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.
Study Period: 2018–2030
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.
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.
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.
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…
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.
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.
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.
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.
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.
Market Insights:
Epidemiology Insights:
Current Treatment Scenario, Marketed Drugs, and Emerging Therapies:
1. Key Insights
2. Report Introduction
3. Netherton Syndrome Market Overview at a Glance
3.1. Market Share (%) Distribution of Netherton Syndrome in 2018
3.2. Market Share (%) Distribution of Netherton Syndrome in 2030
4. Executive Summary of Netherton Syndrome
5. Disease Background and Overview
5.1. Introduction
5.2. Clinical Manifestations
5.3. Pathogenesis
5.4. Diagnosis
5.5. Treatment of Netherton Syndrome (NS)
6. Epidemiology and Patient Population
6.1. Key Findings
6.2. 7MM Total Prevalent Population of Netherton Syndrome
6.3. Epidemiology of Netherton Syndrome
6.4. The United States
6.4.1. Prevalent Cases of Netherton Syndrome in the United States
6.4.2. Severity-specific cases of Netherton Syndrome in the United States
6.5. EU5
6.5.1. Germany
6.5.1.1. Prevalent Cases of Netherton Syndrome in Germany
6.5.1.2. Severity-specific cases of Netherton Syndrome in Germany
6.5.2. France
6.5.2.1. Prevalent Cases of Netherton Syndrome in France
6.5.2.2. Severity-specific cases of Netherton Syndrome in France
6.5.3. Italy
6.5.3.1. Prevalent Cases of Netherton Syndrome in Italy
6.5.3.2. Severity-specific cases of Netherton Syndrome in Italy
6.5.4. Spain
6.5.4.1. Prevalent Cases of Netherton Syndrome in Spain
6.5.4.2. Severity-specific cases of Netherton Syndrome in Spain
6.5.5. United Kingdom
6.5.5.1. Prevalent Cases of Netherton Syndrome in the UK
6.5.5.2. Severity-specific cases of Netherton Syndrome in the United Kingdom
6.6. Japan
6.6.1. Prevalent Cases of Netherton Syndrome in Japan
6.6.2. Severity-specific cases of Netherton Syndrome in Japan
7. Organizations contributing toward the fight against NS
8. Patient Journey 59
9. Case Study
10. Emerging Drugs
10.1. Key Cross: Emerging Therapies
10.2. LM-030: Lifemax Laboratories
10.2.1. Other development activities
10.2.2. Other development activities
10.2.3. Product Profile
10.3. Imsidolimab: AnaptysBio
10.3.1. Drug Description
10.3.2. Clinical development
10.3.3. Clinical trial information
10.3.4. Safety and Efficacy
10.3.5. Product Profile
11. Other Assets
11.1. Key Cross: Other Assets
11.2. Ustekinumab: Janssen Biotech
11.2.1. Drug Description
11.2.2. Clinical Development
11.2.3. Clinical trial information
11.2.4. Product Description
11.3. Secukinumab: Northwestern University
11.3.1. Drug Description
11.3.2. Clinical Development
11.3.3. Clinical trial information
11.3.4. Product Profile
11.4. Dupilumab: University Hospital
11.4.1. Drug Description
11.4.2. Clinical development
11.4.3. Clinical trial information
11.4.4. Product Profile
12. Netherton Syndrome: 7 Major Market Analysis
12.1. Key Findings
12.2. Market Size of Netherton Syndrome in 7MM
12.3. Market Outlook
12.4. United States Market Size
12.4.1. Total Market Size of Netherton Syndrome in the United States
12.4.2. Market Size of Netherton Syndrome by Therapies in the United States
12.5. EU-5 Market Size
12.6. Germany
12.6.1. Total Market size of Netherton Syndrome in Germany
12.6.2. Market Size of Netherton Syndrome by Therapies in Germany
12.7. France
12.7.1. Total Market size of Netherton Syndrome in France
12.7.2. Market Size of Netherton Syndrome by Therapies in France
12.8. Italy
12.8.1. Total Market size of Netherton Syndrome in Italy
12.8.2. Market Size of Netherton Syndrome by Therapies in Italy
12.9. Spain
12.9.1. Total Market size of Netherton Syndrome in Spain
12.9.2. Market Size of Netherton Syndrome by Therapies in Spain
12.10. United Kingdom
12.10.1. Total Market size of Netherton Syndrome in the United Kingdom
12.10.2. Market Size of Netherton Syndrome by Therapies in the United Kingdom
12.11. Japan
12.11.1. Total Market size of Netherton Syndrome in Japan
12.11.2. Market Size of Netherton Syndrome by Therapies in Japan
13. KOL Views
14. Market Drivers
15. Market Barrier
16. SWOT Analysis
17. Unmet Needs
18. Appendix
18.1. Bibliography
18.2. Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight
List of Table
Table 1: Summary of Netherton Syndrome, Market, Epidemiology, and Key Events (2018–2030)
Table 2: Prevalence Population of Netherton Syndrome in 7MM (2018–2030)
Table 3: Prevalent Cases of Netherton Syndrome in the United States (2018–2030)
Table 4: Severity-specific cases of Netherton Syndrome in the United States (2018–2030)
Table 5: Prevalent Cases of Netherton Syndrome in Germany (2018–2030)
Table 6: Severity-specific cases of Netherton Syndrome in Germany (2018–2030)
Table 7: Prevalent Cases of Netherton Syndrome in France (2018–2030)
Table 8: Severity-specific cases of Netherton Syndrome in France (2018–2030)
