Urea Cycle Disorder (UCD) is an assembly of rare genomic ailment which affects approximately 3,000 - 7000 people every year in the U.S. UCD is triggered by an alteration in genes, which causes insufficiency of any of the six vital enzymes essential for the urea cycle. Urea cycle includes a sequence of biochemical steps in which one of the waste products of protein metabolism (nitrogen) in blood is removed and progressively converted into a compound called urea. In end, urea in the blood is removed the body as urine. In UCD, nitrogen amasses in the form of ammonia, which is a highly poisonous substance for the body. It results into a precarious condition called hyperammonia (elevated levels of ammonia in blood). If the ammonia enters the brain cells via blood, it can lead to coma, irremediable brain damage, or in serious condition death.
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According to reports published by the CDC (Centers for Disease Control and Prevention), the anticipated incidence of UCD is 1 in 8500 births. Research findings suggest that many of the cases of UCD persist undiagnosed. In some of the cases, the newborn with UCD dies without a conclusive diagnosis. The precise global incidence of such cases is underrated or unknown. Some of the scientists in Europe suggest that Sudden Infant Death Syndrome (SIDS) cases might be due to an unknown inborn error of metabolism, for example, UCD. These scenarios are anticipated to drive the urea cycle disorders treatment market in the next few years. Additionally, research & development initiatives undertaken by research & academic institutes supported by pharmaceutical companies as well as governments are estimated to drive the global urea cycle disorders treatment market. Consequently, want of diagnosis & treatment options, unavailability of skilled personnel, and lack of reimbursement policies in developing countries are some of the factors hindering the growth of global urea cycle disorders treatment market. The UCD treatment market is less competitive as compared to other markets as UCD is a rare disorder and no conclusive research regarding it has been conducted so far. Government and pharmaceutical companies need to create awareness in the market and collaborative approaches should be undertaken.
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The global urea cycle disorders treatment market can be divided based on type of treatment, enzyme deficiency type, delivery channels, and region. On the basis of treatment type, the global urea cycle disorders treatment market can be classified into amino acid formulas, phenylbutyrate, sodium benzoate, and others. In terms of enzyme deficiency type, the global urea cycle disorders treatment market can be segregated into Carbamyl Phosphate Synthetase (CPS1), N-Acetylglutamate Synthetase (NAGS), Ornithine Transcarbamylase (OTC Deficiency), Argininosuccinic Acid Synthetase (AS), Argininosuccinate Lyase (AL or ASA Lyase), Arginase (AG).
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Based on delivery channel, the global urea cycle disorders treatment market can be segmented into hospital pharmacies, retail pharmacies, and online pharmacies.
Geographically, the global urea cycle disorders treatment market can be bifurcated into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. The urea cycle disorders treatment market in North America is projected to expand rapidly in the next few years owing to the well-developed health care system, appropriate reimbursement scenario, and acceptance of the latest techniques in the region. Following North America, the urea cycle disorders treatment market in Europe and Asia Pacific is anticipated to expand rapidly during the forecast period. Existence of well-established players, rise in investments in research and development by pharmaceutical giants, association of pharmaceutical and biotech companies with research institutes are some of drivers of the global urea cycle disorders treatment market.
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Key players operating in the urea cycle disorders treatment market include Horizon Pharma plc. Lucane Pharma, Recordati Rare Diseases Inc., and Synlogic.
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