GLOBAL HEREDITARY OROTIC ACIDURIA MARKET SIZE AND SHARE ANALYSIS - GROWTH TRENDS AND FORECASTS (2024-2031)

Global hereditary orotic aciduria market was valued at US$ 357.9 Mn in 2023 and is expected to reach US$ 509.8 Mn by 2031, exhibiting at a compound annual growth rate (CAGR) of 4.5% from 2024 to 2031.

Hereditary orotic aciduria is a rare and autosomal recessive genetic disorder caused due to deficiency of an enzyme known as uracil-5'-phosphate decarboxylase. This enzyme is crucial for the breakdown of orotic acid, thereby leading to its accumulation and crystallization in the urine, if deficiency occurs. There are two main types of hereditary orotic aciduria - type I and type II. Type I is caused due to complete deficiency of uracil-5'-phosphate decarboxylase enzyme. Affected individuals experience severe metabolic consequences and most do not survive infancy if left untreated. Type II is relatively mild and is caused due to partial deficiency of this enzyme.

Individuals with type II hereditary orotic aciduria may experience mild developmental delays, seizures or intellectual disabilities, if untreated. The standard treatment involves restricting dietary intake of pyrimidines that break down into orotic acid. Supplementing diet with uridine monophosphate also aids in decreasing orotic acid levels. While treatment helps manage symptoms, it does not cure the condition. Prenatal diagnosis through testing fetal deoxyribonucleic acid (DNA) obtained through amniocentesis or chorionic villus sampling can help detect the condition. Early diagnosis and treatment can help avoid severe complications and reduce long term impact on growth and development in affected individuals.

Analyst View:

Global hereditary orotic aciduria market is expected to witness significant growth over the forecast period. Increasing incidence of hereditary orotic aciduria around the world will be a major driver boosting demands for effective treatment options. Rising awareness regarding the availability of diagnostic tests and management therapies for the condition will further augment the market growth.

North America  dominates the hereditary orotic aciduria market and is expected to retain its position in the near future. High healthcare expenditure and presence of advanced healthcare infrastructure facilitates early detection and treatment in the region. Europe also captures a substantial market share due to growing research and development (R&D) activities for developing novel drugs and supportive government policies.

However, lack of established clinical management guidelines may hamper the standardization of treatment protocols to some extent. High costs that are associated with enzyme replacement therapies and surgical procedures can limit the market potential in price-sensitive regions. Hesitancy regarding long-term usage of medicines poses challenges to the market players as well.

The market in Asia Pacific is anticipated to witness lucrative opportunities with rapid economic development and escalating medical tourism in India and China. Rising per capita incomes have improved access to advanced diagnostics and specialized treatments in these emerging countries. Progress in genetic research would pave way for more effective therapies

Global Hereditary Orotic Aciduria Market Regional Insights

• North America: North America is the largest market for hereditary ortic aciduria, accounting for a share of over 33.5% in 2023. North America remains the dominant region in the global hereditary orotic aciduria market. The U.S. accounts for the major share due  to the high diagnosis and treatment rates. Well-established healthcare infrastructure and rising healthcare expenditure have enabled early diagnosis and management of the condition. Presence of leading market players in the region drives significant research activities for developing advanced treatment options. Several major players have their headquarters in the U.S. and invest heavily in R&D. This has resulted in the early availability of new treatment drugs in the region.
• Europe: Europe is the second-largest market for hereditary ortic aciduria, accounting for a share of over 26.1% in 2023. Europe holds the second position in terms of market share after North America. Countries such as Germany, U.K.,  France possess well-established healthcare systems. Universal healthcare has enabled widespread diagnosis and treatment uptake. Germany has the highest number of patients diagnosed with hereditary orotic aciduria conditions. It is a major manufacturing hub for biopharmaceutical companies. The export-import of drugs between European countries also supports market expansion. However, pricing pressures from generics may hamper the market revenues to some extent over the forecast period.
• Asia Pacific: Asia Pacific is the fastest-growing market for hereditary ortic aciduria, accounting for a share of over 20.3% in 2023. Asia Pacific is identified as the fastest growing regional market. The growth is due to the rising prevalence of hereditary orotic aciduria disorders due to improving diagnostic capabilities. Rapid economic development across major Asian countries such as China and India has led to increased healthcare investments. This has positively impacted the regional market. China dominates the Asia Pacific market due to its huge population size and growing medical needs. Higher patient volumes attract new drug launches and clinical trials. The market is also gaining from increasing healthcare affordability due to expanded insurance coverage. Moreover, improving access to diagnostics and therapies across rural areas is fueling the regional growth.

