Report Code : CVMI2603252601399 | Published Date : March 30, 2025

1 | Market Overview

The global carglumic acid market is experiencing steady growth, driven by the increasing prevalence of hyperammonemia disorders and greater awareness of orphan diseases. Carglumic acid is mainly used to treat N-acetylglutamate synthase (NAGS) deficiency, a rare urea cycle disorder that causes excessive ammonia buildup in the blood.

The market is benefiting from improved newborn screening programs, better diagnostic capabilities, and expanding healthcare infrastructure in emerging economies. Supportive regulatory pathways for orphan drugs and increased R&D investments are further speeding up market adoption.

 

2 | Market Size and Forecast

Year

Market Value (USD Billion)

Notes

2019

0.42

Historical benchmark

2024

0.61

Five-year CAGR 7.8%

2031

1.19

Seven-year CAGR 9.7%

The market is projected to almost double by 2031, driven by increased access to genetic testing and growing demand for rare disease treatments.

 

3 | Key Market Drivers

  • Rising Diagnosis of Urea Cycle Disorders: Enhanced screening protocols globally.
  • Favorable Orphan Drug Policies: Fast-track approvals and tax incentives for rare disease treatments.
  • Increasing R&D in Metabolic Disorders: Pipeline expansion for genetic and enzymatic therapies.
  • Growing Patient Awareness: Support from advocacy groups and healthcare organizations.

 

4 | Market Challenges

  • High Treatment Cost: Carglumic acid therapy is expensive and not accessible in all markets.
  • Limited Patient Pool: Small incidence rates of NAGS deficiency restrict commercial scaling.
  • Dependence on Single Product Players: Market concentration increases supply chain risks.

5 | Competitive Landscape
The carglumic acid market is highly consolidated, mainly controlled by one major player with worldwide regulatory approvals.

Market Share Estimates (2024):

Company

Estimated Market Share

Key Product

Recordati Rare Diseases

87%

Carbaglu® (carglumic acid)

Others (Compounding Pharmacies, Generics in Development)

13%

Pipeline and early market entrants

 

6 | Market Segmentation
By Application:

  • NAGS Deficiency
  • Hyperammonemia Management in Organic Acidemias
  • Other Urea Cycle Disorders (off-label)

 

By Distribution Channel:

  • Hospital Pharmacies
  • Specialty Pharmacies
  • Online Pharmacies

 

By Region:

  • North America
  • Europe
  • Asia-Pacific
  • Latin America
  • Middle East & Africa

 

7 | Strategic Outlook
Future growth will depend on label expansion, the availability of generics, and collaborative research in metabolic genetics. Emerging markets will be crucial as newborn screening and diagnostic networks grow





Reasons To Buy

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Scope

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Key Players

  • Orphan Europe (Recordati)
  • Dipharma
  • Civentichem
  • Manus Aktteva Biopharma
  • Suven Life Sciences
  • Taj Pharmaceuticals

