Lipoid Congenital Adrenal Hyperplasia Market

Market Overview:

The 7 major lipoid congenital adrenal hyperplasia markets reached a value of USD 11.4 Million in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 87.7 Million by 2035, exhibiting a growth rate (CAGR) of 20.42% during 2025-2035.

The lipoid congenital adrenal hyperplasia market has been comprehensively analyzed in IMARC's new report titled "Lipoid Congenital Adrenal Hyperplasia Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2025-2035". Lipoid congenital adrenal hyperplasia (LCAH) is an infrequent and serious disorder of adrenal and gonadal steroidogenesis due to mutations in the STAR (steroidogenic acute regulatory) gene. It results in impairment of cholesterol transport and biosynthesis of steroid hormones, causing adrenal insufficiency and abnormal sexual development. LCAH generally presents in infancy with signs like failure to thrive, salt wasting, dehydration, and potentially lethal adrenal crises. In genetically male patients (46,XY), the disorder may produce female external genitalia by disrupting testosterone formation. Diagnosis of LCAH combines clinical examination, biochemical and genetic studies, and genetic diagnosis. Laboratory testing detects significantly depressed cortisol and aldosterone levels, elevated ACTH and renin activity, and compromised sex steroid formation. Confirmation of STAR mutation differentiates LCAH from other types of congenital adrenal hyperplasia through genetic examination. Imaging tests can help assess adrenal gland abnormalities and gonadal maturation.

The increasing awareness of lipoid congenital adrenal hyperplasia (LCAH) as a result of advances in genetic screening programs is one of the major drivers of the market. Early detection through expanded newborn screening programs enables early intervention and lowers the threat of adrenal crises and death. Further, advances in molecular diagnostic methods, including next-generation sequencing (NGS), have improved the sensitivity of detecting STAR gene mutations and thereby disease characterization and personalized therapeutic approaches. Enhanced application of glucocorticoid and mineralocorticoid replacement therapies continues to be the hallmark of LCAH management with the assurance of hormonal balance and the avoidance of life-threatening consequences. The ease of availability of hydrocortisone preparations optimized for children and neonates has enhanced outcomes. In addition, studies of new therapeutic modalities, such as gene therapy and stem cell-based therapy, are opening new avenues for correcting steroidogenic defects at the molecular level. Drug manufacturers are increasingly investigating targeted treatments, like modified steroid precursors and enzyme modulators, to treat underlying metabolic derangement. As knowledge about LCAH increases, investment in rare disease research and better healthcare infrastructure should propel further market growth.

IMARC Group's new report provides an exhaustive analysis of the lipoid congenital adrenal hyperplasia 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, Japan and Korea have the largest patient pool for lipoid congenital adrenal hyperplasia 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 lipoid congenital adrenal hyperplasia market in any manner.

Recent Developments:

• In December 2024, the U.S. Food and Drug Administration (FDA) approved crinecerfont, which is marketed as Crenessity, to be used along with glucocorticoids in patients 4 years and older with classic CAH. Crinecerfont is a first-in-class oral corticotropin-releasing factor type 1 receptor antagonist that minimizes excess adrenal androgen production, thus making it possible to use lower glucocorticoid dosages.

Key Highlights:

• The exact global prevalence of LCAH is unknown due to its rarity. However, it is considered one of the least common forms of CAH.
• LCAH appears to be more prevalent in certain populations, particularly among individuals of Japanese, Korean, and Palestinian descent. This increased prevalence is often attributed to founder mutations within these communities.
• Due to its rarity, specific incidence rates for LCAH are not well-documented. The condition is less common than other forms of CAH, such as 21-hydroxylase deficiency, which has an incidence ranging from 1 in 5,000 to 1 in 15,000 live births.
• Historically, LCAH was often fatal in infancy if left untreated. With early diagnosis and appropriate management, including hormone replacement therapy, individuals with LCAH can now survive into adulthood.
• LCAH is caused by mutations in the StAR gene, which is crucial for cholesterol transport within adrenal cells. These mutations lead to a severe deficiency in all steroid hormones, resulting in the clinical manifestations of the disorder.

Drugs:

Hydrocortisone is a crucial corticosteroid for managing lipoid congenital adrenal hyperplasia (LCAH), a rare and severe form of adrenal insufficiency. It replaces deficient cortisol, preventing adrenal crises and improving metabolic stability. Early and lifelong hydrocortisone therapy is essential to regulate stress response, immune function, and electrolyte balance in affected individuals.

Fludrocortisone is a synthetic corticosteroid used to manage Lipoid Congenital Adrenal Hyperplasia by mimicking aldosterone, aiding sodium retention and potassium excretion. It helps regulate electrolyte balance and blood pressure in patients with adrenal insufficiency. This therapy is crucial in preventing dehydration, hypotension, and life-threatening imbalances in affected individuals.

Time Period of the Study

• Base Year: 2024
• Historical Period: 2019-2024
• Market Forecast: 2025-2035

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 lipoid congenital adrenal hyperplasia market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the lipoid congenital adrenal hyperplasia market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current lipoid congenital adrenal hyperplasia 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 lipoid congenital adrenal hyperplasia market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2024 and how are they expected to perform till 2035?
• What was the country-wise size of the lipoid congenital adrenal hyperplasia market across the seven major markets in 2024 and what will it look like in 2035?
• What is the growth rate of the lipoid congenital adrenal hyperplasia 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 (​2019-2035) of lipoid congenital adrenal hyperplasia across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of lipoid congenital adrenal hyperplasia by age across the seven major markets?
• What is the number of prevalent cases (​2019-2035) of lipoid congenital adrenal hyperplasia by gender across the seven major markets?
• How many patients are diagnosed (​2019-2035​) with lipoid congenital adrenal hyperplasia across the seven major markets?
• What is the size of the lipoid congenital adrenal hyperplasia patient pool (2019-2024) across the seven major markets?
• What would be the forecasted patient pool (2025-2035) across the seven major markets?
• What are the key factors driving the epidemiological trend of lipoid congenital adrenal hyperplasia?
• What will be the growth rate of patients across the seven major markets?

Lipoid Congenital Adrenal Hyperplasia: 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 lipoid congenital adrenal hyperplasia 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 lipoid congenital adrenal hyperplasia market?
• What are the key regulatory events related to the lipoid congenital adrenal hyperplasia market?
• What is the structure of clinical trial landscape by status related to the lipoid congenital adrenal hyperplasia market?
• What is the structure of clinical trial landscape by phase related to the lipoid congenital adrenal hyperplasia market?
• What is the structure of clinical trial landscape by route of administration related to the lipoid congenital adrenal hyperplasia market?