Ornithine transcarbamylase is one of the six enzymes which is vital in the removal of excess nitrogen in the form of ammonia from the human blood. It is categorized as an inherited X- linked genetic disorder that occurs in more in the males as compared to the females. Lack of treatment will result in hyperammonemia which will cause coma, lethargy, loss of appetite and vomiting, etc.
Ammonia scavenging drugs are leading the treatment segment for the ornithine transcarbamylase (OTC) deficiency market. Prodrug such as glycerol phenylbutyrate and sodium phenylbutyrate forms an acetyl conjugate with the nitrogen present in the glutamine via the acetylation process in the detoxification organs such as liver and kidneys in order to facilitate the excretion of excess nitrogen via the urine. Gene therapy is going to register faster market growth in the near future owing to the strong product pipeline for the treatment of ornithine transcarbamylase (OTC) deficiency. Ultragenyx Pharmaceutical, Inc., has developed DTX-301 to deliver the OTC gene expression in a durable fashion to reduce its complications associated with clinical manifestation of this rare disease
Browse the full report Ornithine Trancarbamylase Deficiency Market – Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 2020 – 2027 at https://www.atlanticmarketresearch.com/ornithine-transcarbamylase-deficiency-market.
Hospital pharmacy is currently leading the distribution channel segment for ornithine transcarbamylase (OTC) deficiency market. The vital factor which is related to its positive market growth is the presence of a hospital pharmacist for dispensing the medicines with precise posology prescribed by the physicians for the treatment of urea cycle disorder associated with ornithine transcarbamylase (OTC) deficiency. Retail pharmacy is going to embark on a rampant market growth during the forecast period on account of the availability of ammonia scavenging drugs at a discounted price. Going forward the government healthcare agencies will be providing gene therapy drugs at a subsidized rate in the retail pharmacy to cater medical needs of low and middle-income groups households.
North America is dominating the geography segment for ornithine transcarbamylase (OTC) deficiency market. Significant increase in the number of newborn male babies suffering from ornithine transcarbamylase deficiency primarily drives its market growth in the region. Additionally domicile of major players such as Selecta Biosciences, Inc., Horizon Therapeutics, Plc., Phase Rx, Inc., etc. further propel the market growth in the region. Europe holds the second-largest market in the geography segment for ornithine transcarbamylase deficiency market. As per the European Commission report the prevalence rate of ornithine transcarbamylase deficiency in the neonates is 9 per 100,000 live births. Supportive regulatory environment provided by the European Medical Agency (EMA), by designating fast track status to the drug development for the treatment of ornithine transcarbamylase deficiency in the region. Asia Pacific is keen to register excellent market growth during the forecast period owing to the increasing trend of hospital pharmacy affiliated with corporate hospitals and increasing funds invested by government healthcare agencies for the drug development of rare diseases.
Biopharmaceutical companies actively engaged in providing medicines for the treatment of ornithine transcarbamylase deficiency are Merck KGaA., Phase Rx, Inc., Promethera Biosciences SA., Horizon Therapeutics, plc., Lucane Pharma SA., Swedish Orphan Biovitrum AB, Selecta Biosciences, Inc., Translate Bio, Inc., Ultragenyx Pharmaceutical, Inc., and Unicyte AG.
Market Key Takeaways:
Constant rise in the neonates worldwide suffering urea cycle disorder associated with ornithine transcarbamylase deficiency
Strong product pipeline comprising of gene therapy and stem cells transplantation to treat ornithine transcarbamylase (OTC) deficiency
Supportive regulatory environment provided by the healthcare agencies throughout the globe by awarding fast track designation for the drug development in treating OTC deficiency which is an orphan disease