Urea cycle disorders (UCDs) are a group of orphan inherited defects of six enzymes and two transporters that constitute the urea cycle in the periportal liver cells. The age of onset symptoms vary between the different types of UCDs. UCDs often occurs during the period of infancy due to triggered postnatal catabolism. The diagnosis is made by analysis of the urine and blood for abnormal metabolites (substance produced by metabolism), and high ammonia levels. Frequent blood tests are done to monitor ammonia levels. The treatment of urea cycle disorders consists of dietary management to limit ammonia production. A liver transplant can be an effective treatment for urea cycle disorder for the proper functioning of urea cycle.
The urea cycle disorder treatment market in the U.S. and Europe is expected to grow rapidly in the next few years. Several pharmaceutical companies are under the development process of their late stage pipeline drugs for urea cycle disorders. Factors such as, increasing healthcare expenditures in the U.S, rise in the U.S. pharmaceutical R&D expenditures and collaboration between pharmaceutical companies and research institutes have driven the growth of the U.S. urea cycle disorder treatment market. Further, upcoming launches of anti-UCD drugs would contribute to the expansion of the urea cycle disorder treatment market in the U.S. as well as in Europe.
The U.S. urea cycle disorder treatment market is dominated by certain key players including Horizon Pharma Inc., Ultragenyx Pharmaceuticals Inc., Aeglea Bio Therapeutics and Synlogix. Among all the key players, Horizon Pharma ruled the U.S. UCD treatment market owing to the strong performance of its product. Horizon’ Ravicti is the market leader due its strong curative impact on the patients with UCD. The remaining key players have their products under the development process of pipeline drugs for UCDs. There would be a generic competition between the upcoming anti-UCD drugs in the coming years.