by The urea cycle is a series of biochemical reactions that removes ammonia from the body and converts it into urea, which is less toxic. Urea cycle disorders occur due to genetic defects in any of the enzymes required for carrying out these reactions. Common symptoms of urea cycle disorders include lethargy, vomiting, seizures, developmental delays and neurological complications. Currently available treatment options are limited to medication, diet management and liver transplantation. However, rising research and development activities focusing on developing innovative therapies such as gene therapy are expected to offer new hope. Biotech companies are conducting extensive research to develop optimized gene transfer methods for long-term expression of missing or defective urea cycle enzymes.
The global U.S. urea cycle disorders treatment market is estimated to be valued at US$ 587.52 Mn in 2024 and is expected to exhibit a CAGR of 5.3% over the forecast period 2024 to 2031, as highlighted in a new report published by Coherent Market Insights.
Market Dynamics:
Rising Incidence of Urea Cycle Disorders: According to the National Organization for Rare Disorders, the estimated prevalence of urea cycle disorders is 1 in 30,000 live births in the United States. The increasing incidence of these inherited disorders along with growing diagnosis rates is expected to drive the market growth during the forecast period.
Focus on Developing Gene Therapies: Major players in the market are investing heavily in researching and developing advanced gene therapies for urea cycle disorders. For instance, Astellas Pharma Inc. and its subsidiary Agistix are working on developing an adeno-associated virus (AAV) gene therapy for ornithine transcarbamylase (OTC) deficiency, a common urea cycle disorder. Efforts towards developing innovative treatment options are expected to present lucrative opportunities in the market.
Segment Analysis
The U.S. Urea Cycle Disorders Treatment market is dominated by the oral route of administration segment. Oral medications are the first line of treatment for urea cycle disorders and account for over 60% of the market share. They are easy to administer and have good patient compliance compared to other routes. However, the intravenous segment is growing at a higher rate owing to the increasing usage of newer therapies like gene and enzyme replacement therapies which require intravenous administration.
PEST Analysis
Political: Healthcare reforms and policies that support funding for rare diseases positively impact the market. However, stringent regulations for approval of new drugs can hamper growth.
Economic: High treatment costs associated with urea cycle disorders burden the patients and healthcare systems. Though rare, the lifelong nature of treatment increases overall expenditure.
Social: Growing medical support communities create awareness about early diagnosis and management of these inherited metabolic conditions. This influences timely intervention and better outcomes.
Technological: Advancements in screening methods aid early diagnosis. New drug delivery approaches, gene and genetic therapies promise more effective solutions and drive the market.
Key Takeaways
The U.S. Urea Cycle Disorders Treatment Market Growth is expected to witness high over the forecast period driven by factors such as increasing diagnosis rates, availability of newer treatment options and supportive regulatory environment. The global U.S. urea cycle disorders treatment market is estimated to be valued at US$ 587.52 Mn in 2024 and is expected to exhibit a CAGR of 5.3% over the forecast period 2024 to 2031.
The western region currently dominates the market owing to concentration of major players, advanced healthcare infrastructure and high patient awareness. However, the southern region is emerging as the fastest growing market due to expanding health insurance coverage and improving access to care in this region.
Key players operating in the U.S. Urea Cycle Disorders Treatment market are Kawasumi Laboratories Inc., Blood Purification Technologies Inc., AWAK Technologies Pte. Ltd, Triomed AB, Asahi Kasei Medical Vo. Ltd, US Kidney Research Corporation, Merit Medical Systems, NIPRO Medical Corporation, Fresenius SE & Co. KGaA. Major players are focusing on new product launches, partnerships and acquisitions for sustained growth in the competitive market.
*Note:
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it
