Crigler-Najjar Syndrome Market

Market Overview:

The 7 major crigler-najjar syndrome markets reached a value of USD 755.9 Million in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 1,724.6 Million by 2035, exhibiting a growth rate (CAGR) of 7.82% during 2025-2035.

The crigler-najjar syndrome market has been comprehensively analyzed in IMARC's new report titled "Crigler-Najjar Syndrome Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2025-2035". Crigler-Najjar Syndrome (CNS) is an uncommon inherited metabolic disorder that is caused by jaundice and results from deficiencies in bilirubin metabolism. It occurs in two forms: Type I, which is a more serious type with no enzyme activity, and Type II, which possesses reduced enzyme function. The disorder occurs due to genetic mutations of the UGT1A1 gene, causing interruptions in the process of processing bilirubin within the liver and its accumulation within the bloodstream. If not treated, the condition can result in kernicterus, a potentially fatal neurological disorder. Crigler-Najjar Syndrome patients suffer from chronic jaundice, lethargy, feeding difficulties, and possible neurotoxicity, especially in Type I. Diagnostic examination usually includes blood work showing elevated unconjugated bilirubin levels, genetic analysis for UGT1A1 mutation, and liver enzyme function testing. Liver biopsy and imaging tests are sometimes performed to exclude other liver abnormalities.

The Crigler-Najjar Syndrome market is being driven by the progress in diagnostic methods, heightened awareness of uncommon genetic disorders, and continued research on new therapies. The increasing incidence of inherited metabolic diseases and the widening newborn screening programs have contributed to significant improvements in early diagnosis, facilitating early interventions. Furthermore, the growing use of phototherapy and liver transplantation as common treatments, especially for Type I cases, is promoting market growth. Advances in gene therapy and enzyme replacement approaches are revolutionizing the treatment paradigm. New adenovirus-mediated UGT1A1 gene therapy has been promising as a definitive cure, treating the very cause of the disorder. Additionally, heme oxygenase inhibitors are also being investigated to decrease bilirubin levels, offering new therapeutic avenues. The robust encouragement of regulatory bodies for the development of orphan drugs, coupled with increased investments in rare disease research, is further driving market growth. Moreover, the increase in biopharmaceutical partnerships to create targeted therapies is creating new opportunities. With continuous clinical trials and rising awareness, the market is expected to grow steadily in the next few years.

IMARC Group's new report provides an exhaustive analysis of the crigler-najjar 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. 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 crigler-najjar syndrome market in any manner.

Recent Developments:

• In December 2024, Genethon and Hansa Biopharma initiated a Phase 2 trial combining imlifidase and GNT-0003 gene therapy to enable treatment for Crigler-Najjar patients with pre-existing antibodies against AAV vectors.
• In January 2023, the New England Journal of Medicine published positive Phase 1–2 trial results showing GNT-0003 gene therapy significantly reduced bilirubin levels, allowing patients to discontinue phototherapy for extended periods.

Key Highlights:

• CNS causes high unconjugated bilirubin due to UGT enzyme deficiency, leading to persistent jaundice.
• Type 1 is severe with no enzyme activity; Type 2 is milder with partial enzyme functionza.
• CNS follows an autosomal recessive pattern, requiring mutations in both UGT1A1 gene copies to manifest.
• Management includes phototherapy, while liver transplantation is a potential cure, especially for severe Type 1 cases.
• Proper treatment allows normal lifespan, but untreated Type 1 cases risk severe neurological damage and early mortality.

Drugs:

Imlifidase, developed by Hansa Biopharma, is an IgG-cleaving enzyme being explored for Crigler-Najjar syndrome. In partnership with Genethon, it is evaluated as a pre-treatment to GNT-0003 gene therapy, aiming to improve therapeutic outcomes in patients with pre-existing anti-AAV8 antibodies by enhancing gene therapy efficacy.

GNT-0003, developed by Genethon, is a gene therapy designed for Crigler-Najjar syndrome, a rare liver disorder caused by UGT1A1 deficiency. Using an AAV vector, it delivers a functional UGT1A1 gene to restore bilirubin metabolism, potentially reducing reliance on phototherapy. Clinical trials are assessing its safety and efficacy.

HepaStem, developed by Cellaion, is a mesenchymal stem cell therapy in development for Crigler-Najjar Syndrome. It utilizes liver-derived progenitor cells to support liver function and enhance bilirubin metabolism. Designed to address the underlying metabolic deficiency, HepaStem is being evaluated in clinical trials for its safety and therapeutic potential.

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

Competitive Landscape:

This report also provides a detailed analysis of the current crigler-najjar 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 crigler-najjar syndrome 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 crigler-najjar syndrome market across the seven major markets in 2024 and what will it look like in 2035?
• What is the growth rate of the crigler-najjar 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 (​2019-2035​) of crigler-najjar syndrome across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of crigler-najjar syndrome by age across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of crigler-najjar syndrome by gender across the seven major markets?
• How many patients are diagnosed (​2019-2035​) with crigler-najjar syndrome across the seven major markets?
• What is the size of the crigler-najjar syndrome 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 crigler-najjar syndrome?
• What will be the growth rate of patients across the seven major markets?

Crigler-Najjar 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 crigler-najjar 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 crigler-najjar syndrome market?
• What are the key regulatory events related to the crigler-najjar syndrome market?
• What is the structure of clinical trial landscape by status related to the crigler-najjar syndrome market?
• What is the structure of clinical trial landscape by phase related to the crigler-najjar syndrome market?
• What is the structure of clinical trial landscape by route of administration related to the crigler-najjar syndrome market?