팀버 파마슈티컬스(Timber Pharmaceuticals)와 크리스탈 바이오텍을 포함한 주요 기업들은 층상어피증 치료를 위해 다양한 임상 개발 단계를 거쳐 선도 후보물질들을 적극적으로 추진하고 있습니다. 팀버 파마슈티컬스는 초기 임상시험에서 유망한 결과를 보인 이소트레티노인의 국소 제형인 TMB-001을 개발 중입니다. 한편 Krystal Biotech는 층판상 어린선의 근본적인 유전적 원인을 표적으로 하는 유전자 치료 접근법을 탐구하고 있습니다. 이러한 노력은 제약사들이 층판상 어린선 치료 부문의 상당한 미충족 수요를 충족시키기 위한 혁신적인 솔루션에 집중하는 시장의 광범위한 추세의 일부입니다. 이러한 치료법들이 임상 시험을 진행함에 따라 층판상 어린선 치료제의 미래 시장은 확대될 것으로 예상되며, 환자에게 새로운 희망과 개선된 결과를 제공할 것입니다.
본 보고서에서는 층판상 어린선(LI)의 주요 7개 시장(미국, 독일, 스페인, 이탈리아, 프랑스, 영국, 일본)에 대한 조사 분석을 실시하고, 각국 시장 규모와 예측, 각 치료법 시장 점유율, 현재의 치료법, 미충족 필요 등의 정보를 제공합니다.
DelveInsight's "Lamellar Ichthyosis - Market Insights, Epidemiology and Market Forecast - 2034" report delivers an in-depth understanding of lamellar ichthyosis, historical and forecasted epidemiology as well as the lamellar ichthyosis market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.
Lamellar Ichthyosis market report provides real-world prescription pattern analysis, emerging drugs, market share of individual therapies, and historical and forecasted 7MM lamellar ichthyosis market size from 2020 to 2034. The report also covers current lamellar ichthyosis treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's underlying potential.
Lamellar Ichthyosis Overview, Country-Specific Treatment Guidelines and Diagnosis
Lamellar ichthyosis is a skin condition that primarily affects infants, who are typically born with a tight, clear sheath covering their skin known as a collodion membrane. This membrane usually dries and peels off within the first few weeks of life, revealing scaly skin and outwardly turned eyelids and lips. The condition is characterized by large, dark, plate-like scales covering most of the body. Infants with lamellar ichthyosis may face several complications, including infections, dehydration due to excessive fluid loss, and respiratory issues. Additional symptoms include hair loss (alopecia), abnormal fingernails and toenails (nail dystrophy), decreased sweating (hypohidrosis), increased heat sensitivity, and thickened skin on the palms and soles (keratoderma). In some cases, individuals may also experience reddened skin (erythema) and joint deformities (contractures).
Prenatal diagnosis of lamellar ichthyosis can be achieved by electron microscopic examination of fetal skin biopsies or amniotic fluid cells obtained via fetoscopy. DNA-based molecular genetic testing is commercially available for TGM1, ABCA12, ALOXE3 and ALOX12B.22 Alternatively, skin biopsies may be examined by light microscopy and electron microscopy for histological features, or for detection of in situ TGM1 expression and activity.
The lamellar ichthyosis report provides an overview of lamellar ichthyosis pathophysiology, diagnostic approaches, and detailed treatment algorithm along with a real-world scenario of a patient's journey beginning from the first symptom, the time taken for diagnosis to the entire treatment process.
Lamellar Ichthyosis Treatment
Lamellar ichthyosis has no cure, so treatment focuses on alleviating symptoms and improving skin condition through a combination of topical and systemic therapies. Topical treatments include emollients such as petrolatum and lanolin to maintain skin hydration, and keratolytics like alpha-hydroxy acids and urea to reduce scale thickness and promote exfoliation. Topical retinoids, such as tretinoin, can also be beneficial. In more severe cases, systemic treatments like oral retinoids are used to enhance epidermal turnover and manage hyperkeratosis, though these can have potential side effects and are used with caution.
