Urea cycle disorders (UCDs) are a group of 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 occur during the period of infancy due to triggered postnatal catabolism. The diagnosis of urea cycle disorder is done by the analysis of urine and blood to check for abnormal metabolites and high ammonia levels. Frequent blood tests are done to monitor ammonia levels in urea cycle disorder patients. 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 global urea cycle disorders treatment market is estimated to be valued at US$ 1,188.9 million in 2020 and is expected to exhibit a CAGR of 3.5 % during the forecast period (2020-2027).
Figure 1. Global Urea Cycle Disorders Treatment Market Value (US$ Mn), 2016-2027
The increasing prevalence of urea cycle disorder is expected to drive growth of the global urea cycle disorders treatment market over the forecast period. For instance, according to the article published in the Orphanet Journal of Rare Diseases in June 2017, in 2017 the estimated prevalence of ornithine transcarbamylase deficiency ranges between 1/56,500 to 1/113,000 live births across the world.
Moreover, the increasing initiatives by key players operating in the market for supporting patients suffering from UCD is expected to fuel growth of the global urea cycle disorders treatment market in the near future.
For instance, in March 2017, Horizon Pharma plc. launched UCD in Common, an initiative for people suffering from urea cycle disorder, in order to offer supportive, educational, and interactive resources to the patients living with UCD, their families, caregivers, and healthcare professionals.
|Base Year:||2019||Market Size in 2020:||US$ 1,188.9 Mn|
|Historical Data for:||2016 to 2019||Forecast Period:||2020 to 2027|
|Forecast Period 2020 to 2027 CAGR:||3.5 %||2027 Value Projection:||US$ 1,510.2 Mn|
Bausch Health Companies, Inc., Recordati Rare Diseases Inc., Lucane Pharma SA, Acer Therapeutics, Ultragenyx Pharmaceutical Inc., Aeglea BioTherapeutics, Arcturus Therapeutics Holdings Inc., Orpharma Pty Ltd., Selecta Biosciences, Inc., Abbott Laboratories, NESTLÉ S.A., DANONE S.A., and Mead Johnson & Company, LLC
|Restraints & Challenges:||
Global Urea Cycle Disorders Treatment Market – Impact of Coronavirus (COVID-19)
Following the outbreak of COVID-19 in December 2019, the disease has spread to over 100 countries across the globe and the World Health Organization declared it as a public health emergency. According to the World Health Organization’s report, manifestation of coronavirus (COVID-19) has resulted in over 162 million infected individuals worldwide as of 17th May, 2021.
The coronavirus (COVID-19) pandemic and resulting lockdown in various countries across the globe has impacted the financial status of major companies in urea cycle disorders treatment market. The private healthcare sector is one of the sectors, which has been majorly impacted by the COVID-19 pandemic. The pandemic has negatively impacted the global urea cycle disorders treatment market in various aspects such as, research and development, production, and supply of medicines. The pandemic has also affected the growth of pharmaceutical businesses of various companies across the globe due to lockdowns implemented by governments of several countries.
Figure 2. Global Urea Cycle Disorders Treatment Market Share (%), By Region, 2020
North America region is expected to hold dominant position in the global urea cycle disorders treatment market over the forecast period, owing to increasing research and development by academic institutes in gene therapy for the treatment of the urea cycle disorders.
For instance, in March 2019, University of Calgary Cumming School of Medicine (CSM) and Alberta Health Services (AHS) collaboratively initiated the clinical trial study on treatment of urea cycle disorders with the help of gene therapy. The clinical trial study was conducted at Clinical Trials Unit (CTU) at the Foothills Medical Centre (FMC), a hospital in Canada.
Moreover, due to long-term therapy, patients may suffer from some complications, which include fussiness, sleepiness or sluggishness, vomiting, low body temperature, problems with posture, seizures, and others. Thus, such complications of long-term therapy is expected to hinder the market growth over the forecast period.
Major players operating in the global urea cycle disorders treatment market include Bausch Health Companies, Inc., Recordati Rare Diseases Inc., Lucane Pharma SA, Acer Therapeutics, Ultragenyx Pharmaceutical Inc., Aeglea BioTherapeutics, Arcturus Therapeutics Holdings Inc., Orpharma Pty Ltd., Selecta Biosciences, Inc., Abbott Laboratories, NESTLÉ S.A., DANONE S.A., and Mead Johnson & Company, LLC
The global urea cycle disorders treatment market includes various therapies such as amino acid supplements, sodium phenylbutyrate, glycerol phenylbutyrate, sodium benzoate, low protein diet, carglumic acid, etc. for the treatment of the inherited disorder such as urea cycle disorders, which affects various enzymes taking part in the urea cycle pathway in the human system. The urea cycle disorders are classified according to the deficiencies observed in different enzymes of the urea cycle such as OTC – ornithine transcarbamylase, ASD – argininosuccinic acid synthetase (citrullinemia), AG – Arginase, ALD – argininosuccinase acid lyase (argininosuccinic aciduria), CPS – carbamoyl phosphate synthetase, and NAGS – N-acetylglutamate synthetase.
Market players are focused on adopting different business strategies such as mergers and acquisitions in order to expand their offerings in the market.
For instance, in February 2017, Mead Johnson Nutrition Company (MJN) announced its merger with Reckitt Benckiser Group plc (RB). Mead Johnson, now a division of RB, added its globally-recognized brands, including Enfa and Nutramigen, which are used in dietary management of urea cycle disorder, to RB’s consumer health portfolio, thus strengthening its position globally.
Moreover, For Instance, In October 2017, Lucane Pharma SA was acquired by the Eurocept Pharmaceuticals, a company developing, distributing, and marketing of medicinal products in therapeutic areas such as anesthesia, oncology, immunology, and neurology. This acquisition has aided Eurocept Pharmaceuticals to invest in research and development of medicines for rare metabolic diseases such as urea cycle disorders.
Furthermore, Key players focused on inorganic strategies such as distribution agreements in order to strengthen their presence in the market.
For Instance, In 2016, Horizon Pharma plc and Swedish Orphan Biovitrum AB (publ) (Sobi), a biotechnological company in Sweden, announced the distribution agreement for the distribution of Ravicti in European countries including the U.K,. Germany, Italy, France, and Spain.
Key features of the study:
“*” marked represents similar segmentation in other categories in the respective section.