Carbamoyl Phosphate Synthetase (CPS) Deficiency Treatment: Introduction

  • Carbamoyl phosphate synthetase I deficiency is a rare inherited disorder characterized by complete or partial lack of the carbamoyl phosphate synthetase (CPS) enzyme
  • This is one of five enzymes that play a key role in the breakdown and removal of nitrogen from the body, a process known as the urea cycle
  • A lack of CPSI enzyme results in excessive accumulation of nitrogen, in the form of ammonia (hyperammonemia), in the blood

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Key Drivers of Global Carbamoyl Phosphate Synthetase (CPS) Deficiency Treatment Market

  • Increase in the incidence of urea accumulation is driving the market. Moreover, the incidence of carbamoyl phosphate synthetase 1 deficiency is estimated to be between 1 in 50,000 and 1 in 300,000.
  • 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.
  • 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 numerous cases of UCD persist undiagnosed. In some cases, a newborn with UCD dies without a conclusive diagnosis.
  • Research & development initiatives undertaken by research & academic institutes supported by pharmaceutical companies as well as governments are estimated to drive the global carbamoyl phosphate synthetase (CPS) deficiency treatment market

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Retail Pharmacies to be Highly Lucrative Segment

  • Based on distribution channel, the global carbamoyl phosphate synthetase deficiency treatment market can be categorized into hospital pharmacies, retail pharmacies, online pharmacies, and others
  • The retail pharmacies segment is likely to account for a major share of the global carbamoyl phosphate synthetase deficiency treatment market by 2030 due to moderate pricing of the products. Additionally, the presence of a large number of retail pharmacies, as compared to online and hospital pharmacies, is attributed to the segment’s prominent share of the global market.

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Key Players Operating in Global Carbamoyl Phosphate Synthetase (CPS) Deficiency Treatment Market

The global carbamoyl phosphate synthetase deficiency treatment market is consolidated, with the presence of less number of international and local players. Key players operating in the global carbamoyl phosphate synthetase deficiency treatment market are:

  • Horizon Pharma plc.
  • Lucane Pharma
  • Recordati Rare Diseases Inc.
  • Synlogic

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