Ornithine Transcarbamylase (OTC) Deficiency Treatment Market (Product: Buphenyl, Ravicti, Ammonul, Dietary Supplements, and Others; Route of Administration: Oral and Intravenous; and Distribution Channel: Hospital Pharmacies, Retail Pharmacies, and Online Pharmacies) - Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 2020-2030

nnovations in IV Medications for Life-threatening OTC Deficiency Surface amidst COVID-19 Outbreak

Since OTC deficiency is a rare X-linked genetic disorder, companies in the ornithine transcarbamylase (OTC) deficiency treatment market are under pressure to increase the availability of drugs amidst the COVID-19 (coronavirus) outbreak. Messenger RNA drug developer Arcturus Therapeutics has not only received the FDA’s (Food & Drug Association) approval for clinical trials of ARCT-810, an investigational treatment for OTC deficiency, but is also testing its coronavirus vaccine candidate. Thus, companies in the ornithine transcarbamylase (OTC) deficiency treatment market are tapping revenue opportunities by innovating in COVID-19 vaccines.

OTC deficiency is a life-threatening disorder, which needs extra care and attention during the COVID-19 pandemic. Companies are collaborating with retail and hospital pharmacies to avoid supply shocks for OTC deficiency medications, as most individuals are opting for remote healthcare diagnosis. They are innovating in intravenous (IV) medications since no FDA-approved drugs exist for the disorder.

Gene Therapy Helps Minimize Neurological Deficits and Liver Damage in Patients

Gene therapy holds promising potentials in ornithine transcarbamylase (OTC) deficiency treatment. The Boston Children’s Hospital is creating awareness about gene therapy, since OTC deficiency is a genetic condition caused due to the mutation of the OTC gene. By providing a healthy copy of the OTC gene through gene therapy, doctors aim to reduce incidence of neurological deficits and liver damage. Liver transplantation is another solution for ornithine transcarbamylase deficiency treatment.

Companies in the ornithine transcarbamylase (OTC) deficiency treatment market are increasing their production capabilities to manufacture BUPHENYL® (sodium phenylbutyrate) tablets and powder for oral administration. Since urea cycle disorders are commonly associated with the OTC deficiency, manufacturers in the ornithine transcarbamylase (OTC) deficiency treatment market are increasing the availability of BUPHENYL® to minimize adverse events in patients. BUPHENYL® is being used as an adjunctive therapy for chronic management of OTC deficiency.

Safety Information Enables Management of Extremely High Levels of Ammonia in Blood

Ravicti has become the gold standard for the treatment of the OTC deficiency. This oral liquid is being increasingly used for long-term management of high blood levels containing ammonia that commonly lead to urea cycle disorders (UCDs). Healthcare practitioners are recommending low-protein diet and dietary supplements to OTC deficiency patients. However, Ravicti has limitations for the treatment of extremely high levels of ammonia in the blood, especially in patients with UCD. This explains why the market is estimated to register a modest CAGR of ~4% during the forecast period. Hence, companies in the ornithine transcarbamylase (OTC) deficiency treatment market are increasing awareness about the safety of Ravicti to avoid serious side effects in patients.

OTC deficiency patients with kidney and liver problems need to seek doctor’s consultation before consuming Ravicti. Clinicians are providing special care to pregnant women and women who are breastfeeding to avoid any adverse events in infants.

Ammonul IV Holds Promising Potentials for Lowering Ammonia Levels in OTC Deficiency Patients

The FDA prescribed information is being used by medical practitioners to administer the doses of Ammonul in OTC deficiency patients. Genetics and metabolic experts are recommending hemodialysis as an effective solution to reduce plasma ammonia in OTC deficiency patients. As such, Ammonul is being extensively taken via intravenous administration to reduce the severity of UCDs. Ammonul IV helps in removing glycine and glutamate from plasma, thereby reducing contribution to ammonia formation. Thus, companies in the ornithine transcarbamylase (OTC) deficiency treatment market are increasing their R&D capabilities to develop improved formulations of Ammonul IV.

Since the management and diagnosis of UCD is complex, pathologists, nephrologists, and geneticists are stepping in to lower ammonia levels in the blood of OTC deficiency patients.

