The Ornithine-Transcarbamylase Deficiency drugs in development market research report provides comprehensive information on the therapeutics under development for Ornithine-Transcarbamylase Deficiency, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. GlobalData’s report assesses key aspects of the companies and drugs in development for Ornithine-Transcarbamylase Deficiency. Buy the report here.

The report also covers the descriptive pharmacological action of the therapeutics and the latest news and press releases. Additionally, the report provides an overview of the key players involved in therapeutic development for Ornithine-Transcarbamylase Deficiency and features dormant and discontinued products.

GlobalData tracks ten drugs in development for Ornithine-Transcarbamylase Deficiency by ten companies/universities/institutes. The top development phase for Ornithine-Transcarbamylase Deficiency is preclinical with five drugs in that stage. The Ornithine-Transcarbamylase Deficiency pipeline has nine drugs in development by companies and one by universities/ institutes. Some of the companies in the Ornithine-Transcarbamylase Deficiency pipeline products market are: Arcturus Therapeutics, Ultragenyx Pharmaceutical and Moderna.

The key targets in the Ornithine-Transcarbamylase Deficiency pipeline products market include Ornithine Carbamoyltransferase Mitochondrial (Ornithine Transcarbamylase or OTC or EC 2.1.3.3).

The key mechanisms of action in the Ornithine-Transcarbamylase Deficiency pipeline product include Ornithine Carbamoyltransferase Mitochondrial (Ornithine Transcarbamylase or OTC or EC 2.1.3.3) Activator with nine drugs in Phase III. The Ornithine-Transcarbamylase Deficiency pipeline products include two routes of administration with the top ROA being Intravenous and two key molecule types in the Ornithine-Transcarbamylase Deficiency pipeline products market including Gene Therapy, and Cell Therapy.

Ornithine-Transcarbamylase Deficiency overview

Ornithine transcarbamylase (OTC) deficiency is a rare X-linked genetic disorder characterized by complete or partial lack of the enzyme OTC. OTC plays an important role in the breakdown and removal of nitrogen in the body (the urea cycle). The lack of the OTC enzyme results in excessive accumulation of nitrogen, in the form of ammonia in the blood (hyperammonemia). Symptoms include vomiting, refusal to eat, progressive lethargy, and coma. Treatment includes nitrogen scavenging agents.

For a complete picture of Ornithine-Transcarbamylase Deficiency’s pipeline drug market, buy the report here.

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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s pipeline drugs offers detailed profiles of pharmaceutical drugs in all stages of pre-clinical and clinical development, from discovery through to pre-registration. Coverage is limited to novel human medicinal drugs and biosimilars seeking market approval proprietary and is one of two primary repositories of pharmaceutical drug information offered by GlobalData through its Pharmaceutical Intelligence Center.