The lamellar ichthyosis epidemiology chapter in the report provides historical as well as forecasted in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan from 2024 to 2034. The lamellar ichthyosis epidemiology is segmented with detailed insights into Total Prevalent Cases of Lamellar Ichthyosis, Total Diagnosed Prevalent Cases of Lamellar Ichthyosis, Genetic Mutation-specific Cases of Lamellar Ichthyosis, Severity-Specific Diagnosed Prevalent Cases of Lamellar Ichthyosis, and Total Treated Cases of Lamellar Ichthyosis.
The drug chapter segment of the lamellar ichthyosis report encloses a detailed analysis of lamellar ichthyosis marketed drugs and late-stage (Phase III and Phase II) pipeline drugs. It also deep dives into the lamellar ichthyosis pivotal clinical trial details, recent and expected market approvals, patent details, the latest news, and recent deals and collaborations.
Emerging Drugs
TMB-001: Timber Pharmaceuticals/ LEO Pharma
TMB-001, a topical form of isotretinoin, is currently being developed for the treatment of moderate to severe subtypes of congenital ichthyosis (CI). In the fourth quarter of 2021, Timber Pharmaceuticals announced positive top-line results from its Phase IIb CONTROL Study. The study demonstrated that TMB-001 has clinically meaningful efficacy and a favorable safety profile, highlighting its potential as an important treatment option for CI. This is especially significant given the lack of FDA-approved treatments and the limited options available in the standard of care for this condition. Timber initiated the Phase III ASCEND clinical trial in Quarter2 2022. TMB-001, has received an orphan and breakthrough designation by the FDA.
KB105: Krystal Biotech
KB105 is an investigational topical gene therapy developed by Krystal Biotech for the treatment of autosomal recessive congenital ichthyosis (ARCI), specifically targeting lamellar ichthyosis. The therapy aims to address the underlying genetic defect associated with LI by delivering a functional copy of the TGM1 gene, which is crucial for proper skin barrier function. Currently, KB105 is undergoing Phase II clinical trials to evaluate its safety and efficacy.
Key players, including Timber Pharmaceuticals and Krystal Biotech, are actively advancing their lead candidates through various stages of clinical development to address lamellar ichthyosis. Timber Pharmaceuticals is progressing with TMB-001, a topical form of isotretinoin, which has shown promising results in early trials. Meanwhile, Krystal Biotech is exploring gene therapy approaches to target the underlying genetic causes of lamellar ichthyosis. These efforts are part of a broader trend in the market, where pharmaceutical companies are focusing on innovative solutions to meet the significant unmet needs in LI treatment. As these therapies progress through clinical trials, the future market for lamellar ichthyosis treatments is expected to expand, offering new hope and improved outcomes for patients.
This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2024-2034, which depends on the competitive landscape, safety, and efficacy data along with order of entry. It is important to understand that the key players evaluating their novel therapies in the pivotal and confirmatory trials should remain vigilant when selecting appropriate comparators to stand the greatest chance of a positive opinion from regulatory bodies, leading to approval, smooth launch, and rapid uptake.
Lamellar Ichthyosis Activities
The report provides insights into different therapeutic candidates in Phase III and Phase II stages. It also analyzes key players involved in developing targeted therapeutics.
Pipeline Development Activities
The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for lamellar ichthyosis emerging therapies.
KOL Views
To keep up with the real-world scenario in current and emerging market trends, we take opinions from Key Industry leaders working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts were contacted for insights on the evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake along with challenges related to accessibility.
DelveInsight's analysts connected with 10+ KOLs to gather insights; however, interviews were conducted with 5+ KOLs in the 7MM. Their opinion helps understand and validate current and emerging treatment patterns of lamellar ichthyosis. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.
Qualitative Analysis
We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of gaps in disease diagnosis, patient awareness, physician acceptability, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided.
Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.
In efficacy, the trial's primary and secondary outcome measures are evaluated; for instance, in event-free survival, one of the most important primary outcome measures is event-free survival and overall survival.
Further, the therapies' safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.
Market Access and Reimbursement
The report provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of currently used therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.
16.4.1.Total Market Size of Lamellar Ichthyosis (LI) in the US
16.4.2.Market Size of Lamellar Ichthyosis (LI) by Therapies in the US
16.6.1.Total Market Size of Lamellar Ichthyosis (LI) in Japan
16.6.2.Market Size of Lamellar Ichthyosis (LI) by Therapies in Japan