Nitrogen Scavenger Therapy Ensures Long-term UCD Management

The ornithine transcarbamylase (OTC) deficiency treatment market is projected to reach ~US$ 900 Mn by the end of 2030. However, limited consensus regarding long-term management approaches for OTC deficiency is potentially inhibiting market growth. Hence, pharmaceutical companies are introducing efficacious dietary supplements to keep ammonia levels in the blood under control. On the other hand, nitrogen scavenger therapy is being highly publicized to ensure long-term UCD management and to minimize the nitrogen load.

Companies in the ornithine transcarbamylase (OTC) deficiency treatment market are increasing awareness about a multidisciplinary approach for long-term UCD management. Since the male population is at a higher risk of being susceptible to the OTC deficiency, biochemical geneticists, dieticians and neuropsychologists are collaborating to introduce guidelines associated with chronic ammonia targets.

Dieticians Deliver Complex Calculations for Intake of Protein and Dietary Supplements

It has been found that a dietician’s consultation helps to improve clinical outcomes in OTC deficiency patients. Along with dietary supplements, nutritionists are recommending low-protein diets to improve the quality of life for OTC deficiency individuals. Patients that are adherent to the UCD therapy are also warranted for genetic testing to ensure successful management of OTC deficiency. Dieticians are playing an instrumental role in complex calculations related to the intake of protein and dietary supplements, which depends on different variables such as age-related growth rate, physical activity, and residual urea cycle function.

Providing the right amount of protein is necessary for efficient management of the OTC deficiency. Hence, companies in the ornithine transcarbamylase (OTC) deficiency treatment market are suggesting routine diagnosis of the rare disorder in order to increase clinical numbers. IV and oral drugs are being routinely used for long-term management of UCD in OTC deficiency patients.

Analysts’  Viewpoint

Since UCD associated with OTC deficiency can be potentially life threating for patients amidst the COVID-19 pandemic, companies in the ornithine transcarbamylase (OTC) deficiency treatment market are boosting their production capacities to increase the availability of IV and oral drugs. Gene therapy and nitrogen scavenger therapy are gaining popularity for long-term management of ammonia in blood streams. Since there is a lack of FDA-approved drugs for the OTC deficiency, companies in the ornithine transcarbamylase (OTC) deficiency treatment market should increase their R&D capabilities to innovate in IV medications. Companies should innovate in dietary supplements, since a close check on daily intake of protein and supplements helps to improve patient quality of life.

Ornithine Transcarbamylase (OTC) Deficiency Treatment Market: Overview

Ornithine transcarbamylase deficiency, a highly common urea cycle disorder and rare X-linked genetic disorder, is characterized by partial or a complete lack of enzyme ornithine transcarbamylase (OTC)

OTC is one of six enzymes that play a role in breakdown and removal of nitrogen in the body, a process known as the urea cycle

OTC deficiency is a genetic disorder characterized by a complete or partial lack of the enzyme ornithine transcarbamylase (OTC). It is an abnormal gene on the X chromosome and manifests mostly in males.

Both males and females may develop symptoms of OTC deficiency during childhood. Females who have a defective gene present on one of their X chromosomes are carriers for the disorder.

Ornithine Transcarbamylase (OTC) Deficiency Treatment Market: Drivers

Rising prevalence of OTC deficiency and development of new therapies for the treatment of OTC deficiency are major factors boosting the global ornithine transcarbamylase (OTC) deficiency treatment market

For instance, according to Orphanet, the estimated prevalence of ornithine transcarbamylase deficiency ranges between 1/56,500 and 1/113,000 live births across the world

Favorable reimbursement scenario and patient assistance programs provided by various international as well as regional organizations are also major drivers of the global Ornithine Transcarbamylase (OTC) deficiency treatment market

Patient compliance is affected due to bitter taste and foul odor of products. Hence, research and development is being carried out to develop new formulations of this drug in order to overcome such serious issues.

Consequently, formulation modification for better patient compliance also propels the global ornithine transcarbamylase (OTC) deficiency treatment market

Market Segmentation: Ornithine Transcarbamylase (OTC) Deficiency Treatment Market

In terms of product, the global ornithine transcarbamylase (OTC) deficiency treatment market has been divided into Buphenyl, Ravicti, Ammonul, dietary supplements, and others

Based on route of administration, the global ornithine transcarbamylase (OTC) deficiency treatment market has been bifurcated into oral and intravenous

In terms of distribution channel, the global ornithine transcarbamylase (OTC) deficiency treatment market has been segmented into hospital pharmacies, retail pharmacies, and online pharmacies

Each of the ornithine transcarbamylase (OTC) deficiency treatment market segments has been analyzed in detail for market trends, recent trends and developments, drivers, restraints, opportunities, and useful insights

The ornithine transcarbamylase (OTC) deficiency treatment market report provides current and future revenues (US$ Mn) for each of the segments for the period from 2018 to 2030, considering 2019 as the base year. The compound annual growth rate (% CAGR) has been provided for each segment and market from 2020 to 2030 along with market size estimations.

