Ornithine Transcarbamylase Deficiency Treatment Market
Ornithine Transcarbamylase Deficiency Treatment Market By Product (Buphenyl, Ravicti, Ammonul, Dietary Supplements), By Route of Administration (Oral, Intravenous), By Distribution Channel, By Region - Global Market Forecast 2020 to 2030
Analysis of Ornithine Transcarbamylase Deficiency Treatment market covering 30 + countries including analysis of US, Canada, UK, Germany, France, Nordics, GCC countries, Japan, Korea and many more
Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Overview
Ornithine transcarbamylase (OTC) deficiency is a genetic disorder arising due to inadequate levels of OTC, an enzyme that helps decompose and eliminate unwanted nitrogen from the human body. This process is also known as the urea cycle.
Deficiency of this enzyme causes excessive nitrogen accumulation, or hyperammonemia, in the blood. Another perilous effect of this genetic disorder is the secretion of elevated levels of ammonia, which is a notorious neurotoxin. Once it reaches the central nervous system, it induces symptoms such as anorexia and overall lethargy.
Fact.MR projects the global ornithine transcarbamylase (OTC) deficiency treatment market to expand steadily over the coming decade, attributed to rising prevalence of this deficiency among the world population.
OTC Deficiency Treatment Market Trends
Rising Prevalence of UCDs to Foster Therapeutic Developments in OTC Deficiency: Rising prevalence of urea cycle disorders (UCDs) is furthering substantial research in developing effective OTC deficiency treatments. UCDs are genetically inherited diseases that affect the human body’s waste removal mechanism.
Just like other organisms, human beings require proteins to survive. Proteins are important for facilitating growth and tissue repair. Excessive proteins are converted into nitrogen and expelled from the body in the form of urea via urine.
Patients afflicted with UCDs lack the catalytic enzyme required to convert excessive nitrogen into urea. This causes excessive ammonia deposition, which is highly toxic to the human body. Against this backdrop, various pharmaceutical companies are investigating possible novel approaches to address this disorder, which will bode well for the expansion of the ornithine transcarbamylase deficiency treatment market size.
Gene Therapy Touted as Possible Effective Remedy: Years of clinical trials and research within the medical fraternity have pointed towards gene therapy as a possible solution to cure or inhibit OTC deficiencies across the world. Gene therapy is designed to provide a healthy copy of the OTC gene.
In laboratory settings, a normal OTC gene is packaged into a modified adeno-associated virus (AAV) that targets liver cells. As a vector, the virus is introduced into the patient via a single intravenous infusion. The virus containing the functional gene copy enters the liver, enabling it to produce the OTC enzyme.
The Boston Children’s Hospital is making significant progress in this regard, with doctors hoping to eradicate this burden in the near future. Liver transplant is being considered as an alternative to OTC deficiency treatment by several healthcare providers, in addition to gene therapy.
Companies in the OTC deficiency treatment market are increasing their production capabilities to manufacture BUPHENYL®, a range of sodium phenyl butyrate-based tablets and powders for oral administration. Presently, the drug is being utilized as an adjunctive therapy to manage OTC deficiency.
OTC Deficiency Treatment Market: Product InsightsOTC Deficiency Treatment Market: Administration Insights
By product type, Ravicti-based OCT deficiency treatment drugs are expected to be the most promising, capturing an impressive revenue share by the end of the forecast period. Higher efficacy combined with cost effectiveness and better patient compliance are responsible for propelling the segment’s growth forward.
The main measure of effectiveness associated with Ravicti is the dramatic reduction of ammonia levels in the patient’s body. A popular study has demonstrated that, Ravicti-treated patients experienced average ammonia levels of 870 micromoles per liter. A similar pattern was observed in children treated with the drug at birth.
Trailing behind is the buphenyl segment, expected to emerge as the 2nd largest selling OTC deficiency drug category during forecast period. Growth is primarily attributed to increasing foray into the sodium phenyl butyrate drug formulation domain by prominent manufacturers.
OTC Deficiency Treatment Market: Administration Insights
Oral OTC deficiency medicine delivery is slated to remain dominant throughout the predicted forecast period, expanding at a notable CAGR. Rising preference for oral medication to eliminate trauma through external skin penetration is driving the segment’s growth in the ornithine transcarbamylase deficiency treatment market.
Simultaneously, the intravenous route of administration is gaining momentum, attributed to faster drug delivery to affected body parts, and hence, reduced length of stay for patients in hospitals and other healthcare settings.
