Urea Cycle Disorder Treatment Market: Global Size, Share, Trends, Growth and Forecast 2022-2032

Urea Cycle Disorder Treatment Market Insights

A rare genetic disorder known as Urea cycle disorder (UCD) affects the body's ability to process protein. The liver is home to a series of biochemical reactions called the urea cycle that is responsible for flushing excess nitrogen from the body. A mutation in one of six genes that encode an enzyme that controls cycling is responsible for the disease. Circulation is impaired and toxic ammonia levels rise in the blood, which can cause severe neurological problems, coma, and even death. UCD is uncommon and its severity and symptoms can vary depending on the type of disease and age at the first onset. Ornithine transcarbamylase deficiency (OTC), arginine succinic acidemia (ASA), and citrullinemia are the types of urea cycle disorders.

Imaging studies, genetic testing, and blood and urine tests are usually used to diagnose UCD. Prevention of serious health problems related to it requires timely diagnosis and treatment. Medications, dietary changes, and sometimes a liver transplant are usually part of the treatment plan. Low-protein diets and specific amino acid supplements help the body process proteins properly and are the mainstay of treatment for urea cycle disorders. Patients with UCD require long-term follow-up to ensure that their smelling salt levels are within a protected range. Depending on the severity of the disease, the age of the patient, and the response to treatment, it may be necessary to adjust the treatment. In severe cases, a liver transplant may be required to restore normal urea cycle activity and replace the damaged liver.

Urea Cycle Disorder Treatment Market Share

The global urea cycle disorder (UCD) treatment market is expected to grow steadily at a CAGR of 11.6%. In 2020, the market was valued at $1.3 billion and is projected to reach $2.2 billion by 2025. The growing need for personalized medicine, advances in diagnostic and therapeutic technologies, government initiatives and support, and the increasing prevalence of UCD are some of the factors driving the market expansion. BioMarin Pharmaceutical, Inc., Ultragenyx Pharmaceutical, Inc., and Amicus Therapeutics, Inc. are only a few companies that develop and manufacture products for the treatment of urea cycle disorders.

Urea Cycle Disorder Treatment Key Drivers

Several factors are driving the growth of the urea cycle disorder treatment market:

1. Reimbursement policies: The market will also be affected by changes in reimbursement policies, such as insurance coverage for urea cycle disorders treatment.
2. Development of new treatments: The development of new treatments such as drugs and liver transplantation can drive the growth of the market.
3. Government initiatives and support: Governments and regulatory agencies around the world have introduced various programs and initiatives to support the development of new treatments for rare diseases such as UCD. As a result, more funds are spent on research and development, thereby helping the market to grow further.
4. Growing demand for personalized medicine: As the need for personalized medicine increases, customized treatments for UCD have been developed.
5. Increase in healthcare spending: Increasing healthcare expenditure, especially for rare diseases, may also drive the growth of the urea cycle disorders market.
6. Increasing collaborations and partnerships: New treatments for UCD are being developed through partnerships and collaborations between different companies, academic institutions, and research organizations, accelerating market expansion.
7. Increasing prevalence of UCDs: With the increasing prevalence of UCD worldwide, treatment for UCD is in high demand. Increasing public awareness of these diseases and the availability of diagnostic tools are also contributing to market expansion.
8. Research and development: Companies manufacturing products for the treatment of urea cycle disorders can also leverage their investments in research and development to drive market expansion.
9. Technological advancements: Early detection and diagnosis of UCD has become easier with the development of advanced diagnostic methods such as newborn screening programs. The UCD treatment market has also grown due to new treatment options such as enzyme replacement therapy and gene therapy.

Urea Cycle Disorder Treatment Market Segmentation

The global market for urea cycle disorder treatments can be segmented in many ways such as:

1. By type of urea cycle disorder: Ornithine transcarbamylase deficiency (OTC), Argininosuccinic acidemia (ASA), Citrullinemia
2. By treatment modality: Medications, Diet, and dietary supplements
3. By age of the patient: Pediatric, Adult
4. By the stage of the disease: Mild, Moderate, Severe
5. By type of healthcare setting: Hospitals, Outpatient clinics, Home Healthcare
6. By geography: North America, Europe, Asia-Pacific, Rest of the world (RoW)

Urea Cycle Disorder Treatment Market Regional Synopsis

North America, Europe, Asia Pacific, and the Rest of the World constitute the urea cycle disorder (UCD) treatment market. North America accounted for the largest share of the UCD treatment market in 2020 and this trend is expected to continue as a result of the presence of improved healthcare infrastructure, increasing incidence of UCD, increasing awareness of the disease, and availability of advanced diagnostic and treatment options.

