Ornithine Transcarbamylase Deficiency Treatment Market Size
| Study Period | 2019 - 2029 |
| Market Size (2024) | USD 0.82 Billion |
| Market Size (2029) | USD 1.01 Billion |
| CAGR (2024 - 2029) | 4.30 % |
| Fastest Growing Market | Asia Pacific |
| Largest Market | North America |
Major Players
*Disclaimer: Major Players sorted in no particular order |
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Ornithine Transcarbamylase Deficiency Treatment Market Analysis
The Ornithine Transcarbamylase Deficiency Treatment Market size is estimated at USD 0.82 billion in 2024, and is expected to reach USD 1.01 billion by 2029, growing at a CAGR of 4.30% during the forecast period (2024-2029).
Ornithine transcarbamylase (OTC) deficiency is a rare X-linked genetic disorder showing a complete or partial lack of the ornithine transcarbamylase (OTC) enzyme. The rising approvals for novel pipeline products for ornithine transcarbamylase (OTC) deficiency treatment are expected to influence market growth. In March 2024, iECURE received the Medicines & Healthcare Products Regulatory Agency (MHRA) approval for its clinical trial authorization application (CTA) to expand the phase I/II OTC-HOPE clinical trial of its investigational gene editing-based therapy, ECUR-506, in the United Kingdom.
In addition, in August 2023, Bloomsbury Genetic Therapies Limited received orphan drug designation from the US Food and Drug Administration (FDA) for the company's investigational liver-targeted gene therapy, BGT-OTCD, for the treatment of ornithine transcarbamylase deficiency.
Furthermore, growing awareness for ornithine transcarbamylase deficiency disease and increasing campaigns organized by public and private entities are expected to fuel market growth. For instance, the NIH's National Center for Advancing Translational Sciences (NCATS) and the NIH Clinical Center sponsor the event, Rare Disease Day, on the last day of February, i.e., 28 or 29, as a part of the global Rare Disease Day observance every year. This day celebrates and includes raising awareness about rare diseases, the people they affect, etc. NIH conducted this event on February 29, 2024, and held it in person at the NIH Main Campus (Natcher Conference Center), Bethesda, United States.
In addition, favorable reimbursement scenarios in developed countries and patient assistance programs, particularly in developing regions, have also been fueling the growth of the OTC deficiency treatment market in recent years. For instance, in January 2024, Medunik USA, a member of Duchesnay Pharmaceutical Group (DPG), announced that Pheburane coverage is added to the Medicaid Preferred Drug List in 10 states with the recent addition of Indiana, Tennessee, Georgia, and Missouri, along with those already in effect in New Hampshire, New Mexico, North Dakota, Oklahoma, Texas, and Utah. Pheburane is also included on the California Contract Drugs List, and no prior authorization is required for the drug. Hence, it is easily accessible to patients with reimbursement. The drug is covered by commercial insurance plans, mainly offered by private players, and is covered by employers, which constitutes around 80% of the total commercially insured US population.
Medunik USA offers a patient program called the UNIK Support Program for Pheburanr, which provides specialized services, including a copay savings program, patient care liaison services, mail-order pharmacy, and other support services. Thus, the factors above are expected to boost the market growth.
However, the high cost of drugs, limited budget, and less awareness of disease and treatment in lower-income countries may restrain the market growth.
Ornithine Transcarbamylase Deficiency Treatment Market Trends
The Glycerol Phenylbutyrate Segment is Expected to Witness Significant Growth During the Forecast Period
Glycerol phenylbutyrate (RAVICTI) is an oral liquid suggested for use as a nitrogen-binding agent for treating patients with chronic urea cycle disorders (UCDs) such as OTC deficiency. Increasing patient compliance with glycerol phenylbutyrate compared to sodium phenylbutyrate is the major driving factor responsible for the dominance of the glycerol phenylbutyrate segment. RAVICTI is nearly odorless and tasteless, which is the primary reason for patient adherence to this drug, and the pungent odor and salty taste of buphenyl have made patients switch from it to RAVICTI in recent years.