OTC Deficiency Treatment Market: Distribution Channel Insights
With respect to distribution channel, the hospital pharmacy segment is expected to generate white spaces for the ornithine transcarbamylase deficiency treatment market. Increasing prevalence of UCDs among the pediatric and adult population are leading to a large number of hospitalizations, prompting them to stock up on adequate OTC deficiency treatment drugs.
Even after patients are discharged, they continue relying on hospital pharmacies to avail their supply of OTC deficiency management drugs. This is attributed to their high confidence and trust within hospital physicians, as they possess the relevant knowledge concerning drug dosage and possible side-effects.
Regional Outlook of OTC Deficiency Treatment Market
North America accounted for a major ornithine transcarbamylase deficiency treatment market share in 2019, owing to rise in the total number of reported cases, favorable reimbursement policies, various clinical studies for urea cycle disorders, and better screening approaches for rare diseases.
According to the U.S National Library of Medicine, the incidence of OTC deficiency ranges from 1 in 14,000 to 1 in 17,000 people across the United States. Consequently, rate of treatment for the disorder has been keeping consistent pace. This has attracted several global players to increase their investments across North America.
The ornithine transcarbamylase deficiency treatment market in Asia Pacific is projected to expand at a higher CAGR during the forecast period, owing to increase in awareness about OTC deficiency and other urea cycle disorders, surge in screening, increase in distribution tie-ups between major companies, and rise in investments by major companies in the region.
OTC Deficiency Treatment Market: Competition Analysis
Product launches, acquisitions, and strategic collaborations form a part and parcel of the aforementioned market players’ key consolidation strategies. In February 2010, Abbott Laboratories acquired Solvay S.A for US$ 6.2 billion. This lead to the company’s pharmaceutical products portfolio expansion and presence in key emerging markets.
The Analyst’s Viewpoint
“Manufacturers are filling in the void resulting due to dearth of FDA-approved OTC treatment drugs by introducing innovative and safe ingredient combination drugs in the global market, generating immense revenue pools.”
OTC Deficiency Treatment Market: About the Report
Fact.MR has published an exclusive forecast report on the ornithine transcarbamylase deficiency treatment market from 2020 to 2030. The foremost objective of this report is to pitch insights on the market scenario, demand generators, and technological advancements in the OTC deficiency treatment market.
Also, the study addresses key dynamics that are expected to diversify the sales and future prominence of OTC deficiency treatment.
The report begins with an executive overview, in which, product definition has been provided. The report further proceeds with the taxonomy of the ornithine transcarbamylase deficiency treatment market, elaborating on key segments.
Also, the report outlines visionary insights on the dynamics of the ornithine transcarbamylase deficiency treatment market, including the drivers, restraints, opportunities, trends, and pricing analysis, along with the key buying factors for OTC deficiency treatment drugs.
OTC Deficiency Treatment Market – Segmentation
Fact.MR’s research study assesses the global ornithine transcarbamylase deficiency treatment market in terms of product, route of administration, distribution channel, and region. This report presents extensive market dynamics and trends associated with different segments of the market, and their influence on the growth prospects of the global OTC deficiency treatment market.
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By Product :
- Buphenyl
- Ravicti
- Ammonul
- Dietary Supplements
- Others
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By Route of Administration :
- Oral
- Intravenous
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By Distribution Channel :
- Hospital Pharmacies
- Retail Pharmacies
- Online Pharmacies
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By Region :
- North America
- Europe
- Asia Pacific
- Latin America
- Middle East & Africa
Table of Content
- 1. Preface
- 2. Assumptions and Research Methodology
- 3. Executive Summary: Global Market
- 4. Market Overview
- 5. Key Insights
- 6. Global Market Analysis and Forecast, by Product
- 6.1. Buphenyl
- 6.2. Ravicti
- 6.3. Ammonul
- 6.4. Dietary Supplements
- 6.5. Others
- 7. Global Market Analysis and Forecast, by Route of Administration
- 7.1. Oral
- 7.2. Intravenous
- 8. Global Market Analysis and Forecast, by Distribution Channel
- 8.1. Hospital Pharmacies
- 8.2. Retail Pharmacies
- 8.3. Online Pharmacies
- 9. Global Market Analysis and Forecast, by Region
- 9.1. North America
- 9.2. Europe
- 9.3. Asia Pacific
- 9.4. Latin America
- 9.5. Middle East & Africa
- 10. North America Analysis and Forecast
- 11. Europe Market Analysis and Forecast
- 12. Asia Pacific Market Analysis and Forecast
- 13. Latin America Market Analysis and Forecast
- 14. Middle East & Africa Market Analysis and Forecast
- 15. Competition Landscape
- 15.1. Abbott
- 15.2. Nutricia (Danone Group)
- 15.3. Mead Johnson (Reckitt Benckiser)
- 15.4. Horizon Therapeutics plc
- 15.5. Nestlé
- 15.6. Bausch Health Companies, Inc.
