Global Orotic Aciduria Market To Grow At A Steady CAGR of 5.10% During The Forecast Period

14-Jun-2023 | Zion Market Research

The global orotic aciduria market size was worth around USD 341.22 million in 2022 and is predicted to grow to around USD 507.96 million by 2030 with a compound annual growth rate (CAGR) of roughly 5.10% between 2023 and 2030.

Global Orotic Aciduria Market Size

The orotic aciduria market refers to the economic landscape and scenarios surrounding the diagnosis, treatment, and management of orotic aciduria. It is a genetic disorder that is characterized by impaired synthesis of pyrimidine. The industry encompasses various stakeholders and includes pharmaceutical companies, research organizations, healthcare providers, regulatory authorities, patient advocacy groups, and patients themselves. It deals with the development and availability of diagnostic tests, therapeutic interventions, and supportive care measures specifically designed for individuals with orotic aciduria. It is noteworthy, that as of 2023, there is a very small set of patients detected with this condition and hence there is a significant lack of large-scale research and trials surrounding the condition.

This review is based on a report by Zion Market Research, titled "Orotic Aciduria Market By Drug Type (Uridine Monophosphate, Cytidine Monophosphate, And Others), By Type (Type II And Type I), And By Region - Global And Regional Industry Overview, Market Intelligence, Comprehensive Analysis, Historical Data, And Forecasts 2023 – 2030"- Report at

The global orotic aciduria market is projected to grow owing to the rising interest of the research organizations especially toward rare conditions, as a main segment of study and orotic aciduria automatically becomes a part of this group. Large pharmaceutical companies with the aid of research groups are focusing on studying several underlying conditions that cause the onset of such conditions. They are also diverting resources toward improving diagnostic tools and educating the healthcare community about the several classes of rare genetic disorders and effective ways of diagnosing them.

In addition to this, such events and activities have received sufficient government assistance in terms of funds, grants, and incentives. This has led to higher growth in the orotic aciduria industry. In some countries, orphan drug designations and incentives are provided to encourage pharmaceutical companies to invest in rare disease therapies. A crucial role is played by advocacy organizations and patient support groups in educating the general public about issues that impact their health. In recent times, these groups have undertaken massive large-scale drives to create public awareness and such efforts are likely to contribute to industry growth.

The global orotic aciduria industry also faces certain growth restrictions with the primary being lack of a sufficient pool of patients to accurately conclude important aspects of the condition. These criteria include the cause of the disease, effective diagnosis, and efficient treatment programs. Additionally, since the disease is extremely rare, the cost of available treatment is high and hence can be undertaken by only a limited set of patients. Other factors such as lack of awareness, limited medical reimbursement, and complex regulatory pathway are also areas of great concern for the industry players.

The investments in personalized medicine approach may provide several growth opportunities while inaccurate diagnosis could challenge the market expansion.

Global Orotic Aciduria Market Size

The global orotic aciduria market is segmented based on drug type, type, and region.

Based on drug type, the global market segments are uridine monophosphate, cytidine monophosphate, and others. The industry registered the highest growth in the uridine monophosphate segment in 2022 since it is one of the most widely used drugs in treating the condition. It is typically administered as a therapeutic agent and it can bypass the deficiency of the enzyme uridine monophosphate synthase (UMPS). By providing this drug, medical professionals can restore the impaired pyrimidine synthesis pathway and aid in the reduction of orotic acid accumulation. However, it should be noted that the specific medication and treatment plan depends on the patient and the intensity of the disease. As of 2018, only 20 patients with this condition had been diagnosed.

Based on type, the orotic aciduria industry segments are type II and type I. Out of the two types, the latter is more common and it is also known as UMP synthase deficiency. The counterpart is referred to as hereditary orotic aciduria II or orotate phosphoribosyltransferase deficiency and this version is much rarer than type I. In type II, there is a deficiency in the enzyme orotate phosphoribosyltransferase (OPRT), which affects the conversion of orotic acid to orotidine-5'-monophosphate (OMP) in the pyrimidine synthesis pathway. As per the National Institute of Health, there are around 7000 rare conditions that are noted in the medical literature.

The global orotic aciduria market is expected to witness the highest growth in North America driven by the presence of robust medical infrastructure and research ecosystem. These nations are equipped with better diagnostic capabilities, access to specialized care, and ongoing research initiatives. Other factors such as better diagnostic capabilities, access to specialized care, and ongoing research initiatives.

Growth in Europe is projected to be led by regional and national initiatives supporting research, diagnosis, and treatment of rare diseases, including orotic aciduria. European Reference Networks (ERNs) facilitate collaboration among healthcare professionals across different countries. The European Medicines Agency (EMA) provides guidelines and incentives for orphan drug development. Access to specialized care, including metabolic centers, may vary across different European countries.

Recent Developments:

  • In February 2023, the Center for Drug Evaluation and Research (CDER) announced the launch of the Accelerating Rare Disease Cures (ARC) Program which is expected to leverage the collective expertise of CDER
  • In August 2022, The Karnataka Government of India launched a new research and training unit focusing on rare diseases

Orotic Aciduria Market: Competitive Analysis

The global orotic aciduria market is led by players like:

  • Alexion Pharmaceuticals
  • Sanofi Genzyme
  • Roche Holding AG
  • Novartis AG
  • Pfizer Inc.
  • Shire
  • BioMarin Pharmaceutical Inc.
  • Vertex Pharmaceuticals Incorporated
  • Ultragenyx Pharmaceutical Inc.
  • Horizon Therapeutics
  • CSL Behring
  • Sarepta Therapeutics
  • Regeneron Pharmaceuticals Inc.
  • Moderna Inc.
  • bluebird bio
  • Sangamo Therapeutics
  • Amicus Therapeutics
  • Orchard Therapeutics
  • Abeona Therapeutics
  • PTC Therapeutics
  • Akcea Therapeutics
  • Catabasis Pharmaceuticals
  • Translate Bio
  • Homology Medicines
  • Harmony Biosciences.

The global orotic aciduria market is segmented as follows:

By Drug Type

  • Uridine Monophosphate
  • Cytidine Monophosphate
  • Others

By Type

  • Type II
  • Type I

By Region

  • North America
    • The U.S.
    • Canada
  • Europe
    • France 
    • The UK
    • Spain
    • Germany
    • Italy
    • Rest of Europe
  • Asia Pacific
    • China
    • Japan
    • India
    • South Korea
    • Southeast Asia
    • Rest of Asia Pacific
  • Latin America
    • Brazil
    • Mexico
    • Rest of Latin America
  • Middle East & Africa
    • GCC
    • South Africa
    • Rest of Middle East & Africa

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