Regional Overview: Ornithine Transcarbamylase (OTC) Deficiency Treatment Market

In terms of region, the global ornithine transcarbamylase (OTC) deficiency treatment market has been segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. The regions have been divided into major countries and sub-regions.

North America dominated the global ornithine transcarbamylase (OTC) deficiency treatment market in 2019, and the trend is projected to continue during the forecast period

The current and future sizes of the ornithine transcarbamylase (OTC) deficiency treatment market, in terms of value (US$ Mn), of regional markets and their major countries have been provided in the report for the period from 2018 to 2030, along with their CAGRs for the period from 2020 to 2030

The study also offers a list of recommendations, highlights, and useful insights of the market, which would help new companies willing to enter the market and existing companies to increase market shares and help in the decision-making process

Major Players

The ornithine transcarbamylase (OTC) deficiency treatment market report concludes with the company profiles section that includes key information about the major players in the market

Key players analyzed in the ornithine transcarbamylase (OTC) deficiency treatment market report are -

• Abbott
• Nutricia (Danone Group)
• Mead Johnson (Reckitt Benckiser)
• Horizon Therapeutics plc
• Nestlé
• Bausch Health Companies, Inc.
• Ultragenyx Pharmaceutical Inc.
• Arcturus Therapeutics, Inc.
• Acer Therapeutics

Each of these players has been profiled in the global ornithine transcarbamylase (OTC) deficiency treatment market report based on parameters such as company overview, financial overview, business strategies, product portfolio, business segments, and recent developments

Key Questions Answered in Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Report

• What is the sales/revenue generated by each product, route of administration, and distribution channel segment of the global ornithine transcarbamylase (OTC) deficiency treatment market across all regions during the forecast period?
• What are the opportunities in the global ornithine transcarbamylase (OTC) deficiency treatment market?
• What are the major drivers, restraints, opportunities, and threats in the market?
• Which region is likely to expand at the fastest CAGR during the forecast period?
• Which ornithine transcarbamylase (OTC) deficiency treatment segments are expected to generate the highest revenue globally in 2030? Which segment is projected to expand at the highest CAGR during the forecast period?
• What is the market share or position of different companies operating in the global market in 2019?