Carglumic Acid Market Report


  1. 1. Carglumic Acid Market Research Report
    1. 1.1 Study Objectives
    2. 1.2 Carglumic Acid Market - Overview
    3. 1.3 Reason to Read This Report
    4. 1.4 Methodology and Forecast Analysis
  2. 2. Carglumic Acid Market Research Report - Preface
    1. 2.1 Carglumic Acid Market Research Report – Detailed Scope and Definitions
      1. 2.1.1 By Application:
      2. 2.1.2 By Distribution Channel:
      3. 2.1.3 By Region
  3. 3. Carglumic Acid Market Dynamics
    1. 3.1. Drivers - Macro-Economic Based, Supply Side, and Demand Side Drivers
    2. 3.2. Restraints – By Application:, By Distribution Channel:, By Country
    3. 3.3. Opportunities – By Application:, By Distribution Channel:, By Country
    4. 3.4. Trends – By Application:, By Distribution Channel:, By Country
    5. 3.5. PEST Analysis
    6. 3.6. Porters Five Rule Analysis
    7. 3.7. Company’s Share Analysis (CSA) by Region or By Country
    8. 3.8. Carglumic Acid Market Research Report – DROTs Impact Analysis
    1. 4. Carglumic Acid Market Research Report, Historic Data 2019 - 2023 and Forecast Analysis Data 2024 - 2031
    2. 4.1. Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
    3. 4.2. Annual Market Trend Assessment – Year-on-Year (YoY) Growth Analysis (%)
    4. 4.3. Incremental Market Value/Volume Opportunity between 2019 - 2023 and 2024 - 2031
    5. 4.4. Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
  1. 5. Carglumic Acid Market, By Application:, 2019 - 2023 and Forecast, 2024 - 2031 (Market Value, In USD Mn)
    1. 5.1 NAGS Deficiency
    2. 5.1.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
    3. 5.1.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
    4. 5.1.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
    5. 5.1.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    6. 5.2 Hyperammonemia Management in Organic Acidemias
    7. 5.2.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
    8. 5.2.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
    9. 5.2.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
    10. 5.2.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    11. 5.3 Other Urea Cycle Disorders (off-label)
    12. 5.3.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
    13. 5.3.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
    14. 5.3.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
    15. 5.3.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    6. Carglumic Acid Market, By Distribution Channel:, 2019 - 2023 and Forecast, 2024 - 2031 (Market Value, In USD Mn)
    1. 6.1 Hospital Pharmacies
    2. 6.1.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
    3. 6.1.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
    4. 6.1.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
    5. 6.1.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    6. 6.2 Specialty Pharmacies
    7. 6.2.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
    8. 6.2.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
    9. 6.2.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
    10. 6.2.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    11. 6.3 Online Pharmacies
    12. 6.3.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
    13. 6.3.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
    14. 6.3.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
    15. 6.3.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
  1. 7. Carglumic Acid Market Forecast, By Region, 2019 - 2023 and 2024 - 2031 (Market Value, In USD Mn)
    1. 7.1 North America
      1. 7.1.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 7.1.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 7.1.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 7.1.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
      7.2 Europe
      1. 7.2.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 7.2.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 7.2.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 7.2.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
      7.3 Asia-Pacific
      1. 7.3.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 7.3.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 7.3.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 7.3.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
      7.4 Latin America
      1. 7.4.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 7.4.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 7.4.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 7.4.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
      7.5 Middle East and Africa
      1. 7.5.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 7.5.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 7.5.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 7.5.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
      7.6 Carglumic Acid Market - Opportunity Analysis Index, By Application:, By Distribution Channel:, and Region, 2024 - 2031
  1. 8. North America Carglumic Acid Market Analysis, 2019 - 2023 and Forecast, 2024 - 2031 (Market Value, In USD Mn)
    1. 8.1 By Application: Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 8.1.1 NAGS Deficiency
      2. 8.1.2 Hyperammonemia Management in Organic Acidemias
      3. 8.1.3 Other Urea Cycle Disorders (off-label)
      8.2 By Distribution Channel: Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 8.2.1 Hospital Pharmacies
      2. 8.2.2 Specialty Pharmacies
      3. 8.2.3 Online Pharmacies
      8.3 Country Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 8.3.1 United States
      2. 8.3.2 Canada
    2. 8.4 North America Carglumic Acid Market – Opportunity Analysis Index, By Application:, By Distribution Channel:, and Country, 2024 - 2031
