Global Ornithine Transcarbamylase (OTC) Treatment Market: Overview
- Ornithine transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia, which is formed when proteins are broken down in the body. Individuals with OTC deficiency can build up excessive levels of ammonia, which accumulates in the blood and travels to the brain, causing coma, brain damage, and death.
- OTC deficiency is the most common of urea-cycle disorders, occurring in one in every 40,000 births. The genetic mutation responsible for OTC occurs on the X chromosome. Hence, women are typically carriers, while their sons with the gene suffer the disease. Symptoms include vomiting, refusal to eat, progressive lethargy, and coma.
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- Currently, the only curative approach is liver transplantation. However, this is often associated with significant morbidity and mortality risks both during the procedure and over the long-term. However, for less severe diseases, daily amino acid drugs could be used as off-label treatment options.
Key Driver of Global Ornithine Transcarbamylase (OTC) Treatment Market
- Ornithine transcarbamylase (OTC) deficiency can occur as a severe neonatal-onset disease in males (but rarely in females) and as a post-neonatal-onset (partial deficiency) disease in males and females
- According to the National Organization of Rare Disorder, the estimated frequency of OTC deficiency is 1 in 50,000 to 80,000. The estimated frequency of urea cycle disorders collectively is 1 in 35,000 and is rising globally. Increase in cases of ornithine transcarbamylase (OTC) deficiency is expected to drive the global ornithine transcarbamylase (OTC) treatment market during the forecast period.
Opportunities in Global Ornithine Transcarbamylase (OTC) Treatment Market
- Key players in the global ornithine transcarbamylase (OTC) treatment market acquire emerging players to expand product portfolio. Companies prefer to acquire R&D units of emerging players, aiming to co-develop innovative technologies. The strategy offers significant opportunities to companies, wherein these can leverage production and commercialize products.
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North America to Account for Major Share of Global Ornithine Transcarbamylase (OTC) Treatment Market
- In terms of region, the global ornithine transcarbamylase (OTC) treatment market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America is expected to dominate the global market during the forecast period.
- North America is projected to account for significant share of the global market due to availability of well-defined reimbursement policies from public and private health insurance firms, which help provide the patients easy access to diagnosis and extensive research & development activities.
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Key Players Operating in Global Ornithine Transcarbamylase (OTC) Treatment Market
Major players operating in the global ornithine transcarbamylase (OTC) treatment market include:
- Ultragenyx Pharmaceutical
- Ucyclyd Pharma
- Horizon Therapeutics plc
- OrphanPacific, Inc.
- Altis Endurance LLC
- SEQENS GROUP
- Bausch Health Companies, Inc.
- Other Prominent Players
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Global Ornithine Transcarbamylase (OTC) Treatment Market: Research Scope
Global Ornithine Transcarbamylase (OTC) Treatment Market, by Drug
- Sodium Phenylbutyrate
- Pharmaceutical Grade L-Citrulline
- Keto-acid
Global Ornithine Transcarbamylase (OTC) Treatment Market, by Distribution Channel
- Hospital Pharmacies
- Retail Pharmacies
- Others
Global Ornithine Transcarbamylase (OTC) Treatment Market, by Region
- North AmericaU.S.Canada
- EuropeGermanyFranceU.K.ItalySpainRussiaRest of Europe
- Asia PacificChinaJapanIndiaRest of Asia Pacific
- Latin AmericaBrazilMexicoRest of Latin America
- Middle East & AfricaGCC CountriesSouth AfricaRest of Middle East & Africa
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