Netherton Syndrome Market

DelveInsight’s ‘Netherton Syndrome – Market Insights, Epidemiology, and Market Forecast – 2030’ report delivers an in-depth understanding of the historical and forecasted epidemiology as well as the market trends of Netherton Syndrome in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.

 

The Netherton Syndrome market report provides current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted Netherton Syndrome market Size from 2018 to 2030, segmented by seven major markets. The report also covers the current Netherton Syndrome treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying market potential.

Geography Covered

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

Study Period: 2018–2030

Netherton Syndrome: Disease Understanding and Treatment Algorithm

Netherton Syndrome Overview

Netherton syndrome (NS) is a rare and severe genetic autosomal recessive disorder of Ichthyosis. It is an inherited skin condition, often present at birth or in the first few weeks of life, where the skin is very red with fine dry scales. The condition tends to last for a lifetime, but the severity can vary from person to person.

 

It was described by Comèl (1949) and Netherton (1958). The syndrome is sometimes called Comèl-NS and is characterized by a clinical trial of ichthyosiform erythroderma, a specific hair shaft abnormality (trichorrhexis invaginata, or bamboo hair), atopic manifestations, and multisystemic complications.

 

NS is caused by loss of function mutations in the SPINK5 gene that encodes a serine peptidase inhibitor, Lympho-epithelial Kazal-type-related inhibitor (LEKTI). The loss of LEKTI leads to dysregulation of epidermal proteases and severe skin barrier impairment. Kallikrien-related peptidases, such as KLK7, and the epidermal elastase 2 (ELA2), which LEKTI inhibits, are reported to play major roles in NS pathology.

 

NS is diagnosed based on the symptoms and confirmed by genetic testing for changes in the SPINK5 gene. Other types of testing, such as close examination of the hair and a skin biopsy to obtain a small skin sample for examination under the microscope, can also be helpful for diagnosis. Often babies or children are not diagnosed for months or even years as red skin at birth can be a symptom of other conditions, such as other types of ichthyosis, severe eczema, or other forms of immune deficiency. Thus, differential diagnosis plays an important role.

 

There is no approved treatment for NS. Current approaches are all limited to symptom relief or supportive care with marginal efficacy and undesirable side effects. The treatment includes cleansing skin by non-detergent liquid cleansing oil, use of emollients, moisturizers, keratolytic, antipruritics, anti-histamines, antibiotics, topical corticosteroids, retinoids, calcipotriol, and Calcineurin inhibitors. Intravenous immunoglobulin and anti-TNF-α are therapeutic options for severe illness.

 

There is an urgent need for a targeted, effective and well-tolerated therapy. Further understanding of the underlying pathophysiology of skin changes will lead to more effective therapeutic modalities, including possible gene therapy.

 

Netherton Syndrome Diagnosis and Treatment

It covers the details of conventional and current medical therapies and diagnosis available in the Netherton Syndrome market to treat the condition. It also provides country-wise treatment guidelines and algorithms across the United States, Europe, and Japan.

 

The DelveInsight Netherton Syndrome market report gives a thorough understanding of Netherton Syndrome by including disease definition, symptoms, causes, pathophysiology, and diagnosis. It also provides the treatment algorithms and treatment guidelines for Netherton Syndrome in the US, Europe, and Japan.

Netherton Syndrome Epidemiology

The Netherton Syndrome epidemiology division provides insights about the historical and current patient pool along with the forecasted trend for every seven major countries. It helps 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 disease epidemiology covered in the report provides historical and forecasted Netherton Syndrome epidemiology segmented as the prevalent population of Netherton Syndrome and severity-specific cases of Netherton Syndrome. The report includes the prevalent scenario of Netherton Syndrome in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2018 to 2030.

Country-Wise Netherton Syndrome Epidemiology

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

 

The prevalent population of Netherton Syndrome in the 7MM countries was estimated to be 3,411 cases in 2020.

Netherton Syndrome Drug Chapters

The drug chapter segment of the Netherton Syndrome report encloses the detailed analysis of Netherton Syndrome marketed drugs and late stage (Phase-III, Phase-II/III, Phase-II, and Phase-I/II) pipeline drugs. It also helps understand the Netherton Syndrome 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.

 

Over the past years, treatment options for NS have not evolved; current approaches are all limited to symptomatic relief or supportive care with marginal efficacy and undesirable side effects. A gentle/soft non-detergent liquid cleansing oil, preferably with an acidic pH (5) to counteract overactive serine proteases, is recommended for daily bath and/or shower. Liberal use of bland emollients along with topical corticosteroids and calcineurin inhibitors (tacrolimus and pimecrolimus) for inflammatory skin lesions is also advisable. However, topical corticosteroids carry a risk of increased systemic absorption due to skin barrier defects.