Table 9: Prevalent Cases of Netherton Syndrome in Italy (2018–2030)
Table 10: Severity-specific cases of Netherton Syndrome in Italy (2018–2030)
Table 11: Prevalent Cases of Netherton Syndrome in Spain (2018–2030)
Table 12: Severity-specific cases of Netherton Syndrome in Spain (2018–2030)
Table 13: Prevalent Cases of Netherton Syndrome in the United Kingdom (2018–2030)
Table 14: Severity-specific cases of Netherton Syndrome in the United Kingdom (2018–2030)
Table 15: Prevalent Cases of Netherton Syndrome in Japan (2018–2030)
Table 16: Severity-specific cases of Netherton Syndrome in Japan (2018–2030)
Table 17: Organizations contributing toward the fight against NS
Table 18: Imsidolimab, Clinical Trial Description, 2021
Table 19: Ustekinumab, Clinical Trial Description, 2021
Table 20: Secukinumab, Clinical Trial Description, 2021
Table 21: Dupilumab, Clinical Trial Description, 2021
Table 22: Seven Major Market Size of Netherton Syndrome in USD Million (2018–2030)
Table 23: The United States Market Size of Netherton Syndrome in USD Million (2018–2030)
Table 24: The United States Market Size of Netherton Syndrome by Therapies in USD Million (2018–2030)
Table 25: Germany Market Size of Netherton Syndrome in USD Million (2018–2030)
Table 26: Germany Market Size of Netherton Syndrome by Therapies in USD Million (2018–2030)
Table 27: France Market Size of Netherton Syndrome in USD Million (2018–2030)
Table 28: France Market Size of Netherton Syndrome by Therapies in USD Million (2018–2030)
Table 29: Italy Market Size of Netherton Syndrome in USD Million (2018–2030)
Table 30: Italy Market Size of Netherton Syndrome by Therapies in USD Million (2018–2030)
Table 31: Spain Market Size of Netherton Syndrome in USD Million (2018–2030)
Table 32: Spain Market Size of Netherton Syndrome by Therapies in USD Million (2018–2030)
Table 33: United Kingdom Market Size of Netherton Syndrome in USD Million (2018–2030)
Table 34: United Kingdom Market Size of Netherton Syndrome by Therapies in USD Million (2018–2030)
Table 35: Japan Market Size of Netherton Syndrome in USD Million (2018–2030)
Table 36: Japan Market Size of Netherton Syndrome by Therapies in USD Million (2018–2030)
List of Figures
Figure 1: The common cutaneous and extra-cutaneous manifestations observed in Netherton syndrome patients
Figure 2: Clinical signs and symptoms of Netherton syndrome
Figure 3: Schematic representation of NS patient skin in comparison to healthy skin.
Figure 4: Signaling pathways underlying the pathophysiology of Netherton syndrome
Figure 5: Treatment options in Netherton Syndrome
Figure 6: Prevalent Population of Netherton Syndrome in 7MM (2018–2030)
Figure 7: Prevalent Cases of Netherton Syndrome in the United States (2018–2030)
Figure 8: Severity-specific cases of Netherton Syndrome in the United States (2018–2030)
Figure 9: Prevalent Cases of Netherton Syndrome in Germany (2018–2030)
Figure 10: Severity-specific cases of Netherton Syndrome in Germany (2018–2030)
Figure 11: Prevalent Cases of Netherton Syndrome in France (2018–2030)
Figure 12: Severity-specific cases of Netherton Syndrome in France (2018–2030)
Figure 13: Prevalent Cases of Netherton Syndrome in Italy (2018–2030)
Figure 14: Severity-specific cases of Netherton Syndrome in Italy (2018–2030)
Figure 15: Prevalent Cases of Netherton Syndrome in Spain (2018–2030)
Figure 16: Severity-specific cases of Netherton Syndrome in Spain (2018–2030)
Figure 17: Prevalent Cases of Netherton Syndrome in the United Kingdom (2018–2030)
Figure 18: Severity-specific cases of Netherton Syndrome in the United Kingdom (2018–2030)
Figure 19: Prevalent Cases of Netherton Syndrome in Japan (2018–2030)
Figure 20: Severity-specific cases of Netherton Syndrome in Japan (2018–2030)
Figure 21: Seven Major Market Size of Netherton Syndrome in USD Million (2018–2030)
Figure 22: Market Size of Netherton Syndrome in the United States, USD Million (2018–2030)
Figure 23: The United States Market Size of Netherton Syndrome by Therapies in USD Million
Figure 24: Market Size of Netherton Syndrome in Germany, USD Million (2018–2030)
Figure 25: Germany Market Size of Netherton Syndrome by Therapies in USD Million
Figure 26: Market Size of Netherton Syndrome in France, USD Million (2018–2030)
Figure 27: France Market Size of Netherton Syndrome by Therapies in USD Million (2018–2030)
Figure 28: Market Size of Netherton Syndrome in Italy, USD Million (2018–2030)
Figure 29: Italy Market Size of Netherton Syndrome by Therapies in USD Million (2018–2030)
Figure 30: Market Size of Netherton Syndrome in Spain, USD Million (2018–2030)
Figure 31: Spain Market Size of Netherton Syndrome by Therapies in USD Million (2018–2030)
Figure 32: Market Size of Netherton Syndrome in the United Kingdom, USD Million (2018–2030)
Figure 33: The United Kingdom Market Size of Netherton Syndrome by Therapies in USD Million
Figure 34: Market Size of Netherton Syndrome in Japan, USD Million (2018–2030)
Figure 35: Japan Market Size of Netherton Syndrome by Therapies in USD Million
• Lifemax Laboratories
• AnaptysBio
• Janssen Biotech
• Northwestern University
• University Hospital
