2021-2030 Analysis and Review Ornithine Transcarbamylase Deficiency Market
Ornithine Transcarbamylase Deficiency Market By Treatment (Gene Therapy, Stem Cells Therapy, Ammonia Scavengers and Others), By Distribution Channel (Hospital Pharmacy, Retail Pharmacy and Others) - Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 2020 – 2027
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
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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.
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