 

Topical tacrolimus has shown efficacy in NS patients, with daily applications reducing erythema. However, their dose and frequency of use should be strictly monitored to avoid toxicity due to systemic absorption, additional barrier damage, or possible persistence of lesions. Oral acitretin has also been demonstrated to improve cutaneous lesions and foster hair growth in regions of alopecia associated with the disease. Furthermore, Narrowband UVB (NB-UVB) phototherapy and psoralen UVA therapy may improve the cutaneous lesion. The adverse effects of NB-UVB phototherapy have been linked to dose and frequency; while erythemal doses damage the barrier, suberythemal doses enhance barrier function and production of antimicrobial peptides

Products detail in the report…

 

Netherton Syndrome Emerging Drugs

 

LM-030 (Lifemax Laboratories), an inhibitor of kallikrein-related peptidase 7 (KLK7) and epidermal elastase 2 (ELA2), targets the underlying cause of Netherton syndrome (NS) and is well-positioned to become the first approved treatment for this debilitating and potentially life-threatening disease. It has completed extensive preclinical testing and demonstrated positive proof of concept (POC) in a Phase IIa study in NS. The drug has received fast track designation, orphan drug designation, and rare pediatric disease designation from the US Food and Drug Administration (FDA) to treat NS and an orphan drug designation from the European Commission (EC). The rare pediatric disease designation may entitle Lifemax to receive a pediatric rare disease priority review voucher upon LM-030 approval. The drug is currently in Phase II/III trial for the treatment of NS.

 

Imsidolimab (AnaptysBio), previously known as ANB019, is an antibody that inhibits the function of the interleukin-36-receptor, or IL-36R. It is being developed as a potential first-in-class therapy for patients who have generalized pustular psoriasis (GPP), EGFRi-mediated skin toxicity, ichthyosis, hidradenitis suppurativa (HS), and acne. The company is currently conducting a placebo-controlled Phase II trial of imsidolimab to treat IL-36 associated ichthyosis subtypes, with topline data anticipated in 2022.

 

Ustekinumab (Jassen Biotech) is a human IgG1қ monoclonal antibody that binds with specificity to the p40 protein subunit used by IL-12 and IL-23 cytokines. IL-12 and IL-23 are naturally occurring cytokines that are involved in inflammatory and immune responses, such as natural killer (NK) cell activation and CD4+ T-cell differentiation and activation. In in vitro models, ustekinumab was shown to disrupt IL-12 and IL-23 mediated signaling and cytokine cascades by disrupting the interaction of these cytokines with a shared cell-surface receptor chain, IL-12Rβ1. The company is currently conducting a Phase I trial in collaboration with Northwestern University to evaluate the efficacy and safety of ustekinumab in the treatment of patients with Ichthyosis.

Products detail in the report…

Netherton Syndrome Market Outlook

The Netherton Syndrome market outlook of the report builds a detailed comprehension of the historical, current, and forecasted Netherton Syndrome market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers, and demand for better technology.

 

This segment gives a thorough detail of Netherton Syndrome 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 for 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.

 

According to DelveInsight, the Netherton Syndrome market in the 7MM is expected to change in the study period 2018–2030.

 

Due to the nature of the disease, over-the-counter (OTC) products like moisturizers and topical emollients are often used by patients either self-prescribed or at the discretion of the treating physician. The forecast, however, only considers active treatment being prescribed. The launch of the key assets (LM-030 and Imsidolimab) during the forecast period (2018–2030) would significantly fuel the market. Also, considering the high unmet needs prevailing the treatment, the willingness to pay for efficient treatment is high among the consumers, which will add to the market growth.

 

Key Findings

This section includes a glimpse of the Netherton Syndrome market in the 7MM. The market size of Netherton Syndrome in the seven major markets was USD 14.6 million in 2020.

 

The United States: Market Outlook

This section provides Netherton Syndrome market size and market size by therapies of Netherton Syndrome in the United States.

 

The United States accounts for the largest market size of Netherton Syndrome compared to the EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.