- 15.7. Ultragenyx Pharmaceutical Inc.
- 15.8. Arcturus Therapeutics, Inc.
- 15.9. Acer Therapeutics
List Of Table
Table 02: Global Market Value (US$ Mn) Forecast, by Product, 2018 to 2030
Table 03: Global Market Value (US$ Mn) Forecast, by Route of Administration, 2018 to 2030
Table 04: Global Market Value (US$ Mn) Forecast, by Distribution Channel, 2018 to 2030
Table 05: Global Market Value (US$ Mn) Forecast, by Region, 2018 to 2030
Table 06: North America Market Value (US$ Mn) Forecast, by Country, 2018 to 2030
Table 07: North America Market Value (US$ Mn) Forecast, by Product, 2018 to 2030
Table 08: North America Market Value (US$ Mn) Forecast, by Route of Administration, 2018 to 2030
Table 09: North America Market Value (US$ Mn) Forecast, by Distribution Channel, 2018 to 2030
Table 10: Europe Market Value (US$ Mn) Forecast, by Country/Sub-region, 2018 to 2030
Table 11: Europe Market Value (US$ Mn) Forecast, by Product, 2018 to 2030
Table 12: Europe Market Value (US$ Mn) Forecast, by Route of Administration, 2018 to 2030
Table 13: Europe Market Value (US$ Mn) Forecast, by Distribution Channel, 2018 to 2030
Table 14: Asia Pacific Market Value (US$ Mn) Forecast, by Country/Sub-region, 2018 to 2030
Table 15: Asia Pacific Market Value (US$ Mn) Forecast, by Product, 2018 to 2030
Table 16: Asia Pacific Market Value (US$ Mn) Forecast, by Route of Administration, 2018 to 2030
Table 17: Asia Pacific Market Value (US$ Mn) Forecast, by Distribution Channel, 2018 to 2030
Table 18: Latin America Market Value (US$ Mn) Forecast, by Country/Sub-region, 2018 to 2030
Table 19: Latin America Market Value (US$ Mn) Forecast, by Product, 2018 to 2030
Table 20: Latin America Market Value (US$ Mn) Forecast, by Route of Administration, 2018 to 2030
Table 21: Latin America Market Value (US$ Mn) Forecast, by Distribution Channel, 2018 to 2030
Table 22: Middle East & Africa Market Value (US$ Mn) Forecast, by Country/Sub-region, 2018 to 2030
Table 23: Middle East & Africa Market Value (US$ Mn) Forecast, by Product, 2018 to 2030
Table 24: Middle East & Africa Market Value (US$ Mn) Forecast, by Route of Administration, 2018 to 2030
Table 25: Middle East & Africa Market Value (US$ Mn) Forecast, by Distribution Channel, 2018 to 2030
List Of Figures
Figure 01: Global Market Value (US$ Mn) Forecast, 2018 to 2030
Figure 02: Global Market Value Share, by Product, 2019
Figure 03: Global Market Value Share, by Route of Administration, 2019
Figure 04: Global Market Value Share, by Distribution Channel, 2019
Figure 05: Global Market Value Share, by Region, 2019
Figure 06: Regulatory Approval Process - U.S.
