Global Urea Cycle Disorder Treatment Market is segmented By Treatment Type (Glycerol Phenylbutyrate, Amino Acid Supplements, Sodium Phenylbutyrate, Sodium Benzoate, Others (Low Protein Diet, Carglumic acid, etc.)), By Enzyme Deficiency Type (OTC – Ornithine Transcarbamylase, AS – Argininosuccinate Synthetase (citrullinemia), AG – Arginase, AL – Argininosuccinate Lyase, CPS1 – Carbamoyl Phosphate Synthase, NAGS – N-acetylglutamate Synthase), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, Online Pharmacies), By Region (North America, Europe, South America, Asia Pacific, Middle East, and Africa) – Share, Size, Outlook, and Opportunity Analysis, 2024-2031
Report Overview
The global urea cycle disorders treatment market size was valued US$ YY billion in 2022 and is estimated to reach US$ YY billion by 2031, growing at a high CAGR during the forecast period (2024-2031).
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 bloodstream. 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 the urea cycle.
Market Dynamics
The urea cycle disorders treatment market growth is driven by the increasing incidence of urea cycle disorders; rising initiatives by key players operating in the market for supporting patients suffering from UCD are the major driving factors of the market.
Increasing incidence of urea cycle disorders, is expected to drive the growth in the forecast period
According to reports published by the CDC (Centers for Disease Control and Prevention), the anticipated incidence of UCD is 1 in 8500 births. Because many urea cycle disorders remain undiagnosed and 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 as a urea cycle disorder expected to drive the urea cycle disorders treatment market forecast period. For instance, according to the article published in the Orphanet Journal of Rare Diseases in June 2017, the estimated prevalence of ornithine transcarbamylase deficiency ranges between 1/56,500 to 1/113,000 live births across the world.
Growing initiatives by key players and government, is expected to drive the growth in the forecast period
The rising initiatives by key players as well as governments for supporting patients suffering from UCD. For instance, in March 2017, Horizon Pharma plc launched UCD in Common, an initiative for people suffering from urea cycle disorder, to offer supportive, educational, and interactive resources to the patients living with UCD, their families, caregivers, and healthcare professionals.
Challenges in diagnosing and managing adult patients with urea cycle disorders in long-term therapies is likely to hinder the market growth
Early diagnosis is critical for effective treatment and to prevent long-term complications of hyperammonemia. The challenges of management of adults include, for example:
- Poor compliance to dietary and medical treatment, which can result in recurrent hospital admissions
- Severe neurological dysfunction
- The management of pregnancy and the postpartum period
- Access to multidisciplinary care peri-operatively
COVID-19 Impact Analysis
Following the COVID-19 outbreak in December 2019, the disease has spread to over 100 countries globally, and the World Health Organization has declared it a public health emergency. According to the World Health Organization’s report, the coronavirus (COVID-19) manifestation has resulted in more than 52.7 million infected individuals worldwide as of November 13, 2020.
The coronavirus (COVID-19) pandemic and resulting lockdown in various countries across the globe have impacted major companies' financial status in the urea cycle disorders treatment market. The private healthcare sector is one of the sectors, which the COVID-19 pandemic has majorly impacted. 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.
Market Segmentation Analysis
The glycerol phenylbutyrate segment is expected to hold largest share in this market segment
Glycerol phenylbutyrate (Ravicti) is an adjunctive therapy for chronic management of patients with urea cycle disorders. Ravicti is the first licensed oral liquid of phenylbutyrate and shares the same mechanism of action and metabolic pathway as sodium phenylbutyrate. Ravicti provides an alternative option in a formulation that may reduce the substantial treatment burden and unpleasant taste and odor associated with sodium phenylbutyrate.
The Ornithine transcarbamylase segment is expected to hold largest share in this market segment
Ornithine transcarbamylase deficiency is the most common urea cycle disorder in humans. It is an inherited disorder that causes toxic levels of ammonia to build up in the blood. Ornithine transcarbamylase, the defective enzyme in this disorder, is the final enzyme in the proximal portion of the urea cycle. For instance, according to the article published in the Orphanet Journal of Rare Diseases in June 2017, the estimated prevalence of ornithine transcarbamylase deficiency ranges between 1/56,500 to 1/113,000 live births across the world.
The hospital pharmacies segment is expected to hold largest share in this market segment
The hospital pharmacies held the largest share in the interleukin inhibitors market. This is mainly owing to increasing number of patients suffering from conditions which are treated in hospitals equipped with advanced infrastructure and adequate facilities. Moreover, growing number of hospitals along with adequate reimbursement policies is also contributing to the growth of this segment.
Market Geographical Share
North America region holds the largest market share of global urea cycle disorders treatment market
North America region is expected to hold a 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 to treat urea cycle disorders.
For instance, in March 2019, the University of Calgary Cumming School of Medicine (CSM) and Alberta Health Services (AHS) collaboratively initiated the clinical trial study on the treatment of urea cycle disorders with the help of gene therapy. The clinical trial study was conducted at the 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, including fussiness, sleepiness or sluggishness, vomiting, low body temperature, problems with posture, seizures, and others. Thus, such complications of long-term therapy are expected to hinder the market growth over the forecast period.
Competitive Landscape
The global urea cycle disorders treatment market is sightly competitive with presence of global companies. Some of the key players which are contributing to the growth of the market include Horizon Pharma plc. Lucane Pharma, Recordati Rare Diseases Inc., Synlogic, Acer Therapeutics, Ultragenyx Pharmaceutical Inc., Aeglea BioTherapeutics, Arcturus Therapeutics Holdings, Orpharma Pty Ltd, Bausch Health Companies, Inc. The major players are adopting several growth strategies such as product launches, acquisitions, and collaborations, which are contributing to the growth of the market globally.
Urea Cycle Disorders Treatment Market Key Companies to Watch
Recordati Rare Diseases Inc
Overview: Recordati Rare Diseases is a pharmaceutical company that provides treatment for patients with rare diseases. Created in 1990, Recordati Rare Diseases is one of the most active companies in the field of rare diseases.
Our specialty rare disease products are marketed directly by Recordati Rare Diseases in Europe, the Middle East, the U.S.A., Canada, Russia, Japan and Australia, in some Latin American countries, and through selected partners in other parts of the world. Recordati founded in Correggio, Reggio Emilia, Italy.
Product Portfolio: The Company’s portfolio comprised of include Hyperammonaemia, Acromegaly, Rare cancers, Cushing’s disease and syndrome, Wilson’s disease, Cystic fibrosis etc.
Why Purchase the Report?
- Visualize the composition of the urea cycle disorders treatment market segmentation by treatment type, enzyme deficiency type, distribution channel and region highlighting the key commercial assets and players.
- Identify commercial opportunities in urea cycle disorders treatment market by analyzing trends and co-development deals.
- Excel data sheet with thousands of data points of urea cycle disorders treatment market - level 4/5 segmentation.
- PDF report with the most relevant analysis cogently put together after exhaustive qualitative interviews and in-depth market study.
- Product mapping in excel for the key product of all major market players
The global urea cycle disorders treatment market report would provide an access to an approx. 61 market data table, 57 figures and 200 pages.
Target Audience
- Service Providers/ Buyers
- Industry Investors/Investment Bankers
- Education & Research Institutes
- Research Professionals
- Emerging Companies
- Manufacturers