There is an urgent need for a targeted, effective and well-tolerated therapy. Kallikrein inhibitors represent a new potential therapy for NS. Kallikrein inhibition is typically absent in NS, and thus, the inhibitor properties of this treatment may prevent or minimize the inflammatory cutaneous features that are generally present. Sixera Pharma has identified benzoxazinone derivatives as selective inhibitors of KLK5, KLK7, and KLK14. Final preclinical development of the lead molecule SXR1096 and preparation for clinical testing is currently ongoing. SXR1096 has been granted an orphan drug designation in the EU and US for the treatment of NS. In addition, BridgeBio is developing a topically formulated small-molecule inhibitor BBP-561 which targets both KLK5 and KLK7. The BBP-561 molecule is currently in a preclinical development stage.

 

Another lead molecule, LM-030 (previously known as BPR277), now licensed to LifeMax, has been granted Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation by the FDA for topical treatment of NS. It is an inhibitor of kallikrein-related peptidase 7 (KLK7) and epidermal elastase 2 (ELA2), targets the underlying cause of NS, and is well-positioned to become the first approved treatment for this debilitating and potentially life-threatening disease. It has completed extensive preclinical testing and demonstrated positive proof of concept (PoC) in a Phase IIa study in NS. Other promising assets are Dupixent (dupilumab, anti-IL-4Rα antibody), Cosentyx (Secukinumab, anti-IL-17A antibody), and ustekinumab (anti-IL-12 and IL-23 antibody); the outcome of these trials has not been published.

 

EU-5 Countries: Market Outlook

The total Netherton Syndrome market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are also mentioned.

 

Japan Market Outlook

The total Netherton Syndrome market size and market size by therapies of Netherton Syndrome in Japan are also mentioned.

Netherton Syndrome Drugs Uptake

This section focuses on the uptake of the potential drugs recently launched or expected to get launched in the market during the study period 2018–2030. The analysis covers Netherton Syndrome market uptake by drugs, patient uptake by therapies, and drug sales. 

 

This 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 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.

Netherton Syndrome Pipeline Development Activities

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

 

Major players include Lifemax Laboratories and AnaptysBio, whose key products are expected to get launched in the US market by 20XX.

 

Pipeline Development Activities

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

KOL Views

To keep up with current market trends, we take KOLs and SME’s opinion working in the Netherton Syndrome domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps understand and validate current and emerging therapies treatment patterns or Netherton Syndrome 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

We perform a Competitive and Market Intelligence analysis of the Netherton Syndrome market by using various Competitive Intelligence tools: 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 Netherton Syndrome, explaining its causes, signs and symptoms, pathophysiology, and currently available therapies
• Comprehensive insight has been provided into the Netherton Syndrome epidemiology and treatment in the 7MM
• Additionally, an all-inclusive account of both the current and emerging therapies for Netherton Syndrome is provided, along with the assessment of new therapies, which will impact the current treatment landscape.
• A detailed review of the Netherton Syndrome 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 Netherton Syndrome market

Report Highlights

• In the coming years, the Netherton Syndrome 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 Netherton Syndrome 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 Netherton Syndrome. The launch of emerging therapies will significantly impact the Netherton Syndrome market
• A better understanding of disease pathogenesis will also contribute to developing novel therapeutics for Netherton Syndrome.
• 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-competitor, 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

Netherton Syndrome Report Insights

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

Netherton Syndrome Report Key Strengths

• 10-years Forecast
• 7MM Coverage
• Netherton Syndrome Epidemiology Segmentation
• Key Competitors
• Highly Analyzed Market
• Drugs Uptake

Netherton Syndrome Report Assessment

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

Key Questions

Market Insights:

• What was the Netherton Syndrome Market share (%) distribution in 2018, and how would it look like in 2030?
• What would be the Netherton Syndrome total market Size and market Size by therapies across the 7MM during the forecast period (2018–2030)?
• What are the key findings pertaining to the market across 7MM, and which country will have the largest Netherton Syndrome market Size during the forecast period (2018–2030)?
• At what CAGR, the Netherton Syndrome market is expected to grow in the 7MM during the forecast period (2018–2030)?
• What would be the Netherton Syndrome market outlook across the 7MM during the forecast period (2018–2030)?
• What would be the Netherton Syndrome market growth till 2030, and what will be the resultant market Size in the year 2030?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

 

Epidemiology Insights:

• What are the disease risk, burden, and unmet needs of Netherton Syndrome?
• What is the historical Netherton Syndrome 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 Netherton Syndrome in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
• What will be the growth opportunities in the 7MM with respect to the patient population pertaining to Netherton Syndrome?
• Out of all 7MM countries, which country would have the highest prevalence of Netherton Syndrome during the forecast period (2018–2030)?
• At what CAGR the population is expected to grow in the 7MM during the forecast period (2018–2030)?

 

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies:

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

Reasons to buy

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