Market Overview

According to national urea cycle disorders foundation of USA, a urea cycle disorder is a genetic disorder caused by a mutation that results in a deficiency of one of the six enzymes in the urea cycle. These enzymes are responsible for removing ammonia from the blood stream.

The urea cycle involves a series of biochemical steps in which nitrogen, a waste product of protein metabolism, is removed from the blood and converted to a compound called urea in the blood. Normally, the urea is transferred into the urine and removed from the body.

In urea cycle disorders, the nitrogen accumulates in the form of ammonia, a highly toxic substance, resulting in hyperammonemia (elevated blood ammonia). Ammonia then reaches the brain through the blood, where it can cause irreversible brain damage, coma and/or death.

The estimated incidence of urea cycle disorders is 1 in 8500 births. Because many cases of urea cycle disorders remain undiagnosed and/or infants born with the disorders die without a definitive diagnosis, the exact incidence of these cases is unknown and underestimated.

It is believed that up to 20% of Sudden Infant Death Syndrome (SIDS) cases may be attributed to an undiagnosed inborn error of metabolism such a urea cycle disorder which are expected to drive the urea cycle disorders treatment market for over the forecast period. However, lack of definitive treatment options & diagnostic procedures, dearth of diseases prognosis data are expected to impact the urea cycle disorders treatment market revenue growth negatively over the forecast period.

Urea cycle disorders treatment market has been segmented on the basis of treatment type, enzyme deficiency type, and region

Based on the treatment type	Amino Acid Formulas Phenylbutyrate Sodium benzoate Others
Based on the enzyme deficiency type	Carbamyl Phosphate Synthetase (CPS1) N-Acetylglutamate Synthetase (NAGS) Ornithine Transcarbamylase (OTC Deficiency) Argininosuccinic Acid Synthetase (AS) Argininosuccinate Lyase (AL or ASA Lyase) Arginase (AG)
Based on end user	Hospital Pharmacies Retail Pharmacies Online Pharmacies

Urea cycle disorders treatment market is less competitive as few molecules and players are operating in the market place. Players in the market working to create awareness regarding disease among healthcare professionals, family members. For instance, in March 2017, Horizon Pharma plc. launched “UCD in Common”, an initiative for people impacted by a urea cycle disorder (UCD) in collaboration with people living with a UCD, their families, caregivers and healthcare professionals to offer supportive, educational and interactive resources.

Geographically, urea cycle disorders treatment market is segmented into regions viz. North America, Latin America, Europe, Asia Pacific and Japan, Middle East and Africa. North America and Europe regions are expected to garner larger market revenue share over the forecast period owing to presence of key players in the regions and rising awareness among key stakeholders which are expected to propel the demand for urea cycle disorders treatment during the forecast period in the above mentioned region.

The players in urea cycle disorders (UCD) Treatment market include Horizon Pharma plc. Lucane Pharma, Recordati Rare Diseases Inc., and Synlogic. to name a few.