Date: 03/25/2020

  • ID: 5006323 
  • Drug Pipelines 
  • March 2020
  • Region: Global 
  • 200 Pages 
  • DelveInsight


  • Galderma/Mayne Pharma
  • Krystal Biotech
  • Timber Pharmaceuticals
  • MORE
‘Lamellar Ichthyosis (LI)-Market Insights, Epidemiology and Market Forecast-2030’ report delivers an in-depth understanding of the Lamellar Ichthyosis, historical and forecasted epidemiology as well as the Lamellar Ichthyosis market trends in the United States, EU5 (Germany, France, Italy, Spain, and United Kingdom) and Japan.

The Lamellar Ichthyosis market report provides current treatment practices, emerging drugs, Lamellar Ichthyosis market share of the individual therapies, current and forecasted Lamellar Ichthyosis market Size from 2017 to 2030 segmented by seven major markets. The Report also covers current Lamellar Ichthyosis treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses underlying potential of the market.

Geography Covered
  • The United States
  • EU5 (Germany, France, Italy, Spain and the United Kingdom)
  • Japan
Study Period: 2017–2030

Lamellar Ichthyosis (LI): Disease Understanding and Treatment Algorithm:

Lamellar Ichthyosis Overview

Ichthyosis is the term used to define repetitive and widespread scaling of the skin. It may be inherited (genetic) or acquired during the lifespan. The inherited forms are rare, usually present from infancy, and generally lifelong situations. Acquired Ichthyosis can progress at any age due to several medical problems, such as kidney disease. The most common forms of inherited Ichthyosis are mild, and it might improve during warm climate. There are many very rare conditions where Ichthyosis occurs with problems in other systems of the body. Inherited Ichthyosis is due to a single genetic trait that is passed on either from one or both parents or develops as a new error in the gene very early in fetal life. It can be mild as with Ichthyosis Vulgaris, or severe.

Lamellar Ichthyosis is a rare genetic disorder, where skin cells are produced at a normal rate, but they do not separate normally at the surface of the outermost layer of skin and are not shed as quickly as they should be which lead to the formation of scale.

The signs and symptoms of Lamellar Ichthyosis differ from individual to individual. Some have mild signs and symptoms, while for others it might be severe.

Lamellar Ichthyosis Diagnosis:

It is mainly caused by the mutations affecting several different genes, the genes which mainly involved in Lamellar Ichthyosis include TGM1, ABCA12, CYP4F22, NIPAL4 and others like LIPN, ALOX12B. However, most of the cases (~90%) are caused due to mutation in the TGM1 gene.

Mutations in lamellar Ichthyosis commonly transmitted through autosomal recessive inheritance. Individuals must inherit two recessive genes for lamellar Ichthyosis to show the disease, one from each parent. Also, the diagnosis of a genetic or rare disease can often be puzzling. Healthcare specialists usually look at an individuals’ medical history, symptoms, physical exam, and laboratory test results to make a diagnosis. The patient’s history includes thorough family history.

The diagnosis of Lamellar Ichthyosis is mainly done skin biopsy for histological analysis, electron microscopy, DNA sequencing analysis and immunohistochemical analysis and Ultrasonography.

Lamellar Ichthyosis Treatment:

Currently, there is no approved therapy for the treatment of Lamellar Ichthyosis, however, the off-label therapies like retinoids, emollients, and keratolytics are available for symptomatic treatment like removal of scales.

The general treatment course is aimed at controlling symptoms with exfoliating creams and ointments application being found to be crucial. Prescription creams and ointments containing alpha hydroxy acids, such as lactic acid and glycolic acid, help control scaling and increase skin moisture. Keratolytic agents such as 1-5% salicylic acid may be added to emollient cream bases to encourage exfoliation of scale, but are often too irritant for regular use.

Lamellar Ichthyosis Epidemiology:

The Lamellar Ichthyosis epidemiology division provide the insights about historical and current Lamellar Ichthyosis patient pool and forecasted trend for each 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 report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

Key Findings:

The total prevalent cases of Lamellar Ichthyosis in the 7MM were found to be 4,038 in 2017 which is expected grow during the study period, i.e., 2017–2030.

