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Rising Demand For Gene Therapies Driving The Growth Of The Market Due To Increasing Prevalence Of Genetic Disorders: A Significant Driver Propelling The Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market In 2025
Ornithine Transcarbamylase Deficiency (OTC Deficiency)
What Is the Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Size and Projected Growth Rate?
In the past few years, there has been significant growth in the market size for ornithine transcarbamylase deficiency (OTC deficiency). The market, valued at $0.83 billion in 2024, is expected to escalate to $0.89 billion in 2025, indicating a compound annual growth rate (CAGR) of 7.1%. This upward trend during the historical term has been driven by greater recognition and understanding of uncommon metabolic disorders, advancements in genetic screening and diagnostic tools, enhancements in neonatal screening programs, an increased occurrence of genetic testing in rare diseases, as well as further improvements in neonatal screening initiatives.
Anticipations of robust expansion are being seen in the market for ornithine transcarbamylase deficiency (OTC deficiency), with predictions estimating it to reach $1.16 billion by 2029, maintaining a compound annual growth rate (CAGR) of 6.8%. Factors contributing to this predicted growth during the forecast period encompass heightened awareness and diagnosis, an increase in the occurrence of urea cycle disorders, intensified attention on treatments for rare diseases, advancements in research and clinical trials, and better access to treatment for patients. Key market trends in this period are expected to include progression in gene therapies and gene editing technologies, the development of enzyme replacement therapies, alliances for research and cooperative initiatives, advancement in diagnostic tool technology, and government financial support and incentives for orphan drugs.
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What Are the Major Segments in the Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market?
The ornithine transcarbamylase deficiency (OTC deficiency) market covered in this report is segmented -
1) By Product Type: DTX-301, SEL-313, SHP-641, PRX-OTC
2) By Treatment Type: Gene Therapy, Liver Transplant, Dietary Management, Other Treatement Type
3) By Diagnosis: Genetic Testing, Ammonia Level Testing
4) By End-User: Hospitals, Specialty Clinics, Research Institutes, Other End Users
Subsegmentss:
1) By DTX-301: Adeno-Associated Virus (AAV)-Based Gene Therapy, Intravenous Dosing Formulation
2) By SEL-313: Immune Tolerance-Inducing Therapy, Enzyme Replacement Therapy (ERT) Combination
3) By SHP-641: Small Molecule Therapy, mRNA-Based Therapeutic Approach
4) By PRX-OTC: Recombinant Enzyme Replacement Therapy, Protein Engineering-Based Therapy
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What Are The Driving Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Evolution?
The escalation in the need for gene therapies is projected to catalyze the expansion of the ornithine transcarbamylase deficiency market in the future. Gene therapy is a medical method or intervention aimed at modifying or manipulating an individual's genetic makeup to treat or ward off diseases. The rising prevalence of genetic disorders, which often possess few treatment alternatives, is creating a demand for gene therapies. This is due to their potential to address the underlying problem rather than merely controlling symptoms. By rectifying the genetic anomaly that interrupts the urea cycle, gene therapies illustrate their potential in treating ornithine transcarbamylase deficiency, offering specialized treatment to regain metabolic activity and halt the harmful buildup of ammonia. For example, in January 2024, as per the American Society of Gene & Cell Therapy, there was a 10% quarter-on-quarter increase in the number of gene therapies in Phase III in Q4 of 2023 - a first time increase since Q3 2022. Hence, the escalating demand for gene therapies is fueling the expansion of the ornithine transcarbamylase deficiency market.
Which Firms Dominate The Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Segments?
Major companies operating in the ornithine transcarbamylase deficiency (OTC deficiency) market are Thermo Fisher Scientific, Novartis AG, GlaxoSmithKline plc, Roche Holding AG, Eli Lilly and Company, Amgen Inc., Merck & Co. Inc, Biogen Idec, Bausch Health Companies Inc, Alexion Pharmaceuticals, Sobi, BioMarin Pharmaceutical Inc, Glenmark Pharmaceuticals Ltd., Nutricia, Vertex Pharmaceuticals Incorporated, Ultragenyx Pharmaceutical Inc, Arcturus Therapeutics, Regenxbio, OrphanPacific Inc, Ucyclyd Pharma Inc.
What Trends Are Expected to Dominate the Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market in the Next 5 Years?
Primary corporations in the ornithine transcarbamylase deficiency (OTC Deficiency) market are concentrating on designing unique treatments like mRNA therapeutics to boost the effectiveness of treatment and tackle the inherent genetic reason for OTC deficiency. mRNA therapeutics are a category of treatments that utilize messenger RNA (mRNA) to instruct cells to generate certain proteins for therapeutic objectives. These treatments utilize the body's innate protein synthesis equipment to counter diseases, encompassing genetic disorders, infectious diseases, and cancers. For instance, in June 2023, Arcturus Therapeutics Holdings Inc., a biotech firm based in the United States, was awarded Fast Track Designation from the U.S. Food and Drug Administration (FDA) for its mRNA therapeutic candidate for OTC deficiency, ARCT-810. This designation aims to quicken the development and review of the drug, providing potential advantages such as more regular discussions with the FDA and priority assessment. ARCT-810 is contrived to empower patients with OTC deficiency, a urea cycle disorder, to generate a functional OTC enzyme in their liver cells, rectifying the inherent cause of the disorder and potentially enhancing their lifestyle.
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Which Is The Largest Region In The Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market?
North America was the largest region in the ornithine transcarbamylase deficiency (OTC Deficiency) market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in the ornithine transcarbamylase deficiency (OTC deficiency) market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.
Frequently Asked Questions:
1. What Is the Market Size and Growth Rate of the Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market?
2. What is the CAGR expected in the Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market?
3. What Are the Key Innovations Transforming the Ornithine Transcarbamylase Deficiency (OTC Deficiency) Industry?
4. Which Region Is Leading the Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market?
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