Figure 1. Global Hereditary Orotic Aciduria Market Share (%), By Region, 2024

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Global Hereditary Orotic Aciduria Market Drivers:

• Government support in the form of funding for rare disease research: Government support is playing a crucial role in driving the growth of the global hereditary orotic aciduria market. Many countries have recognized rare diseases as a serious public health concern and have launched various initiatives and programs to support research in these areas. For instance, in January 2020, the National Health Commission established an office for the National Network for Collaboration in Rare Disease Diagnosis and Treatment in order to enhance the organization and management of the National Network for Rare Disease Diagnosis and Treatment, to facilitate collaboration and to effectively fulfil the overall role of a collaborative network.
• Focus on the development of orphan drugs: Development of orphan drugs is a major factor driving the growth of the global hereditary orotic aciduria market. Orphan drugs are pharmaceutical agents intended for rare medical conditions termed as orphan diseases, which affect a small percentage of population. Hereditary orotic aciduria is an ultra-rare genetic disorder characterized by an inability to metabolize orotic acid.
• Increasing research and development (R&D) investments by key players: Increasing investments in research and development by leading pharmaceutical players is a major factor driving the growth of the global hereditary orotic aciduria market. Hereditary orotic aciduria is a rare metabolic disorder characterized by an inability to break down orotic acid, which leads to a buildup of orotic acid in the blood and urine. As there are no approved treatments available, it represents an area of high unmet need.

Global Hereditary Orotic Aciduria Market Opportunities:

• Growth opportunities in developing countries: Developing countries present a significant growth opportunity for the global hereditary orotic aciduria market. Countries such as India, China, Brazil, and others are experiencing rapid economic development and population growth. As incomes rise in these nations, access to advanced healthcare and diagnostic testing is also improving. With larger populations that are becoming more able to afford treatment, developing markets allow for substantial scale and market expansion potential in the near future.
• Increasing awareness about rare disease: Increasing awareness about rare disease is expected to drive the growth of the market over the forecast period. For instance, in November 2023, 13 Canadian rare disease organizations, in partnership with Takeda Canada Inc., a pharmaceutical company united to launch I Am Number 12, to raise awareness of the 1 in 12 Canadians who were affected by a rare disease in their lifetime. The campaign highlights the challenges and needs of people living with rare diseases, and how collectively, rare diseases are not really that rare.

Global Hereditary Orotic Aciduria Market Trends:

• Shift towards personalized medicine: The rise of personalized medicine has had a significant influence on the global hereditary orotic aciduria market. Personalized medicine aims to tailor medical treatment to an individual's genetic characteristics. With the advancement of genetic testing technologies, it is possible to determine if a person carries the genetic mutations that cause hereditary orotic aciduria. This ability to accurately diagnose the condition at the genetic level has driven demand for genetic testing services related to this rare disease. More patients are able to receive a confirmed diagnosis rather than relying solely on symptoms and clinical testing. Armed with a precise genetic diagnosis, physicians can recommend personalized treatment plans for patients. This may involve certain dietary modifications and vitamin supplementation that are tailored to an individual's requirements, or monitoring of symptoms. In some cases, gene therapy is also emerging as a potential customized treatment as scientists gain deeper understanding of the genetic factors behind hereditary orotic aciduria. The demand for monitoring tests and medicines that target the disease pathway has increased as doctors move towards individualized care.

Global Hereditary Orotic Aciduria Market Restraints:

• High cost of treatment: Orotic aciduria is a rare genetic disorder that requires lifelong treatment and management. The treatments available are largely supportive and aimed at managing symptoms. This includes a special low-protein diet that helps reduce excess ammonia production in the body. Such a specialized diet needs to be strictly followed from birth, which causes significant financial burden on patients and their families. Developing customized dietary plans and constantly monitoring their implementation requires frequent consultations with expert dieticians and nutritional therapists. This makes the overall treatment very expensive.
• Lack of awareness in developing regions: Lack of awareness about hereditary orotic aciduria and its treatment options in developing regions is significantly restraining the growth of the global hereditary orotic aciduria market. In many underdeveloped parts of the world, there is very little knowledge about lung cancer and its symptoms amongst the general population as well as healthcare workers. This is largely due to the lack of patient education programs and resources to spread information about such a critical disease. People in rural and remote areas remain highly uninformed about the early signs and risk factors of lung cancer. As a result, most cases go undiagnosed until it reaches advanced stages, thereby reducing the chances of effective surgery.
• Counterbalances: As a result, the key market players need to focus on spreading awareness about hereditary orotic aciduria and its treatment options.

Recent Developments

• In October 2023, the University of Oxford and Harrington Discovery Institute at University Hospitals in Cleveland, Ohio launch  the Oxford-Harrington Rare Disease Centre Therapeutics Accelerator with an official signing event at the University of Oxford. The event was hosted by the Oxford-Harrington Rare Disease Centre (OHC), a partnership established in 2019 between the University of Oxford and Harrington Discovery Institute. Attendance at the signing included leaders from OHC, the University of Oxford, the Harrington family, University Hospitals, and Oxford Science Enterprises.

Partnerships

• In January 2022, Pfizer Inc., a pharmaceutical company and Beam, a pharmaceutical company, entered into an exclusive multi-target research collaboration to advance novel in vivo base editing programs for a range of rare diseases.

Figure 2. Global Hereditary Orotic Aciduria Market Share (%), By Indication, 2024

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Top companies in Global Hereditary Orotic Aciduria Market

• Merck & Co. Inc.
• SERB Pharmaceuticals

Definition: Hereditary orotic aciduria is an extremely rare genetic disorder. When untreated, affected infants can develop a blood (hematologic) disorder called megaloblastic anemia as well as failure to thrive, susceptibility to infection, and orotic acid crystals in the urine (crystalluria) resulting from excretion of orotic acid in the urine.