The disease epidemiology covered in the report provides historical as well as forecasted Lamellar Ichthyosis epidemiology [segmented as Total Prevalent Cases of Lamellar Ichthyosis, Total Diagnosed Cases of Lamellar Ichthyosis, Genetic Mutation-specific Cases of Lamellar Ichthyosis, Severity-Specific Cases of Lamellar Ichthyosis, and Total Treated Cases of Lamellar Ichthyosis] scenario of Lamellar Ichthyosis in the 7MM covering United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan from 2017 to 2030.

Country Wise- Lamellar Ichthyosis Epidemiology:

Estimates show the highest prevalent population of Lamellar Ichthyosis is in the United States followed by Japan, Germany, France, and the United Kingdom in the year 2017.

Lamellar Ichthyosis Drug Chapters:

Drug chapter segment of the Lamellar Ichthyosis report encloses the detailed analysis of Lamellar Ichthyosis marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the Lamellar Ichthyosis 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.

Lamellar Ichthyosis Emerging Drugs:

TMB-001 (Isotretinoin, formerly known as PAT-001): Timber Pharmaceuticals

TMB-001 (topical isotretinoin) is under clinical development for the treatment of moderate to severe subtypes of congenital ichthyosis. It is a topical ointment formulation (0.05% and 0.1%) of isotretinoin, utilizing Timber's proprietary IPEG delivery system.

In April 2014, the US FDA granted an orphan drug designation to TMB 001 for the treatment of Congenital Ichthyosis. Also in February 2019, Timber Pharmaceuticals, and Patagonia Pharmaceuticals entered into the “Asset Acquisition Agreement”, under which Timber Pharmaceuticals purchased the topical formulation of isotretinoin for the treatment of Congenital Ichthyosis and identified as PAT-001.

Trifarotene (CD5789): Galderma/Mayne Pharma

Trifarotene is a new chemical entity, currently being investigated by Galderma and Mayne Pharma for autosomal recessive ichthyosis with lamellar scale. It is a novel retinoid in a topical cream formulation with high selectivity for the type of Retinoic Acid Receptor (RAR) found specifically on the skin. As per the company, the drug absorb quickly and might have a low potential for toxicity than retinoid given orally or other existing topical retinoid.

In July 2014, the US FDA granted orphan drug designation to Trifarotene molecule for the treatment of congenital ichthyosis. Based on this decision, Galderma planned to implement a clinical development plan, reinforcing its commitment to exploring new treatment options for rare diseases, as well as meeting the needs of all patients with skin diseases over the course of their lives.

Also, in June 2017, Mayne Pharma entered into a global licensing agreement with Nestlé Skin Health (parent entity of leading global dermatology and skin health franchise, Galderma) to develop and commercialize a new chemical entity, Trifarotene, in rare disease indications.

KB105: Krystal Biotech

Krystal Biotech is investigating KB105, which is an HSV-1 based gene therapy engineered to deliver a human transglutaminase-1 (TGM1) gene to patients with TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI).

KB105 works by entering permeabilized skin and by transducing keratinocytes (native TGM1- producing cells). After that, it gets transported episomally, into the nucleus of transduced cells and the vector genome is deposited. Now, TGM1 transcripts are generated, which allows the cell to produce functional TGM1 protein that localizes to the cell membrane. TGM1 crosslinks target proteins to aid in the formation of the cornified cell envelope. This layer formed is known as the Stratum Corneum which acts as a mechanical barrier to protect against transepidermal water loss (TEWL) and entry of infectious agents. It is important to mention that, in vitro and in vivo proof-of-concept and safety had established that KB105 efficiently transduces patient keratinocytes to express functional human TGM1.

In October 2019, the US FDA granted Fast Track designation to KB105 in patients with TGM1 deficient ARCI. Additionally, in the same month, the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) also granted orphan drug designation to KB105.

Before this, in August 2018, the US FDA had also granted a rare Pediatric Disease Designation to KB105 for the treatment of patients with TGM-1-deficient Autosomal Recessive Congenital Ichthyosis (HCPLive, 2018).

Lamellar Ichthyosis Market Outlook:

As of now, there is no therapy approved by the regulatory authorities for the treatment of this rare condition. The treatment of Lamellar Ichthyosis comprises of mainly oral retinoids and a wide variety of creams containing hydrating and keratolytic agents such as urea, propylene glycol, salicylic acid, and alpha-hydroxy acids.

Topical or oral retinoids are mainly used in the severe forms of Lamellar Ichthyosis but the use of retinoids is limited due to their side effects and expense. The commonly used retinoids are isotretinoin and acitretin. Although these agents are very effective in removing scale but they can cause side effects like elevated blood fats, and calcification of tendons and ligaments. The decision to treat with systemic retinoids requires consultation with a physician experienced in their use for ichthyosis.

