Citrullinemia Type 1 Market

Market Overview:

The 7 major citrullinemia type 1 markets reached a value of USD 410.1 Million in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 585.7 Million by 2035, exhibiting a growth rate (CAGR) of 3.30% during 2025-2035.

The citrullinemia type 1 market has been comprehensively analyzed in IMARC's new report titled "Citrullinemia Type 1 Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2025-2035". Citrullinemia type 1 (CTLN1) is a rare and severe autosomal recessive hereditary urea cycle condition characterized by elevated blood citrulline levels and neurotoxic ammonia. CTLN1 commonly appears in the first few days of life. Affected newborns often experience a progressive loss of energy (lethargy), poor feeding, vomiting, convulsions, and loss of consciousness as a result of acute episodes of hyperammonemia known as hyperammonemia crises. These crises can cause hyperammonemic encephalopathy within a few hours or days of life, resulting in severe and irreparable neurological damage. Later onset (childhood or adulthood) form represents 10-20% of cases and manifests as recurrent lethargy and somnolence, intellectual disability, and chronic less severe or recurrent hyperammonemia. The disorder is primarily diagnosed based on the presence of characteristic symptoms and newborn screening programs. The healthcare professional may also perform biochemical tests, which involve measuring blood ammonia and amino acid levels with elevated citrulline and ammonia levels. Furthermore, molecular genetic testing might be recommended to identify the gene mutations and confirm the diagnosis.

The escalating prevalence of the mutations in the argininosuccinate synthase (ASS1) gene, which leads to a deficiency in the enzyme ASS, essential for processing ammonia in the urea cycle, is primarily driving the citrullinemia type 1 market. In addition to this, the inflating utilization of effective medications, such as sodium phenylbutyrate and sodium benzoate, that aid in preventing high ammonia levels in the blood is also creating a positive outlook for the market. Moreover, the widespread adoption of intravenous (IV) hydration and glucose solutions for CTLN1 is further bolstering the market growth. This intervention helps stabilize ammonia levels in patients, thereby preventing neurological damage and coma. Apart from this, the rising usage of plasma exchange procedure on account of its several benefits, like rapid reduction of ammonia levels, decreased metabolic stress, and improved quality of life, is acting as another significant growth-inducing factor. Additionally, the emerging popularity of gene therapy, since it works by correcting the faulty gene responsible for the condition, is expected to drive the citrullinemia type 1 market during the forecast period.

IMARC Group's new report provides an exhaustive analysis of the citrullinemia type 1 market in the United States, EU5 (Germany, Spain, Italy, France, and 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 citrullinemia type 1 and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and 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 citrullinemia type 1 market in any manner.

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 citrullinemia type 1 market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the citrullinemia type 1 market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current citrullinemia type 1 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

Key Questions Answered in this Report:

Market Insights

• How has the citrullinemia type 1 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 citrullinemia type 1 market across the seven major markets in 2024 and what will it look like in 2035?
• What is the growth rate of the citrullinemia type 1 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 citrullinemia type 1 across the seven major markets?
• What is the number of prevalent cases (2019-2035) of citrullinemia type 1 by age across the seven major markets?
• What is the number of prevalent cases (2019-2035) of citrullinemia type 1 by gender across the seven major markets?
• How many patients are diagnosed (2019-2035) with citrullinemia type 1 across the seven major markets?
• What is the size of the citrullinemia type 1 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 citrullinemia type 1?
• What will be the growth rate of patients across the seven major markets?

Citrullinemia Type 1: 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 citrullinemia type 1 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 citrullinemia type 1 market?
• What are the key regulatory events related to the citrullinemia type 1 market?
• What is the structure of clinical trial landscape by status related to the citrullinemia type 1 market?
• What is the structure of clinical trial landscape by phase related to the citrullinemia type 1 market?
• What is the structure of clinical trial landscape by route of administration related to the citrullinemia type 1 market?