RAVICTI is dispensed in a pharmacy and does not require administration from a healthcare practitioner. Patients are typically given 132 bottles annually, each with a list price of USD 5,031. RAVICTI has an annual list price of USD 664,092 per patient. Hence, manufacturer Horizon Therapeutics offers a way for some patients to save on RAVICTI through the Horizon Cares Patient Assistance Program. The company also educates healthcare professionals about gaining access to medicine and various patient financial support programs.
Additionally, companies are gaining commercial rights for glycerol phenylbutyrate in untapped geographies to provide better accessibility to patients to treat OTC deficiency. For instance, in May 2022, Immedica Pharma AB and Japanese company OrphanPacific Inc. entered an agreement under which OrphanPacific gains exclusive rights to RAVICTI in Japan.
Hence, due to the abovementioned factors, the glycerol phenylbutyrate segment is expected to grow significantly in the upcoming years.
North America is Expected to Dominate the Market Studied
North America is expected to dominate the market owing to factors such as increasing product approvals, ongoing clinical trials in the region, and comparatively better diagnosis and treatment rates with favorable reimbursement scenarios. According to the Association for Diagnostics & Laboratory Medicine, the estimated incidence of urea cycle disorders is 1 in 8,200 births in the United States. The calculated overall average birth prevalence of UCD is approximately 1 in 35,000, with two-thirds having symptoms in the neonatal period. OTC deficiency is observed in around 60% of the urea cycle disorder cases.
Moreover, patient assistance programs in terms of finance, education, awareness, etc., provided by public and private organizations are also propelling the growth of the market studied. In February 2024, the National Urea Cycle Disorders Foundation initiated the NUCDF Mentor Program to provide newly diagnosed families an opportunity to receive personalized guidance, education, and support specific to their family and disorder situation.
Therefore, the factors mentioned above are expected to boost market growth in the region.
Ornithine Transcarbamylase Deficiency Treatment Industry Overview
The ornithine transcarbamylase (OTC) deficiency treatment market is consolidated due to key players capturing the bulk of market share globally and regionally. The competitive landscape includes an analysis of a few international and local companies with substantial market shares. Some key players include Amgen Inc. (Horizon Therapeutics PLC), Bausch Health Companies Inc., Ultragenyx Pharmaceutical Inc., Arcturus Therapeutics Inc., Zevra Therapeutics (Acer Therapeutics), iECURE, Danone Group (Nutricia), Abbott, Reckitt Benckiser, Nestle, Moderna Inc., Duchesnay Pharmaceutical Group-DPG (Medunik USA), and Eurocept Pharmaceuticals Holding (Lucane Pharma).
Ornithine Transcarbamylase Deficiency Treatment Market Leaders
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Amgen Inc. (Horizon Therapeutics plc)
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Bausch Health Companies Inc.
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Ultragenyx Pharmaceutical Inc.
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Arcturus Therapeutics, Inc.
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Zevra Therapeutics (Acer Therapeutics)
*Disclaimer: Major Players sorted in no particular order
Ornithine Transcarbamylase Deficiency Treatment Market News
- November 2023: Zevra Therapeutics Inc. acquired Acer Therapeutics Inc., which marked an important milestone in executing Zevra's strategy to become a leader in products of treatments for rare diseases such as urea cycle disorders.
- October 2023: Amgen acquired Horizon Therapeutics PLC with a valuation of around USD 27.8 billion. This acquisition strengthened Amgen's position in the rare disease portfolio market.
- December 2022: Acer Therapeutics Inc. and its collaboration partner, Relief Therapeutics Holding SA, announced the US Food and Drug Administration (FDA) approval for OLPRUVA (sodium phenylbutyrate) for oral suspension in the United States. This drug is indicated for the treatment of urea cycle disorders (UCDs), including OTC deficiency.