Figure 07: Regulatory Approval Process - Europe
Figure 08: Regulatory Approval Process - Japan
Figure 09: Global Market Value Share Analysis, by Product, 2019 and 2030
Figure 10: Global Market Value (US$ Mn), by Buphenyl, 2018 to 2030
Figure 11: Global Market Value (US$ Mn), by Ravicti, 2018 to 2030
Figure 12: Global Market Value (US$ Mn), by Ammonul, 2018 to 2030
Figure 13: Global Market Value (US$ Mn), by Dietary Supplements, 2018 to 2030
Figure 14: Global Market Value (US$ Mn), by Others, 2018 to 2030
Figure 15: Global Market Attractiveness Analysis, by Product, 2020 to 2030
Figure 16: Global Market Value Share Analysis, by Route of Administration, 2019 and 2030
Figure 17: Global Market Value (US$ Mn), by Oral, 2018 to 2030
Figure 18: Global Market Value (US$ Mn), by Intravenous, 2018 to 2030
Figure 19: Global Market Attractiveness Analysis, by Route of Administration, 2020 to 2030
Figure 20: Global Market Value Share Analysis, by Distribution Channel, 2019 and 2030
Figure 21: Global Market Value (US$ Mn) and Y-o-Y Growth (%), by Hospital Pharmacies, 2018 to 2030
Figure 22: Global Market Value (US$ Mn) and Y-o-Y Growth (%), by Retail Pharmacies, 2018 to 2030
Figure 23: Global Market Value (US$ Mn), by Online Pharmacies, 2018 to 2030
Figure 24: Global Market Attractiveness Analysis, by Distribution Channel, 2020 to 2030
Figure 25: Global Market Value Share Analysis, by Region, 2019 and 2030
Figure 26: Global Market Attractiveness Analysis, by Region, 2020 to 2030
Figure 27: North America Market Value (US$ Mn) Forecast and Y-o-Y Growth (%) Projection, 2018 to 2030
Figure 28: North America Market Value Share (%), by Country, 2019 and 2030
Figure 29: North America Market Attractiveness, by Country, 2020 to 2030
Figure 30: North America Market Value Share (%), by Product, 2019 and 2030
Figure 31: North America Market Attractiveness, by Product, 2020 to 2030
Figure 32: North America Market Value Share (%), by Route of Administration, 2019 and 2030
Figure 33: North America Market Attractiveness, by Route of Administration, 2020 to 2030
Figure 34: North America Market Value Share (%), by Distribution Channel, 2019 and 2030
Figure 35: North America Market Attractiveness, by Distribution Channel, 2020 to 2030
Figure 36: Europe Market Value (US$ Mn) Forecast and Y-o-Y Growth (%) Projection, 2018 to 2030
Figure 37: Europe Market Value Share (%), by Country/Sub-region, 2019 and 2030
Figure 38: Europe Market Attractiveness, by Country/Sub-region, 2020 to 2030
Figure 39: Europe Market Value Share (%), by Product, 2019 and 2030
Figure 40: Europe Market Attractiveness, by Product, 2020 to 2030
Figure 41: Europe Market Value Share (%), by Route of Administration, 2019 and 2030
Figure 42: Europe Market Attractiveness, by Route of Administration, 2020 to 2030
Figure 43: Europe Market Value Share (%), by Distribution Channel, 2019 and 2030
Figure 44: Europe Market Attractiveness, by Distribution Channel, 2020 to 2030
Figure 45: Asia Pacific Market Value (US$ Mn) Forecast and Y-o-Y Growth (%) Projection, 2018 to 2030
Figure 46: Asia Pacific Market Value Share (%), by Country/Sub-region, 2019 and 2030
Figure 47: Asia Pacific Market Attractiveness, by Country/Sub-region, 2020 to 2030
Figure 48: Asia Pacific Market Value Share (%), by Product, 2019 and 2030
Figure 49: Asia Pacific Market Attractiveness, by Product, 2020 to 2030
Figure 50: Asia Pacific Market Value Share (%), by Route of Administration, 2019 and 2030
Figure 51: Asia Pacific Market Attractiveness, by Route of Administration, 2020 to 2030
Figure 52: Asia Pacific Market Value Share (%), by Distribution Channel, 2019 and 2030
Figure 53: Asia Pacific Market Attractiveness, by Distribution Channel, 2020 to 2030
Figure 54: Latin America Market Value (US$ Mn) Forecast and Y-o-Y Growth (%) Projection, 2018 to 2030
Figure 55: Latin America Market Value Share (%), by Country/Sub-region, 2019 and 2030
Figure 56: Latin America Market Attractiveness, by Country/Sub-region, 2020 to 2030
Figure 57: Latin America Market Value Share (%), by Product, 2019 and 2030
Figure 58: Latin America Market Attractiveness, by Product, 2020 to 2030