Furthermore, commonly used keratolytics include salicylic acid, urea, and alpha-hydroxy acids such as lactic and glycolic acids; these are contained in many over the counter and prescription lotions, creams, and ointments. However, some keratolytics such as salicylic acid can be absorbed through the skin with potentially harmful side effects on the body. Side effect, in addition to some concerns about potential toxicity, limits their usefulness in infants and pre-school children.

Key Findings:

According to the publisher, Lamellar Ichthyosis market in the 7MM is expected to change in the study period 2017–2030. The therapeutic market of Lamellar Ichthyosis in seven major markets was found to be USD 3.9 million in 2017 which is expected to increase during the study period (2017–2030).

The United States Market Outlook:

In 2017, the total market size of Lamellar Ichthyosis therapies was estimated to be USD 1.9 million in the United States which is expected to increase in the study period (2017–2030).

EU-5 Countries: Market Outlook:

In 2017, the total market size of Lamellar Ichthyosis therapies was found to be USD 1.4 million in the EU-5 countries which is expected to increase in the study period (2017–2030).

Japan Market Outlook:

The total market size of Lamellar Ichthyosis therapies in Japan was found to be USD 0.5 million in 2017 which is also expected to increase during the study period (2017–2030).

Lamellar Ichthyosis Pipeline Development Activities:

The drugs which are in pipeline includes:

1. TMB-001 (Isotretinoin) (Timber Pharmaceuticals): Phase II
2. Trifarotene (CD5789) (Galderma/Mayne Pharma): Phase II
3. KB105 (Krystal Biotech): Phase I/II

Pipeline Development Activities

Key Points

1. In September 2018, the US FDA awarded USD 1.5 million to support phase IIa and Phase IIb clinical trials evaluating TMB-001 through its Orphan Products Grant program.
2. Timber initiated a Phase IIb randomized, parallel, double-blind, vehicle-controlled study in December 2019. The purpose of this study is to investigate the efficacy and safety of two concentrations of topically applied TMB-001 in subjects nine years of age and older. In order to generate high quality data that will allow the appropriate Phase III dose selection in the ongoing Phase IIb dose ranging study, Timber is working with leading experts in the field.
3. In June 2019, Krystal Biotech submitted an IND application with the US FDA to initiate a Phase I/II of KB105.

Lamellar Ichthyosis Drugs Uptake:

Due to in vitro and in vivo proof-of-concept and safety which showed that KB105 (gene therapy) efficiently transduces patient keratinocytes to express functional human TGM1 and designations like fast track, rare pediatric disease and orphan designation KB105 is expected to impact the therapeutic market of Lamellar Ichthyosis significantly also Trifarotene (CD5789 Cream) owing to more specific mechanism as claimed by company, is expected to have significant impact on the market

Access and Reimbursement Scenario in Lamellar Ichthyosis Therapies

The access and reimbursement of the therapies for Lamellar Ichthyosis are difficult as current agents are associated with side effects and cost-effectiveness is also a major concern. Additionally, these agents are not approved by the US FDA which is making their access and reimbursement even more difficult.

Other barriers like conducting the clinical trials that would be needed to support US FDA approval of retinoids for ichthyoses. Because of these barriers, the present therapies are usually not reimbursed by insurance carriers and therapies can result in significant out-of-pocket expenses for families.

KOL - Views

To keep up with current market trends, the publisher takes KOL’s and SME’s opinion working in Lamellar Ichthyosis domain through primary research to fill the data gaps and validate the secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or Lamellar Ichthyosis 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 Lamellar Ichthyosis 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 Lamellar Ichthyosis, explaining its causes, signs and symptoms, pathophysiology and currently available therapies
  • Comprehensive insight has been provided into the Lamellar Ichthyosis epidemiology and treatment in the 7MM
  • Additionally, an all-inclusive account of both the current and emerging therapies for Lamellar Ichthyosis are provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape
  • A detailed review of Lamellar Ichthyosis 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 Lamellar Ichthyosis market
Report Highlights
  • In the coming years, Lamellar Ichthyosis 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 Lamellar Ichthyosis 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 Lamellar Ichthyosis. Launch of emerging therapies, will significantly impact the Lamellar Ichthyosis market
  • A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for Lamellar Ichthyosis.
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