Ornithine Transcarbamylase Deficiency Treatment Market Report - Table of Contents
1. INTRODUCTION
- 1.1 Study Assumptions and Market Definition
- 1.2 Scope of the Study
2. RESEARCH METHODOLOGY
3. EXECUTIVE SUMMARY
4. MARKET DYNAMICS
- 4.1 Market Overview
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4.2 Market Drivers
- 4.2.1 Rising Product Approvals With Novel Pipeline Products for Ornithine Transcarbamylase (OTC) Deficiency Treatment
- 4.2.2 Growing Awareness for Disease with Campaigns Organized by Public and Private Entities
- 4.2.3 Favorable Reimbursement Scenario and Patient Assistance Programs
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4.3 Market Restraints
- 4.3.1 High Cost of Drugs and Limited Budget
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4.4 Porter's Five Forces Analysis
- 4.4.1 Threat of New Entrants
- 4.4.2 Bargaining Power of Buyers/Consumers
- 4.4.3 Bargaining Power of Suppliers
- 4.4.4 Threat of Substitute Products
- 4.4.5 Intensity of Competitive Rivalry
5. MARKET SEGMENTATION (Market Size by Value - USD)
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5.1 By Treatment Type
- 5.1.1 Glycerol Phenylbutyrate
- 5.1.2 Sodium Phenylbutyrate
- 5.1.3 Sodium Phenylacetate and Sodium Benzoate
- 5.1.4 Other Treatment Types (Gene Therapy, Dietary Supplements, etc.)
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5.2 By Route of Administration
- 5.2.1 Oral
- 5.2.2 Intravenous
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5.3 By Distribution Channel
- 5.3.1 Hospital Pharmacies
- 5.3.2 Retail Pharmacies
- 5.3.3 Online Pharmacies
- 5.3.4 Other Distribution Channels (Specialty Stores, Convenience Stores, etc.)
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5.4 Geography
- 5.4.1 North America
- 5.4.1.1 United States
- 5.4.1.2 Canada
- 5.4.1.3 Mexico
- 5.4.2 Europe
- 5.4.2.1 Germany
- 5.4.2.2 United Kingdom
- 5.4.2.3 France
- 5.4.2.4 Italy
- 5.4.2.5 Spain
- 5.4.2.6 Rest of Europe
- 5.4.3 Asia-Pacific
- 5.4.3.1 China
- 5.4.3.2 Japan
- 5.4.3.3 India
- 5.4.3.4 Australia
- 5.4.3.5 South Korea
- 5.4.3.6 Rest of Asia-Pacific
- 5.4.4 Rest of the World
6. COMPETITIVE LANDSCAPE
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6.1 Company Profiles
- 6.1.1 Amgen Inc. (Horizon Therapeutics PLC)
- 6.1.2 Bausch Health Companies Inc.
- 6.1.3 Ultragenyx Pharmaceutical Inc.
- 6.1.4 Arcturus Therapeutics Inc.
- 6.1.5 Zevra Therapeutics (Acer Therapeutics)
- 6.1.6 iECURE
- 6.1.7 Danone Group (Nutricia)
- 6.1.8 Abbott
- 6.1.9 Reckitt Benckiser
- 6.1.10 Nestle
- 6.1.11 Moderna, Inc.
- 6.1.12 Duchesnay Pharmaceutical Group-DPG(Medunik�USA)
- 6.1.13 Eurocept Pharmaceuticals Holding (Lucane Pharma)
- *List Not Exhaustive
7. MARKET OPPORTUNITIES AND FUTURE TRENDS
** Subject To AvailablityOrnithine Transcarbamylase Deficiency Treatment Industry Segmentation
Ornithine transcarbamylase deficiency is a rare genetic condition that results in ammonia building up in the blood. This condition is more prominently seen in boys than girls and tends to be more severe when symptoms emerge shortly after birth. Ornithine transcarbamylase deficiency is caused by changes in the OTC gene, which instructs the body to make the OTC enzyme. In OTC deficiency, the OTC gene is either damaged or missing.
