail of Krabbe Disease 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 Krabbe Disease market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

According to DelveInsight, the Krabbe Disease market in 7MM is expected to witness a major change in the study period 2020-2034.

Key Findings

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

The United States Market Outlook

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

EU-5 Countries: Market Outlook

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

Japan Market Outlook

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

Krabbe Disease Drugs Uptake

This section focuses on the rate of uptake of the potential Krabbe Disease drugs recently launched in the Krabbe Disease market or expected to get launched in the market during the study period 2020-2034. The analysis covers Krabbe Disease market uptake by drugs; patient uptake by therapies; and sales of each drug.   

Krabbe Disease 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 Krabbe Disease market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

Krabbe Disease Pipeline Development Activities

The Krabbe Disease report provides insights into different Krabbe Disease clinical trials Phase II, and Phase III stage. It also analyses Krabbe Disease key players involved in developing targeted therapeutics.

Pipeline Development Activities

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

Reimbursement Scenario in Krabbe Disease

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 Krabbe Disease market trends, we take KOLs and SMEs ' opinion working in the Krabbe Disease 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 Krabbe Disease 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 Krabbe Disease 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 Krabbe Disease Market Report

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

Krabbe Disease Market Report Highlights

• In the coming years, the Krabbe Disease 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 Krabbe Disease 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 Krabbe Disease. The launch of emerging therapies will significantly impact the Krabbe Disease market
• A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for Krabbe Disease
• 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

Krabbe Disease Market Report Insights

• Krabbe Disease Patient Population
• Krabbe Disease Therapeutic Approaches
• Krabbe Disease Pipeline Analysis
• Krabbe Disease Market Size and Trends
• Krabbe Disease Market Opportunities
• Impact of Upcoming Krabbe Disease Therapies

Krabbe Disease Market Report Key Strengths

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

Krabbe Disease Market Report Assessment

• Current Krabbe Disease Treatment Practices
• Krabbe Disease Unmet Needs
• Krabbe Disease Pipeline Product Profiles
• Krabbe Disease Market Attractiveness
• Krabbe Disease Market Drivers
• Krabbe Disease Market Barriers

Key Questions Answered In The Krabbe Disease Market Report:

Krabbe Disease Market Insights:

• What was the Krabbe Disease drug class share (%) distribution in 2020 and how it would look like in 2034?
• What would be the Krabbe Disease total market size as well as market size by therapies across the 7MM during the forecast period (2020-2034)?
• What are the key findings pertaining to the market across 7MM and which country will have the largest Krabbe Disease market size during the forecast period (2020-2034)?
• At what CAGR, the Krabbe Disease market is expected to grow by 7MM during the forecast period (2020-2034)?
• What would be the Krabbe Disease market outlook across the 7MM during the forecast period (2020-2034)?
• What would be the Krabbe Disease market growth till 2034, and what will be the resultant market Size in the year 2034?
• How would the unmet needs affect the market dynamics and subsequent analysis of the associated trends?

Krabbe Disease Epidemiology Insights:

• What are the disease risk, burden, and regional/ethnic differences of the Krabbe Disease?
• What are the key factors driving the epidemiology trend for seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
• What is the historical Krabbe Disease 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 Krabbe Disease 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 Krabbe Disease?
• Out of all 7MM countries, which country would have the highest prevalent population of Krabbe Disease during the forecast period (2020-2034)?
• At what CAGR the patient population is expected to grow in 7MM during the forecast period (2020-2034)?

Current Krabbe Disease Treatment Scenario, Marketed Drugs and Emerging Therapies:

• What are the current options for the Krabbe Disease treatment in addition to the approved therapies?
• What are the current treatment guidelines for the treatment of Krabbe Disease in the USA, Europe, and Japan?
• What are the Krabbe Disease 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 Krabbe Disease?
• How many therapies are in-development by each company for Krabbe Disease treatment?
• How many are emerging therapies in mid-stage, and late stage of development for Krabbe Disease treatment?
• What are the key collaborations (Industry - Industry, Industry - Academia), Mergers and acquisitions, licensing activities related to the Krabbe Disease 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 clinical studies going on for Krabbe Disease and their status?
• What are the current challenges faced in drug development?
• What are the key designations that have been granted for the emerging therapies for Krabbe Disease?
• What are the global historical and forecasted market of Krabbe Disease?

Reasons to buy Krabbe Disease Market Forecast Report

• The report will help in developing business strategies by understanding trends shaping and driving the Krabbe Disease market
• To understand the future market competition in the Krabbe Disease market and Insightful review of the key market drivers and barriers
• Organize sales and marketing efforts by identifying the best opportunities for Krabbe Disease 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 Krabbe Disease market
• To understand the future market competition in the Krabbe Disease market