Ornithine Transcarbamylase (OTC) Deficiency Treatment Market – Segmentation 

Product

• Buphenyl
• Ravicti
• Ammonul
• Dietary Supplements
• Others

Route of Administration

• Oral
• Intravenous

Distribution Channel

• Hospital Pharmacies
• Retail Pharmacies
• Online Pharmacies

Region

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

1. Preface

    1.1. Market Definition and Scope

    1.2. Market Segmentation

    1.3. Key Research Objectives

    1.4. Research Highlights

2. Assumptions and Research Methodology

3. Executive Summary : Global Ornithine Transcarbamylase (OTC) Deficiency Treatment Market

4. Market Overview

    4.1. Introduction

        4.1.1. Product Definition

        4.1.2. Industry Evolution / Developments

    4.2. Overview

    4.3. Market Dynamics

        4.3.1. Drivers

        4.3.2. Restraints

        4.3.3. Opportunities

    4.4. Global Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Analysis and Forecast, 2018–2030

        4.4.1. Market Revenue Projections (US$ Mn)

5. Key Insights

    5.1. Disease Prevalence & Incidence Rate globally with key countries

    5.2. Pipeline Analysis

    5.3. Standard Guidelines for Approval of Drugs

    5.4. Overview of Diagnostics approach for OTC Deficiency

    5.5. COVID-19 Pandemics Impact on Industry (Value Chain and Short / Mid / Long Term Impact)

6. Global Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Analysis and Forecast, by Product 

    6.1. Introduction & Definition

    6.2. Key Findings / Developments

    6.3. Global Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Product, 2018–2030

        6.3.1. Buphenyl

        6.3.2. Ravicti

        6.3.3. Ammonul

        6.3.4. Dietary Supplements

        6.3.5. Others

    6.4. Global Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Attractiveness, by Product 

7. Global Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Analysis and Forecast, by Route of Administration 

    7.1. Introduction & Definition

    7.2. Key Findings / Developments

    7.3. Global Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Route of Administration, 2018–2030

        7.3.1. Oral

        7.3.2. Intravenous

    7.4. Global Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Attractiveness, by Route of Administration 

8. Global Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Analysis and Forecast, by Distribution Channel 

    8.1. Introduction & Definition

    8.2. Key Findings / Developments

    8.3. Global Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Distribution Channel, 2018–2030

        8.3.1. Hospital Pharmacies

        8.3.2. Retail Pharmacies

        8.3.3. Online Pharmacies

    8.4. Global Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Attractiveness, by Distribution Channel 

9. Global Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Analysis and Forecast, by Region

    9.1. Key Findings

    9.2. Market Value Forecast, by Region

        9.2.1. North America 

        9.2.2. Europe 

        9.2.3. Asia Pacific 

        9.2.4. Latin America 

        9.2.5. Middle East & Africa 

    9.3. Global Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Attractiveness, by Country/Region

10. North America Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Analysis and Forecast

    10.1. Introduction

        10.1.1. Key Findings

    10.2. North America Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Product, 2018–2030

        10.2.1. Buphenyl

        10.2.2. Ravicti

        10.2.3. Ammonul

        10.2.4. Dietary Supplements

        10.2.5. Others

    10.3. North America Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Route of Administration, 2018–2030

        10.3.1. Oral

        10.3.2. Intravenous

    10.4. North America Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Distribution Channel, 2018–2030

        10.4.1. Hospital Pharmacies

        10.4.2. Retail Pharmacies

        10.4.3. Online Pharmacies

    10.5. North America Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Country, 2018–2030

        10.5.1. U.S.

        10.5.2. Canada

    10.6. Market Attractiveness Analysis 

        10.6.1. By Product 

        10.6.2. By Route of Administration 

        10.6.3. By Distribution Channel 

        10.6.4. By Country

11. Europe Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Analysis and Forecast

    11.1. Introduction

        11.1.1. Key Findings

    11.2. Europe Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Product, 2018–2030

        11.2.1. Buphenyl

        11.2.2. Ravicti

        11.2.3. Ammonul

        11.2.4. Dietary Supplements

        11.2.5. Others

    11.3. Europe Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Route of Administration, 2018–2030

        11.3.1. Oral

        11.3.2. Intravenous

    11.4. Europe Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Distribution Channel, 2018–2030

        11.4.1. Hospital Pharmacies

        11.4.2. Retail Pharmacies

        11.4.3. Online Pharmacies

    11.5. Europe Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Country/Sub-region, 2018–2030

        11.5.1. Germany

        11.5.2. U.K.

        11.5.3. France

        11.5.4. Spain

        11.5.5. Italy

        11.5.6. Rest of Europe

    11.6. Europe Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Attractiveness Analysis 

        11.6.1. By Product 

        11.6.2. By Route of Administration 

        11.6.3. By Distribution Channel 

        11.6.4. By Country/Sub-region

12. Asia Pacific Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Analysis and Forecast

    12.1. Introduction

        12.1.1. Key Findings

    12.2. Asia Pacific Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Product, 2018–2030

        12.2.1. Buphenyl

        12.2.2. Ravicti

