The ornithine transcarbamylase deficiency market was valued at USD813.6Mn by 2019. As per the research findings provided by the National Organization for Rare Disease (NORD), ornithine transcarbamylase deficiency (OTC) is a rare disease with a global prevalence rate of 1 per 50,000 to 80,000 per newborn babies. Strong product pipelines comprising of gene therapy and stem cell therapy to treat OTC deficiency will drive its market growth in the near future.

Ornithine transcarbamylase deficiency is an inherited X- linked genetic disorder that occurs in more in the males as compared to the females. It is one of the six enzymes which is essential in degrading and removing excess nitrogen in the human body via the urea cycle process. Lack of ornithine transcarbamylase results in the storage of excess nitrogen in the form of ammonia in the blood which reaches the nervous systems causing the occurrence of severe clinical manifestations such as coma, lethargy, vomiting, loss of appetite, etc.

The major segments related to the ornithine transcarbamylase deficiency market are:

By Treatment (2017–2027; US$ Mn)

Gene Therapy

Stem Cells Therapy

Ammonia Scavengers

Others

By Distribution Channel (2017–2027; US$ Mn)

Hospital Pharmacy

Retail Pharmacy

Others

Geography Segment(2017–2027; US$ Mn)

North America

United States
Canada

Europe

United Kingdom
Germany
Russia
France
Italy
Spain
Ukraine
Rest of Europe

Asia Pacific

China
Japan
Rest of Asia Pacific

Latin America (LATAM)

Brazil
Mexico
Rest of Latin America

Middle East and Africa (MEA)

GCC
Rest of MEA 

Justification for study?

• The intention of the study is to give a comprehensive outlook of the global ornithine transcarbamylase deficiency market
• The overall segmentation of the ornithine transcarbamylase deficiency market, by treatment, distribution channel, and geography is minutely studied. Ammonia scavengers and hospital pharmacy are dominating the treatment and distribution channel segments respectively
• Significant increase in the ornithine transcarbamylase deficiency in the newborns primarily determines the market growth
• Promising product pipeline and fast track status awarded to develop orphan drugs further bolster the market growth

Report gist?

• The study of the global ornithine transcarbamylase deficiency market includes qualitative analysis of factors such as drivers, restraints, and opportunities
• The report covers qualitative and quantitative analysis of the overall market segmented on the basis of treatment and distribution channel and categorization of the same at the geography level
• This research report presents the analysis of each segment from 2017 to 2027 in which 2019 is considered as the base year. Compounded annual growth rate is calculated for the respective segments from 2020 to 2027
• The study includes the profiles of major market players with a significant global and regional presence along with top company positioning

Significant customers?

• This study is suitable for industry participants and stakeholders in the biopharmaceutical companies engaged in manufacturing and marketing of drugs for the treatment of ornithine transcarbamylase (OTC) deficiency which is gaining huge demand owing to rising prevalence of OTC deficiency and fast track designated to develop drugs for its treatment worldwide
• The report will benefit researchers engaged in the drug development for the treatment of ornithine transcarbamylase deficiency
• Managers with financial institutions looking to publish recent and forecasted statistics pertaining to ornithine transcarbamylase deficiency market
• Financial institutions venture capitalist, analysts, investors, government organizations, policymakers, regulatory authorities, researchers, looking for insights into the market to determine future strategies 

Segment Analysis

Ammonia scavengers are currently considered as the first-line treatment for treating the urea cycle disorder associated with ornithine transcarbamylase deficiency. Ammonia scavenging drugs such as glycerol phenylbutyrate and sodium phenylbutyrate are available as a prodrug which forms a conjugate with the nitrogen present in the glutamine via the acetylation especially in the liver and kidneys in order to form phenylacetylglutamine and is excreted in the urine. Gene therapy is going to showcase magnificent market growth during the forecast period on account of a strong product pipeline for the treatment of ornithine transcarbamylase deficiency. DTX-301 developed by Ultragenyx Pharmaceutical, Inc., will be used to deliver the OTC gene expression in a durable fashion and critically reduce the complications associated with this orphan disease.

Hospital pharmacy is spearheading the distribution channel segment for ornithine transcarbamylase deficiency market. The presence of a hospital pharmacist is vital in compounding the drug regimen prescribed by the doctor for the treatment of orphan diseases such as ornithine transcarbamylase deficiency. Retail pharmacy will be growing at a rapid pace in the near future owing to the availability of ammonia scavenging drugs at a relatively lower price. The gene therapy drugs will be available at a subsidized rate at retail pharmacy owing to government intervention to cater to the medical needs of poor patients.

North America is presently holding the largest market share in the geography segment for ornithine transcarbamylase deficiency market. The constant rise in the number of newborn babies reported to suffer from urea cycle disorder associated with ornithine transcarbamylase deficiency drives its market growth. Furthermore the presence of a well-established healthcare infrastructure and domicile of key players such as Selecta Biosciences, Inc., Horizon Therapeutics, Plc., Phase Rx, Inc., etc. buttress the market growth in the region. Europe lags behind in the 2nd place chiefly due to the rising prevalence of ornithine transcarbamylase (OTC)deficiency in neonates which is 9 per 100,000 newborn babies. The fast track status designated by the European Medical Agency (EMA) for drug development in the treatment of OTC deficiency further accentuates its market growth in the region. Asia Pacific is keen to register magnificent market growth in the near future on account of developing healthcare infrastructure and huge government funding invested for drug development in the treatment of ornithine transcarbamylase deficiency in neonates born in the Asia Pacific region.