The ornithine transcarbamylase (OTC) deficiency treatment market is segmented into treatment type, route of administration, distribution channel, and geography. The market is segmented by treatment type into glycerol phenylbutyrate, sodium phenylbutyrate, sodium phenylacetate and sodium benzoate, and other treatment types. By route of administration, the market is segmented into oral and intravenous. By distribution channel, the market is segmented into hospital pharmacies, retail pharmacies, online pharmacies, and other distribution channels. By geography, the market is segmented into North America, Europe, Asia-Pacific, and Rest of the World. The report also offers the market sizes and forecasts for 13 countries across the region. For each segment, the market size and forecast are provided in terms of value (USD).
| By Treatment Type | Glycerol Phenylbutyrate | |
| Sodium Phenylbutyrate | ||
| Sodium Phenylacetate and Sodium Benzoate | ||
| Other Treatment Types (Gene Therapy, Dietary Supplements, etc.) | ||
| By Route of Administration | Oral | |
| Intravenous | ||
| By Distribution Channel | Hospital Pharmacies | |
| Retail Pharmacies | ||
| Online Pharmacies | ||
| Other Distribution Channels (Specialty Stores, Convenience Stores, etc.) | ||
| Geography | North America | United States |
| Canada | ||
| Mexico | ||
| Geography | Europe | Germany |
| United Kingdom | ||
| France | ||
| Italy | ||
| Spain | ||
| Rest of Europe | ||
| Geography | Asia-Pacific | China |
| Japan | ||
| India | ||
| Australia | ||
| South Korea | ||
| Rest of Asia-Pacific | ||
| Geography | Rest of the World |
Ornithine Transcarbamylase Deficiency Treatment Market Research FAQs
How big is the Ornithine Transcarbamylase Deficiency Treatment Market?
The Ornithine Transcarbamylase Deficiency Treatment Market size is expected to reach USD 0.82 billion in 2024 and grow at a CAGR of 4.30% to reach USD 1.01 billion by 2029.
What is the current Ornithine Transcarbamylase Deficiency Treatment Market size?
In 2024, the Ornithine Transcarbamylase Deficiency Treatment Market size is expected to reach USD 0.82 billion.
Who are the key players in Ornithine Transcarbamylase Deficiency Treatment Market?
Amgen Inc. (Horizon Therapeutics plc), Bausch Health Companies Inc., Ultragenyx Pharmaceutical Inc., Arcturus Therapeutics, Inc. and Zevra Therapeutics (Acer Therapeutics) are the major companies operating in the Ornithine Transcarbamylase Deficiency Treatment Market.
Which is the fastest growing region in Ornithine Transcarbamylase Deficiency Treatment Market?
Asia Pacific is estimated to grow at the highest CAGR over the forecast period (2024-2029).
Which region has the biggest share in Ornithine Transcarbamylase Deficiency Treatment Market?
In 2024, the North America accounts for the largest market share in Ornithine Transcarbamylase Deficiency Treatment Market.
What years does this Ornithine Transcarbamylase Deficiency Treatment Market cover, and what was the market size in 2023?
In 2023, the Ornithine Transcarbamylase Deficiency Treatment Market size was estimated at USD 0.78 billion. The report covers the Ornithine Transcarbamylase Deficiency Treatment Market historical market size for years: 2019, 2020, 2021, 2022 and 2023. The report also forecasts the Ornithine Transcarbamylase Deficiency Treatment Market size for years: 2024, 2025, 2026, 2027, 2028 and 2029.
Ornithine Transcarbamylase Deficiency Treatment Industry Report
Statistics for the 2024 Ornithine Transcarbamylase (OTC) Deficiency Treatment market share, size and revenue growth rate, created by Mordor Intelligence™ Industry Reports. Ornithine Transcarbamylase (OTC) Deficiency Treatment analysis includes a market forecast outlook for 2024 to 2029 and historical overview. Get a sample of this industry analysis as a free report PDF download.