Figure 59: Latin America Market Value Share (%), by Route of Administration, 2019 and 2030
Figure 60: Latin America Market Attractiveness, by Route of Administration, 2020 to 2030
Figure 61: Latin America Market Value Share (%), by Distribution Channel, 2019 and 2030
Figure 62: Latin America Market Attractiveness, by Distribution Channel, 2020 to 2030
Figure 63: Middle East & Africa Market Value (US$ Mn) Forecast and Y-o-Y Growth (%) Projection, 2018 to 2030
Figure 64: Middle East & Africa Market Value Share (%), by Country/Sub-region, 2019 and 2030
Figure 65: Middle East & Africa Market Attractiveness, by Country/Sub-region, 2020 to 2030
Figure 66: Middle East & Africa Market Value Share (%), by Product, 2019 and 2030
Figure 67: Middle East & Africa Market Attractiveness, by Product, 2020 to 2030
Figure 68: Middle East & Africa Market Value Share (%), by Route of Administration, 2019 and 2030
Figure 69: Middle East & Africa Market Attractiveness, by Route of Administration, 2020 to 2030
Figure 70: Middle East & Africa Market Value Share (%), by Distribution Channel, 2019 and 2030
Figure 71: Middle East & Africa Market Attractiveness, by Distribution Channel, 2020 to 2030
Figure 72: Market Position Analysis, 2019, by Tier and Size of Company
Figure 73: Abbott, Nutritional Business Segment - Revenue (US$ Mn) and Y-o-Y Growth (%), 2017 to 2019
Figure 74: Abbott, Nutritional Business Segment - Breakdown of Net Sales (%), by Region/Country, 2019
Figure 75: Abbott, Breakdown of Net Sales (%), by Nutritional Business Segment, 2019
Figure 76: Abbott, R&D Expenses (US$ Mn) Y-o-Y Growth (%), 2016 to 2019
Figure 77: Danone - Revenue (US$ Mn) and Y-o-Y Growth (%), 2017 to 2019
Figure 78: Danone - Breakdown of Net Sales (%), by Region/Country, 2019
Figure 79: Danone - Breakdown of Net Sales (%), by Business Segment, 2019
Figure 80: Reckitt Benckiser - Revenue (US$ Mn), 2016 to 2018
Figure 81: Reckitt Benckiser - Breakdown of Net Sales (%), by Region/Country, 2018
Figure 82: Reckitt Benckiser - Breakdown of Net Sales (%), by Business Segment, 2018
Figure 83: Horizon Therapeutics plc - Revenue (US$ Mn) and Y-o-Y Growth (%), 2017 to 2019
Figure 84: Horizon Therapeutics plc - Breakdown of Net Sales (%), by Region/County, 2019
Figure 85: Horizon Therapeutics plc - Breakdown of Net Sales (%), by Business Segment, 2019
Figure 86: RAVICTI & BUPHENYL - Product Revenue (US$ Mn), 2017 to 2019
Figure 87: Nestlé - Revenue (US$ Mn) and Y-o-Y Growth (%), 2016 to 2019
Figure 88: Nestlé - Breakdown of Net Sales (%), by Region/County, 2019
Figure 89: Nestlé - Breakdown of Net Sales (%), by Product Line, 2019
Figure 90: Bausch Health Companies, Inc., Revenue (US$ Mn) and Y-o-Y Growth (%), 2016 to 2019
Figure 91: Bausch Health Companies, Inc., Breakdown of Net Sales (%), by Region/County, 2019
Figure 92: Bausch Health Companies, Inc., Breakdown of Net Sales (%), by Business Segment, 2019
Figure 93: Bausch Health Companies, Inc., R&D Expenses (US$ Mn) and Y-o-Y Growth (%), 2017 to 2019
Figure 94: Ultragenyx Pharmaceutical, Inc., Revenue (US$ Mn) and Y-o-Y Growth (%), 2017 to 2019
Figure 95: Ultragenyx Pharmaceutical, Inc., Breakdown of Net Sales (%), by Region/County, 2019
Figure 96: Ultragenyx Pharmaceutical, Inc., DTX301 - R&D Expenses (US$ Mn) Y-o-Y Growth (%), 2018 to 2019
Figure 97: Ultragenyx Pharmaceutical, Inc., R&D Expenses (US$ Mn) Y-o-Y Growth (%), 2016 to 2019
Figure 98: Arcturus Therapeutics, Inc., Revenue (US$ Mn) and Y-o-Y Growth (%), 2016 to 2019
Figure 99: Arcturus Therapeutics, Inc., R&D Expenses (US$ Mn) and Y-o-Y Growth (%), 2016 to 2019
- FAQs -
Which segment of ornithine transcarbamylase deficiency treatment market is expected to account for the largest revenue?
Ravicti-based OCT deficiency treatment drugs are expected to capture major revenue share.
Which distribution channel is expected to lead the market share?
Hospital pharmacies are expected to be the primary point-of-sale for OCT deficiency treatment drugs.
Which is the leading regional market for ornithine transcarbamylase deficiency treatment?
North America accounts for a major ornithine transcarbamylase deficiency treatment market share.
Ornithine Transcarbamylase Deficiency Treatment Market