Europe is the second-largest UCD treatment market and is expected to witness significant expansion during the forecast period. The presence of large pharmaceutical companies in the region, the spread of UCD, and the growing demand for personalized medicine are driving the expansion. Asia Pacific is expected to witness the highest growth rate due to rising demand for advanced treatments, expanding healthcare infrastructure, and rising awareness of UCD. Additionally, the market expansion in the region is expected to be fueled by government initiatives to increase access to healthcare services and expand partnerships and collaborations among pharmaceutical companies.

Africa, the Middle East, and Latin America are included in the Rest of the World (RoW) section. The increasing prevalence of UCD, rising healthcare costs, and availability of state-of-the-art diagnostic and therapeutic options are key factors that will drive moderate growth of the RoW segment over the forecast period.

Urea Cycle Disorder Treatment market share in Japan

Although the urea cycle disorder (UCD) treatment market in Japan is relatively small, it is steadily expanding as UCD diagnosis and awareness improve. It was valued at approximately $13 million in 2020 and is expected to grow at a CAGR of approximately 4.5% during the period 2021-2026. Nippon Kayaku Co. Ltd., Sumitomo Dainippon Pharma Co. Ltd., and Recordati Rare Diseases Japan K.K. are the major players in the Japanese UCD treatment market. These companies provide a variety of medications and supplements for UCD treatment, and they are anticipated to maintain their position as market leaders throughout the forecast period.

Urea Cycle Disorder Treatment Market Challenges

There are several challenges facing the urea cycle disorder treatment market.

1. Adverse side effects of treatment: Side effects of some UCD treatments, such as gastrointestinal problems, can impair patient compliance and treatment outcomes.
2. High cost of treatment: Because treatment of urea cycle disorders often involves drug therapy, dietary changes, and sometimes liver transplantation, it can be expensive. Therefore, patients and their families may face financial difficulties that limit the availability of UCD treatment, especially in developing countries.
3. Limited availability of treatment options: There are several different treatment options for UCD, but many are still in their infancy or not widely available. As a result, patients and healthcare providers have fewer options.
4. Limited awareness: Because UCDs are uncommon and poorly understood by healthcare professionals, diagnosis and treatment can be difficult for patients. Delayed diagnosis and treatment can lead to serious health complications. It also creates challenges for companies developing products to treat these diseases, as the target patient population may be limited.
5. Rare disease: As UCDs are rare genetic disorders, it can be difficult for companies to justify the high costs associated with developing new treatments for these conditions.
6. Reimbursement issues: Patients and their families may face difficulties because insurance coverage for the treatment of urea cycle disorders may be limited.
7. Stringent regulatory requirements: Strict regulatory requirements must be met before UCD treatments are developed and approved making it harder and more expensive to get new treatments to market.

Urea Cycle Disorder Treatment Market Key Players

Many different companies manufacture and sell urea cycle disorder treatments, and the market is highly competitive. Some of the key players in the market include:

1. Amicus Therapeutics, Inc.: Amicus, a biopharmaceutical company, creates and manufactures medicines for rare and rare diseases, such as urea cycle disorders.
2. BioMarin Pharmaceutical, Inc.: Products for the treatment of rare genetic diseases such as urea cycle disorders are developed and manufactured by the biotechnology company BioMarin.
3. Horizon Therapeutics: Drugs for rheumatism and rare diseases such as UCD are being developed by biopharmaceutical company Horizon Therapeutics. Their UCD treatment, RAVICTI (glyceryl phenylbutyrate), is an oral medication indicated for adults and children over two years of age with UCD.
4. Nippon Kayaku Co. Ltd.: Ammonia scavengers and amino acid supplements are two UCD medications developed and manufactured by the Japanese pharmaceutical company Nippon Kayaku. Citrulline pellet and Arginine pellet are their brand names.
5. Recordati Rare Diseases: Recordati Rare Diseases, a biopharmaceutical company, develops and markets medicines for rare and rare diseases such as UCD. RAVICTI and Carbaglu (carglumic acid) are two of their UCD treatment products.
6. Summit Therapeutics: Summit Therapeutics, a biopharmaceutical company, is developing treatments for rare diseases such as UCD. Their UCD treatment product, Ezutromid, is a small molecule drug candidate designed to treat UCD by reducing the production of urea in the liver.
7. Takeda Pharmaceutical Company Limited: Urea cycle disorders are just one of the many diseases that the global pharmaceutical company Takeda is working to treat. Takeda creates and manufactures products for these conditions.
8. Ultragenyx Pharmaceutical: Ultragenyx Pharmaceutical, a biopharmaceutical company, focuses on the development of medicines for the treatment of very rare or extremely rare genetic diseases such as UCD. Their UCD treatment, BUPHENYL (sodium phenylbutyrate), is an oral medication for adults and children with UCD.