        12.2.3. Ammonul

        12.2.4. Dietary Supplements

        12.2.5. Others

    12.3. Asia Pacific Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Route of Administration, 2018–2030

        12.3.1. Oral

        12.3.2. Intravenous

    12.4. Asia Pacific Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Distribution Channel, 2018–2030

        12.4.1. Hospital Pharmacies

        12.4.2. Retail Pharmacies

        12.4.3. Online Pharmacies

    12.5. Asia Pacific Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Country/Sub-region, 2018–2030

        12.5.1. China

        12.5.2. Japan

        12.5.3. India

        12.5.4. Australia & New Zealand

        12.5.5. Rest of Asia Pacific

    12.6. Asia Pacific Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Attractiveness Analysis 

        12.6.1. By Product 

        12.6.2. By Route of Administration 

        12.6.3. By Distribution Channel 

        12.6.4. By Country/Sub-region

13. Latin America Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Analysis and Forecast

    13.1. Introduction

        13.1.1. Key Findings

    13.2. Latin America Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Product, 2018–2030

        13.2.1. Buphenyl

        13.2.2. Ravicti

        13.2.3. Ammonul

        13.2.4. Dietary Supplements

        13.2.5. Others

    13.3. Latin America Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Route of Administration, 2018–2030

        13.3.1. Oral

        13.3.2. Intravenous

    13.4. Latin America Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Distribution Channel, 2018–2030

        13.4.1. Hospital Pharmacies

        13.4.2. Retail Pharmacies

        13.4.3. Online Pharmacies

    13.5. Latin America Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Country/Sub-region, 2018–2030

        13.5.1. Brazil

        13.5.2. Mexico

        13.5.3. Rest of Latin America

    13.6. Latin America Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Attractiveness Analysis 

        13.6.1. By Product 

        13.6.2. By Route of Administration 

        13.6.3. By Distribution Channel 

        13.6.4. By Country/Sub-region

14. Middle East & Africa Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Analysis and Forecast

    14.1. Introduction

        14.1.1. Key Findings

    14.2. Middle East & Africa Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Product, 2018–2030

        14.2.1. Buphenyl

        14.2.2. Ravicti

        14.2.3. Ammonul

        14.2.4. Dietary Supplements

        14.2.5. Others

    14.3. Middle East & Africa Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Route of Administration, 2018–2030

        14.3.1. Oral

        14.3.2. Intravenous

    14.4. Middle East & Africa Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Distribution Channel, 2018–2030

        14.4.1. Hospital Pharmacies

        14.4.2. Retail Pharmacies

        14.4.3. Online Pharmacies

    14.5. Middle East & Africa Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Value Forecast, by Country/Sub-region, 2018–2030

        14.5.1. GCC Countries

        14.5.2. South Africa

        14.5.3. Rest of Middle East & Africa

    14.6. Middle East & Africa Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Attractiveness Analysis 

        14.6.1. By Product 

        14.6.2. By Route of Administration 

        14.6.3. By Distribution Channel 

        14.6.4. By Country/Sub-region

15. Competition Landscape

    15.1. Market Player – Competition Matrix (By Tier and Size of companies)

    15.2. Market Share/Position Analysis, by Company (2019)

    15.3. Company Profiles

        15.3.1. Abbott

            15.3.1.1. Company Overview (HQ, Business Segments, Employee Strength)

            15.3.1.2. Company Financials

            15.3.1.3. Growth Strategies

            15.3.1.4. SWOT Analysis

        15.3.2. Nutricia (Danone Group)

            15.3.2.1. Company Overview (HQ, Business Segments, Employee Strength)

            15.3.2.2. Company Financials

            15.3.2.3. Growth Strategies

            15.3.2.4. SWOT Analysis

        15.3.3. Mead Johnson (Reckitt Benckiser)

            15.3.3.1. Company Overview (HQ, Business Segments, Employee Strength)

            15.3.3.2. Company Financials

            15.3.3.3. Growth Strategies

            15.3.3.4. SWOT Analysis

        15.3.4. Horizon Therapeutics plc

            15.3.4.1. Company Overview (HQ, Business Segments, Employee Strength)

            15.3.4.2. Company Financials

            15.3.4.3. Growth Strategies

            15.3.4.4. SWOT Analysis

        15.3.5. Nestlé

            15.3.5.1. Company Overview (HQ, Business Segments, Employee Strength)

            15.3.5.2. Company Financials

            15.3.5.3. Growth Strategies

            15.3.5.4. SWOT Analysis

        15.3.6. Bausch Health Companies, Inc.

            15.3.6.1. Company Overview (HQ, Business Segments, Employee Strength)

            15.3.6.2. Company Financials

            15.3.6.3. Growth Strategies

            15.3.6.4. SWOT Analysis

        15.3.7. Ultragenyx Pharmaceutical Inc.

            15.3.7.1. Company Overview (HQ, Business Segments, Employee Strength)

            15.3.7.2. Company Financials

            15.3.7.3. Growth Strategies

            15.3.7.4. SWOT Analysis

        15.3.8. Arcturus Therapeutics, Inc.

            15.3.8.1. Company Overview (HQ, Business Segments, Employee Strength)

            15.3.8.2. Company Financials

            15.3.8.3. Growth Strategies

            15.3.8.4. SWOT Analysis

        15.3.9. Acer Therapeutics

            15.3.9.1. Company Overview (HQ, Business Segments, Employee Strength)

            15.3.9.2. Company Financials

            15.3.9.3. Growth Strategies

            15.3.9.4. SWOT Analysis