Ornithine Transcarbamylase Deficiency (OTC Deficiency) Industry Valued at $1.16 Billion by 2029 With CAGR of 6.8% | Size and Share Insights
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What Is the Forecast for the Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market from 2024 to 2025?
The OTC deficiency market size has seen robust growth over the past few years. It is predicted to expand from $0.83 billion in 2024 to $0.89 billion in 2025, signaling a Compound Annual Growth Rate (CAGR) of 7.1%. Factors leading to remarkable growth in the historical period include heightened awareness of uncommon metabolic disorders, advancements in genetic screening and diagnostic methods, enhanced neonatal screening programs, the rise in genetic testing for rare diseases, and improvements in neonatal screening procedures.
What Is the Projected Market Size of the Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market?
The market size for ornithine transcarbamylase deficiency (OTC deficiency) is anticipated to experience robust expansion in the upcoming years, reaching $1.16 billion by 2029 with a Compound Annual Growth Rate (CAGR) of 6.8%. The predicted growth during the forecast period is ascribed to the expanding awareness and detection, an escalating incidence of urea cycle disorders, increasing emphasis on rare disease treatments, augmented research and clinical trials, and enhanced access to treatments for patients. Noteworthy trends during the forecast period encompass advancements in the field of genetic therapies and gene-editing methodologies, the development of enzyme replacement therapies, collective research efforts and partnerships, technological progress in diagnostic instruments, and government investments and incentives for orphan drugs.
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Who are the Major Competitors in the Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Outlook?
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 Is Fueling Growth in the Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market?
The anticipated expansion of the ornithine transcarbamylase deficiency market is largely attributed to the increasing need for gene therapies. Gene therapy involves modifying or changing an individual’s genetic material as a means to cure or hinder diseases. The surging interest in gene therapies is largely due to the growing occurrence of genetic disorders. These disorders often have scarce treatment options, making gene therapy an expected solution as it directly targets the primary issue instead of simply managing the symptoms. Gene therapies are particularly promising for disorders like ornithine transcarbamylase deficiency where a gene mutation disrupts the urea cycle. By correcting this mutation, gene therapy offers a specific solution to restore metabolic functionality and prevent the harmful buildup of ammonia. Notably, the American Society of Gene & Cell Therapy reported in January 2024 that the number of gene therapies in Phase III had grown by 10% in Q4 2023. This was the first increase since Q3 2022, indicating the escalating demand for gene therapies is fueling the growth of the ornithine transcarbamylase deficiency market.
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Which Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Segments Are Growing the Fastest?
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
Which Industry Trends Are Shaping the Future of the Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market?
The key players engaged in the ornithine transcarbamylase deficiency (OTC Deficiency) market are actively working on the creation of groundbreaking treatments like mRNA therapeutics to increase the quality of treatment and tackle the genetic root of OTC deficiency. Therapies that utilize messenger RNA (mRNA) are known as mRNA therapeutics, they function by directing cells to generate specific proteins for medicinal purposes. These cutting-edge therapies take advantage of the body’s innate protein synthesis system to address diseases including genetic conditions, infectious diseases, and even cancers. For instance, Arcturus Therapeutics Holdings Inc., a biotechnology firm based in the U.S, was granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) in June 2023 for their mRNA therapeutic prospect, ARCT-810, aimed at OTC deficiency. This designation seeks to hurry up the development and evaluation process of the drug, with possible advantages such as increased interaction with the FDA and prioritized review. The goal of ARCT-810 is to help OTC deficiency patients, individuals who suffer from a urea cycle disorder, to generate functional OTC enzyme in their hepatic cells, addressing the fundamental cause of the ailment and potentially enhancing their life quality.
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Which Countries Are Leading 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.
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This Report Delivers Insight On:
1. How big is the ornithine transcarbamylase deficiency (otc deficiency) market, and how is it changing globally?
2. Who are the major companies in the ornithine transcarbamylase deficiency (otc deficiency) market, and how are they performing?
3. What are the key opportunities and risks in the ornithine transcarbamylase deficiency (otc deficiency) market right now?
4. Which products or customer segments are growing the most in the ornithine transcarbamylase deficiency (otc deficiency) market?
5. What factors are helping or slowing down the growth of the ornithine transcarbamylase deficiency (otc deficiency) market?
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