by The urea cycle disorders (UCDs) treatment market involves screening newborns for UCDs using tandem mass spectrometry, managing acute hyperammonemic crises, and minimizing long-term complications using drug therapies and dietary protein restriction. UCDs are inherited disorders caused by deficiencies of any of the enzymes and transporters involved in the urea cycle. Some of the common UCDs include ornithine transcarbamylase (OTC) deficiency, citrullinemia, and argininosuccinic aciduria. Current treatment options mainly involve a low-protein diet with essential amino acid supplements, nitrogen scavenger drugs such as sodium benzoate and sodium phenylbutyrate, and emergency management of acute episodes with dialysis and liver transplantation.
The global urea cycle disorders treatment market is estimated to be valued at US$ 510 Mn in 2023 and is expected to exhibit a CAGR of 6.0% over the forecast period 2023 to 2030, as highlighted in a new report published by Coherent Market Insights.
Market Dynamics:
The U.S. urea cycle disorders treatment market is driven by the growing adoption of gene therapy. Gene therapy is a promising approach for treating UCDs by delivering functional copies of defective genes into liver cells to restore enzymatic activities. Ongoing clinical trials are assessing the safety and efficacy of adeno-associated virus (AAV) vector-mediated gene therapy. For instance, in February 2022, Homology Medicines reported positive interim data from an ongoing Phase 1/2 pheNIX clinical trial of HMI-102, an investigational gene therapy for the treatment of adults with OTC deficiency. The trial demonstrated sustained plasma ornithine levels and normalized ammonia handling. Another driver is supportive regulatory framework. The U.S. FDA has granted orphan drug designation, rare pediatric disease, and fast track designation to several gene therapies in development for UCDs, which provides incentives to support their research and development. However, high costs associated with gene therapy and lack of long-term efficacy data are some of the challenges in the market.
Segment Analysis
The U.S. Urea Cycle Disorders Treatment market is dominated by the drugs segment which accounts for nearly 60% of the total market share. This is because drugs such as sodium phenylbutyrate, sodium benzoate, arginine, and glycerol phenylbutyrate are the preferred line of treatment for urea cycle disorders. These drugs help to bypass or supplement the missing enzymatic step in individual urea cycle disorders.
PEST Analysis
Political: The rising government support for rare disease treatment in the US is driving the market growth. The recent rare disease legislation and tax credits for clinical trials are positively impacting the market.
Economic: The high treatment cost of urea cycle disorders poses economic burden on patients. However, availability of various reimbursement programs are supporting the market growth.
Social: Growing awareness about rare diseases and availability of supportive communities are encouraging patients to seek diagnosis and treatment.
Technological: Advancements in newborn screening, diagnostic tools, and development of novel therapeutics are expected to provide opportunities in the coming years.
Key Takeaways
Global U.S. Urea Cycle Disorders Treatment Market Growth is expected to witness high growth. The global U.S. Urea Cycle Disorders Treatment Market is estimated to be valued at US$ 510 Mn in 2023 and is expected to exhibit a CAGR of 6.0% over the forecast period 2023 to 2030.
The Northeast region dominates the market owing to concentration of major healthcare systems and availability of specialized treatment facilities in this region.
Key players
Key players operating in the U.S. Urea Cycle Disorders Treatment market are BB, Ascenz Solutions Pte Ltd., and Aquametro Oil & Marine AG. BB dominates the market with its wide product portfolio to treat different types of urea cycle disorders.
*Note:
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it
Money Singh is a seasoned content writer with over four years of experience in the market research sector. Her expertise spans various industries, including food and beverages, biotechnology, chemical and materials, defense and aerospace, consumer goods, etc.
