Lamellar Ichthyosis (LI) Industry Outlook, 2030 Featuring Emerging Drugs TMB-001, Trifarotene (CD5789), and KB105
DUBLIN, March 23, 2020 /PRNewswire/ -- The "Lamellar Ichthyosis (LI) - Market Insights, Epidemiology and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.
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This 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.
Study Period: 2017-2030
Lamellar Ichthyosis Epidemiology
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 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.
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 Nestl 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
The 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).
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.
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.
For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/x77hfx
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