U.S. Urea Cycle Disorders Treatment Market, By Treatment Type (Amino Acid Supplements, Sodium Phenylbutyrate, Glycerol Phenylbutyrate, Sodium Benzoate, and Others), By Enzyme Deficiency Type (OTC – Ornithine Transcarbamylase, AS – Argininosuccinate Synthetase (citrullinemia), AG – Arginase, AL – Argininosuccinate Lyase, CPS1 – Carbamoyl Phosphate Synthase and NAGS – N-acetylglutamate Synthase), By Route of Administration (Oral and Injectable), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, and Online Pharmacies)-

Published In : Mar 2023

Code : CMI5594

Pages :155

Formats :

Excel and PDF

Industry : Pharmaceutical

Market Challenges And Opportunities

U.S. Urea Cycle Disorders Treatment Market– Impact of Coronavirus (COVID-19) Pandemic

Supply chain and manufacturing activities in the U.S. have been disrupted due to lockdowns implemented by governments in the past year, and also facing problems with regards to the transportation of drug products. The coronavirus or COVID-19 outbreak that started in Wuhan, China has spread across continents, affecting various industries globally. The supply of key materials has been severely disrupted due to forced quarantine, and a lack of labor and raw materials. As the link between regional warehouses is not smooth, the transportation of raw materials between regions cannot be carried out successfully. This shortage of raw materials and components has affected the supply chain of the U.S. urea cycle disorders treatment market. Due to the severe shortage of medical resources at the front line, only patients diagnosed with serious conditions can be hospitalized. Unfortunately, the pathogenic mechanism of the virus has not been identified completely, therefore, there is no specific drug and treatment except for symptomatic and supportive treatments. Respiratory support devices such as life-support machines, atomizers, oxygen generators, and monitors are primary clinical treatment medical devices. Thus, from diagnosis to cure, the need for instruments for measuring temperature, nucleic acid diagnostic kits, antiviral medical products, and life-support machines has increased consistently. Furthermore, the COVID-19 pandemic has impacted the global as well as the U.S. economy and in turn, the urea cycle disorders treatment market.

U.S. Urea Cycle Disorders Treatment Market - Driver

Increasing product approvals for urea cycle disorders

Increasing product approvals for urea cycle disorders treatment by regulatory bodies is expected to drive market growth over the forecast period. For instance, in December 2022, Acer Therapeutics Inc., a pharmaceutical company, and its collaboration partner, Relief Therapeutics Holding AG, a commercial-stage biopharmaceutical company, announced that the U.S. Food and Drug Administration (FDA) had approved OLPRUVA (sodium phenylbutyrate) for oral suspension in the U.S. for the treatment of certain patients living with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).

High Cost Of Urea Cycle Disorders Treatment

Drugs such as RAVICTI is a nitrogen-binding agents approved for the treatment of chronic management of urea cycle disorder (UCD) in adults and children. RAVICTI is developed by Horizon Therapeutics plc. a global biotechnology company, and it costs around US$ 793,632 for a year of treatment of a patient suffering from urea cycle disorder. RAVICTI is one of the most expensive drugs available in the U.S. for the treatment of UCD.