July 20, 2024
Ornithine Transcarbamylase Deficiency Treatment Market

Emergence of Gene Therapy is anticipated to open up the new avenue for Ornithine Transcarbamylase Deficiency Treatment

The Ornithine Transcarbamylase Deficiency Treatment Market is estimated to be valued at US$ 810.4 Mn in 2023 and is expected to exhibit a CAGR of 4.2% over the forecast period 2023 to 2030, as highlighted in a new report published by Coherent Market Insights.

Market Overview:
Ornithine transcarbamylase deficiency (OTCD) is an inherited urea cycle disorder that prevents the body from eliminating excess levels of ammonia through the urine. Symptoms of OTCD range from mild to severe, depending on the extent of the enzyme deficiency. Current treatment options for OTCD include dietary protein restriction, carnitine supplementation, and prescription drugs such as Ravicti and Buphenyl for acute management of ammonia levels. Gene therapy is an emerging modality that aims to treat the root cause of urea cycle disorders.

Market Dynamics:
The major drivers fueling growth of the ornithine transcarbamylase deficiency treatment market include rising research & development activities focused on gene therapies and emerging drugs for long-term treatment of OTCD. According to CMI analysis, over 30 drugs and gene therapies are currently in clinical trials for treating urea cycle disorders like OTCD. Emerging drug candidates like DNL310 from Arcturus Therapeutics aim to cure OTCD through RNA editing technology. In addition, several gene therapy programs from Horizon Therapeutics, Axovant Gene Therapies and others hold promise for long-term management of OTCD without lifelong restrictions.

Furthermore, rising awareness initiatives by patient advocacy groups are encouraging affected families to actively seek diagnosis and treatment. Support networks play a crucial role in disseminating information on new therapeutic options in clinical trials. They strive to enhance understanding of OTCD signs among healthcare professionals to facilitate early diagnosis and timely intervention. These efforts are expected to improve screening and diagnosis rates over the forecast period.
Segment Analysis
The ornithine transcarbamylase deficiency treatment market is segmented based on the type of treatment, route of administration, and distribution channel. Among these, the medication segment dominates the market owing to the widespread availability and approval of medication for ornithine transcarbamylase deficiency treatment. Medication aims to lower ammonia levels in the blood and help avoid further long term complications. Some of the widely used medication includes citrulline, sodium phenylbutyrate, and glycerol phenylbutyrate which help to bypass or inhibit enzymes involved in ammonia production.

PEST Analysis
Political: Governments across various countries have offered support through funding and investments for rare disease research including improved treatment for OTC deficiency. This is likely to positively impact the market growth.
Economic: The rising healthcare expenditure and increasing focus on orphan drugs is supplementing the market expansion. Furthermore, new product approvals and launches coupled with increasing patient affordability and demand for effective treatment are fueling the market revenues.
Social: Growing patient support groups and associations spreading awareness about early diagnosis and management of OTC deficiency is driving the social aspect of market growth.
Technological: Advancements in screening and diagnostic tests help in early detection of urea cycle disorders. In addition, research focusing on gene therapy and other novel drug delivery options are expected to bring more efficacious treatment solutions.

Key Takeaways
The global Ornithine Transcarbamylase Deficiency Treatment Market Share is expected to witness high growth, exhibiting CAGR of 4.2% over the forecast period, due to increasing product pipelines, rise in R&D investments for orphan diseases, and surging demand for personalized treatment.

Regional analysis: North America dominates the ornithine transcarbamylase deficiency treatment market owing to the strong presence of major players and advanced healthcare infrastructure. Furthermore, Europe holds a significant market share due to the rising healthcare expenditure and government support in rare disease research. Asia Pacific exhibits the fastest growth rate over the forecast period with rising healthcare standards and increasing patient population.

Key players operating in the ornithine transcarbamylase deficiency treatment market are Horizon Therapeutics Plc, Bausch Health Companies Inc., Danone, Nestlé, Ultragenyx Pharmaceutical., Arcturus Therapeutics, Inc., Abbott., Swedish Orphan Biovitrum AB, Acer Therapeutics Inc., and Assertio Holdings, Inc., iECURE, and Translate Bio, Inc. The key players are majorly focusing on obtaining approvals for advanced treatment options and strengthening their product portfolio through partnerships and collaborations.
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it