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Ornithine-Transcarbamylase deficiency market was expected to be valued at USD 896.6 million in the 2028 market and growing at a CAGR of 3.4% over the forecast period by 2028. Ornithine transcarbamylase deficiency is a hereditary condition that causes ammonia to build up in the blood. It is recognised by the absence of the enzyme ornithine transcarbamylase, which can be full or partial (OTC). The enzyme ornithine transcarbamylase (OTC) is required for the breakdown and elimination of nitrogen in the body. The absence of the OTC enzyme results in an excessive buildup of nitrogen in the blood in the form of ammonia. The most prevalent urea-cycle abnormality is OTC deficiency, which affects one in every 40,000 babies. OTC is caused by a mutation on the X-chromosome. As a result, most women are carriers, whereas their boys who have the gene are affected. Progressive lethargy, vomiting, reluctance to eat, and unconsciousness are all indications of OTC deficiency. Nitrogen scavenging agents can help with this deficit. It is possible to develop an OTC deficit at any age. Males are more commonly affected by this neonatal-onset illness, while females are extremely rare. Certain framework which have to be put on for the production of drug because there is no FDA (Food and Drug Administration) approved drug which exists for this urea cycle disorder which is consider to be a biggest opportunity for global Ornithine-Transcarbamylase. Market players have witnessed the strategic collaboration and acquisition to expand product portfolio and strengthen their position in the market to enhance the revenue of the company. In Dec 2018- The Food and Drug Administration (FDA) has expanded the approval of Ravicti (glycerol phenyl butyrate; Horizon Pharma) a nitrogen-binding agent, which is indicated for the chronic management of urea cycle disorders (UCDs) in adults and children. In April 2020- The San Diego biotech announced it had received the FDA’s permission to enter into the clinic with ARCT-810, an investigational treatment for Ornithine Transcarbamylase (OTC) deficiency.
