Gorlin Syndrome Market

 Market Overview:

The 7 major Gorlin syndrome markets reached a value of US$ 5.4 Billion in 2023. Looking forward, IMARC Group expects the 7MM to reach US$ 12.1 Billion by 2034, exhibiting a growth rate (CAGR) of 7.6% during 2024-2034.

The Gorlin syndrome market has been comprehensively analyzed in IMARC's new report titled "Gorlin Syndrome Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Gorlin syndrome, also referred to as nevoid basal cell carcinoma syndrome (NBCCS), is a rare genetic disorder characterized by the presence of skeletal, ophthalmologic, and neurologic abnormalities. The disease is inherited in an autosomal dominant manner that primarily affects the skin, bones, and other systems of the body. Individuals suffering from this illness might have numerous basal cell nevi, which are benign skin growths resembling small, flesh-colored, or brownish bumps. Some of the common symptoms of the ailment include malformations of the skull and facial bones, deformities of the spine, missing or extra teeth, jaw cysts, pigmented lesions on the retina, mild intellectual disability or developmental delays, etc. Diagnosing Gorlin syndrome typically requires a combination of the patient's clinical evaluation, medical history, and physical examination. The healthcare provider may also perform blood workups to identify the specific gene mutations associated with the illness. Numerous diagnostic procedures, such as ultrasounds and magnetic resonance imaging, are recommended to check for the indications of this ailment and rule out other possible causes.

The increasing cases of genetic mutations, which disrupt the normal functioning of the signaling pathway involved in embryonic development and tissue maintenance, are primarily driving the Gorlin syndrome market. Besides this, the widespread adoption of hedgehog pathway inhibitors, including vismodegib and sonidegib, to help in slowing down the spread of unhealthy cells, thereby preventing disease progression, is acting as another significant growth-inducing factor. Furthermore, the escalating usage of photodynamic therapy, which involves the application of topical photosensitizing agents to the skin followed by exposure to light of a specific wavelength to produce reactive oxygen species that disrupt targeted abnormal tissues, is also bolstering the market growth. Additionally, the emerging popularity of topical medications, such as imiquimod and 5-fluorouracil cream, for treating superficial lesions that are small in size and located in low-risk areas is creating a positive outlook for the market. Moreover, the inflating demand for Mohs micrographic surgery, owing to its various advantages, like maximal tissue conservation, high cure rates, better treatment outcomes for patients, etc., is expected to drive the Gorlin syndrome market in the coming years. 

IMARC Group's new report provides an exhaustive analysis of the Gorlin syndrome market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report, the United States has the largest patient pool for Gorlin syndrome and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the Gorlin syndrome market in any manner.

Recent Developments:

• In April 2024, DermBiont published beneficial preliminary results from its current open label, multi-center Phase 2a research for the treatment of basal cell carcinoma (BCC). As a result, DermBiont has reprioritized SM-020, a first-in-class, patient-applied, topical kinase inhibitor, for the development of two lead orphan disease oncology indications: locally advanced basal cell carcinoma (laBCC) treatment and BCC prevention in Gorlin Syndrome patients.
• In November 2023, Sol-Gel Technologies reported that it had commenced Phase 3 testing of SGT-610 (patidegib gel, 2%) for Gorlin syndrome, with the first patient screened. SGT-610 is a novel topical hedgehog inhibitor being developed to prevent new basal cell carcinoma lesions in Gorlin syndrome patients. It is predicted to have a better safety profile than oral hedgehog inhibitors.

Key Highlights:

• The estimated prevalence of Gorlin syndrome ranges from 1/30,827 to 1/256,000, with a minimum of 1/57,000.
• In Europe, the prevalence is projected to be between 1/31,000 and 164,000.
• Gorlin syndrome is estimated to affect 1 in 31,000 people in the United States.
• The condition affects males and women roughly equally (1:1.3).
• Gorlin syndrome can result in premature death in 10% of cases.

Drugs:

PTX-022 is a unique topical formulation of the mTOR inhibitor rapamycin based on Palvella's patented and patent-pending QTORIN formulation and delivery technology. QTORIN uses a highly tailored excipient composition to distribute mTOR inhibitors into basal keratinocytes that have mutant keratin genes.

SGT-610, a topical inhibitor of the hedgehog signaling system, has the potential to be the first preventive treatment for Gorlin syndrome if authorized. Patidegib, the active ingredient in SGT-610, is intended to block the smoothened, frizzle class receptor (SMO) signal, allowing cells to operate properly while decreasing the creation of new tumors.

Vismodegib (Erivedge) is a prescription medication that helps cure basal cell carcinomas in Gorlin syndrome patients. Vismodegib, an oncogene inhibitor, acts by disrupting the aberrant Hedgehog cell signaling system, which is responsible for tumor formation in most basal cell carcinomas.

Time Period of the Study

• Base Year: 2023
• Historical Period: 2018-2023
• Market Forecast: 2024-2034
   

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan
   

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the Gorlin syndrome market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the Gorlin syndrome market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current Gorlin syndrome marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the Gorlin syndrome market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
• What was the country-wise size of the Gorlin syndrome market across the seven major markets in 2023 and what will it look like in 2034?
• What is the growth rate of the Gorlin syndrome market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?
   

Epidemiology Insights

• What is the number of prevalent cases (2018-2034) of Gorlin syndrome across the seven major markets?
• What is the number of prevalent cases (2018-2034) of Gorlin syndrome by age across the seven major markets?
• What is the number of prevalent cases (2018-2034) of Gorlin syndrome by gender across the seven major markets?
• How many patients are diagnosed (2018-2034) with Gorlin syndrome across the seven major markets?
• What is the size of the Gorlin syndrome patient pool (2018-2023) across the seven major markets?
• What would be the forecasted patient pool (2024-2034) across the seven major markets?
• What are the key factors driving the epidemiological trend of Gorlin syndrome?
• What will be the growth rate of patients across the seven major markets?
   

Gorlin Syndrome: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for Gorlin syndrome drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the Gorlin syndrome market?
• What are the key regulatory events related to the Gorlin syndrome market?
• What is the structure of clinical trial landscape by status related to the Gorlin syndrome market?
• What is the structure of clinical trial landscape by phase related to the Gorlin syndrome market?
• What is the structure of clinical trial landscape by route of administration related to the Gorlin syndrome market?