    3. 8.5 Regional Trends Analysis
    4. 8.6 North America Carglumic Acid Market Research Report - Company Profiles
      1. 8.6.1 Company 1 (United States)
      2. 8.6.2 Company 2 (Canada)
      3. 8.6.3 Company 3 (Canada)
    9. Europe Carglumic Acid Market Analysis, 2019 - 2023 and Forecast, 2024 - 2031 (Market Value, In USD Mn)
    1. 9.1 By Application: Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 9.1.1 NAGS Deficiency
      2. 9.1.2 Hyperammonemia Management in Organic Acidemias
      3. 9.1.3 Other Urea Cycle Disorders (off-label)
      9.2 By Distribution Channel: Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 9.2.1 Hospital Pharmacies
      2. 9.2.2 Specialty Pharmacies
      3. 9.2.3 Online Pharmacies
      9.3 Country Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 9.3.1 Germany
      2. 9.3.2 United Kingdom
      3. 9.3.3 France
      4. 9.3.4 Spain
      5. 9.3.5 Italy
      6. 9.3.6 Russia
    2. 9.4 Europe Carglumic Acid Market – Opportunity Analysis Index, By Application:, By Distribution Channel:, and Country, 2024 - 2031
    3. 9.5 Regional Trends Analysis
    4. 9.6 Europe Carglumic Acid Market Research Report - Company Profiles
      1. 9.6.1 Company 1 (Germany)
      2. 9.6.2 Company 2 (United Kingdom)
      3. 9.6.3 Company 3 (United Kingdom)
    10. Asia-Pacific Carglumic Acid Market Analysis, 2019 - 2023 and Forecast, 2024 - 2031 (Market Value, In USD Mn)
    1. 10.1 By Application: Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 10.1.1 NAGS Deficiency
      2. 10.1.2 Hyperammonemia Management in Organic Acidemias
      3. 10.1.3 Other Urea Cycle Disorders (off-label)
      10.2 By Distribution Channel: Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 10.2.1 Hospital Pharmacies
      2. 10.2.2 Specialty Pharmacies
      3. 10.2.3 Online Pharmacies
      10.3 Country Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 10.3.1 China
      2. 10.3.2 Japan
      3. 10.3.3 India
      4. 10.3.4 South Korea
      5. 10.3.5 Australia & New Zealand
      6. 10.3.6 ASEAN
    2. 10.4 Asia-Pacific Carglumic Acid Market – Opportunity Analysis Index, By Application:, By Distribution Channel:, and Country, 2024 - 2031
    3. 10.5 Regional Trends Analysis
    4. 10.6 Asia-Pacific Carglumic Acid Market Research Report - Company Profiles
      1. 10.6.1 Company 1 (China)
      2. 10.6.2 Company 2 (Japan)
      3. 10.6.3 Company 3 (Japan)
    11. Latin America Carglumic Acid Market Analysis, 2019 - 2023 and Forecast, 2024 - 2031 (Market Value, In USD Mn)
    1. 11.1 By Application: Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 11.1.1 NAGS Deficiency
      2. 11.1.2 Hyperammonemia Management in Organic Acidemias
      3. 11.1.3 Other Urea Cycle Disorders (off-label)
      11.2 By Distribution Channel: Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 11.2.1 Hospital Pharmacies
      2. 11.2.2 Specialty Pharmacies
      3. 11.2.3 Online Pharmacies
      11.3 Country Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 11.3.1 Brazil
      2. 11.3.2 Mexico
      3. 11.3.3 Argentina
      4. 11.3.4 Peru
      5. 11.3.5 Colombia
      6. 11.3.6 Rest of Latin America
    2. 11.4 Latin America Carglumic Acid Market – Opportunity Analysis Index, By Application:, By Distribution Channel:, and Country, 2024 - 2031
    3. 11.5 Regional Trends Analysis
    4. 11.6 Latin America Carglumic Acid Market Research Report - Company Profiles
      1. 11.6.1 Company 1 (Brazil)
      2. 11.6.2 Company 2 (Mexico)
      3. 11.6.3 Company 3 (Mexico)
    12. Middle East and Africa Carglumic Acid Market Analysis, 2019 - 2023 and Forecast, 2024 - 2031 (Market Value, In USD Mn)
    1. 12.1 By Application: Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 12.1.1 NAGS Deficiency
      2. 12.1.2 Hyperammonemia Management in Organic Acidemias
      3. 12.1.3 Other Urea Cycle Disorders (off-label)
      12.2 By Distribution Channel: Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 12.2.1 Hospital Pharmacies
      2. 12.2.2 Specialty Pharmacies
      3. 12.2.3 Online Pharmacies
      12.3 Country Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 12.3.1 Saudi Arabia
      2. 12.3.2 UAE
      3. 12.3.3 South Africa
      4. 12.3.4 Egypt
      5. 12.3.5 Israel
      6. 12.3.6 Rest of Middle East and Africa
    2. 12.4 Middle East and Africa Carglumic Acid Market – Opportunity Analysis Index, By Application:, By Distribution Channel:, and Country, 2024 - 2031
    3. 12.5 Regional Trends Analysis
    4. 12.6 Middle East and Africa Carglumic Acid Market Research Report - Company Profiles
      1. 12.6.1 Company 1 (Saudi Arabia)
      2. 12.6.2 Company 2 (UAE)
      3. 12.6.3 Company 3 (UAE)
    13. Competition Landscape
    1. 13.1 Strategic Dashboard of Top Market Players
    2. 13.2 Company Profiles (Introduction, Financial Assessments, Portfolio of Offerings, Milestones and Achievements, Strategic Initiative, and SWOT Analysis)
      1. 13.2.1 Recordati Rare Diseases Inc
      2. 13.2.2 Dipharma Francis S.r.l
      3. 13.2.3 Biophore India Pharmaceuticals Pvt Ltd
      4. 13.2.4 Torrent Pharmaceuticals Ltd
      5. 13.2.5 Zydus Lifesciences Ltd
      6. 13.2.6 Civentichem LLC
      7. 13.2.7 Manus Aktteva Biopharma LLP
      8. 13.2.8 Apothecon Pharmaceuticals Pvt Ltd
      9. 13.2.9 Eton Pharmaceuticals
      10. 13.2.10 Nuray Chemicals Private Limited
      11. 13.2.11 Novitium Pharma LLC
      12. 13.2.12 Rhyme Organics & Chemicals Ltd
      13. 13.2.13 Suven Life Sciences Ltd
      14. 13.2.14 ANI Pharmaceuticals (marketed via Eton)
      15. 13.2.15 Navinta LLC.
  1. 14. Data Collection Method and Research Approach
  2. 15. Principal Presumptions and Acronyms