The U.S. Urea Cycle Disorders Treatment Market to grow at highest pace owing to rising awareness and diagnosis rate

The U.S. urea cycle disorders (UCD) treatment market involves therapeutics targeting a group of rare inborn errors of metabolism caused due to genetic defects in the enzymes and transporters of the urea cycle present in liver. The key symptoms of UCD include vomiting, lack of appetite, failure to thrive and neurological complications like seizures, delayed development. The treatments include sodium phenylbutyrate, glycerol phenylbutyrate and nitrogen-scavenging drugs that help in removal of toxic ammonia from the blood. The dietary protein restriction is also crucial for management of UCD. Rising awareness among physicians and patients about early screenings and diagnosis is leading to timely intervention, improving clinical outcomes. The Global 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.

Key Takeaways

Key players operating in the U.S. urea cycle disorders (UCD) treatment 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. Kawasumi Laboratories holds a major share owing to its diversified portfolio of sodium phenylbutyrate and glycerol phenylbutyrate formulations.

The growing awareness and diagnosis rates have augmented the demands for U.S. Urea Cycle Disorders Treatment Market Demand therapeutics. According to a survey, around 1 in 35 Americans is diagnosed with a urea cycle disorder by the age of five. This growing patient pool is propelling the market revenues.

Researchers are developing new formulations, drug delivery techniques and innovative testing techniques for early diagnosis of UCD. Trials are ongoing to evaluate the safety and efficacy of gene therapies and hepatocyte transplantation as potential curative options for UCD.

Market Trends

- Personalized medicine approach: Healthcare providers are adopting personalized treatment plans catering to individual patient's metabolite levels, tolerance, and responsiveness to drugs. Pharmacogenetic testing is guiding optimum drug selection and dosing.

- M&A activities: Large players are acquiring smaller biotech firms engaged in developing novel UCD therapeutics to expand their product portfolios and strengthen geographical presence. For instance, Fresenius Kabi acquired Therachon in 2020.

Market Opportunities

- Combination therapy: Combining available drugs judiciously to achieve better nitrogen scavenging is an area attracting research focus. This can help controlled disease management.

- Novel drug delivery: Researchers are evaluating sustained/controlled release formulations, pill-sized implants, and ingestible electronic devices to replace frequent injectables and improve patient adherence to treatment regimens.

Impact of COVID-19 on U.S. Urea Cycle Disorders Treatment Market

The COVID-19 pandemic has significantly impacted the growth of the U.S. urea cycle disorders treatment market. In the initial phase of the pandemic, the hospitals and clinics faced shortages of critical care beds and medical equipment. This resulted in cancelling or postponing non-emergency medical procedures including treatment of Urea Cycle Disorders (UCD). Furthermore, patients postponed their regular treatment and therapy visits to hospitals due to fear of contracting the infection. This negatively affected the demand for UCD treatment and associated medications during 2020-2021.

However, with gradual reduction in daily COVID-19 cases and increased vaccination rates, the healthcare systems resumed normal functioning from late 2021. The hospitals prioritized treatment of chronic and life-threatening conditions like UCD. The pharmaceutical companies also ramped up production of essential UCD drugs to meet the growing demand. Various government initiatives to promote online consultations and home delivery of medicines aided continuity of treatment during the pandemic. It is expected that with increased awareness about management of UCD and expansion of telehealth, the U.S. urea cycle disorders treatment market will witness steady growth in the forecast period.

In terms of geography, the Northeast region accounts for the largest share in the U.S. urea cycle disorders treatment market in terms of value. States like New York and Pennsylvania have reported high prevalence of UCD and have well-established healthcare infrastructure to effectively manage the condition. The presence of leading pharmaceutical manufacturers and research organizations in the Northeast further aids new drug development and timely access to advanced treatment options.

The Southwest region including states like California and Texas is poised to be the fastest growing region for U.S. urea cycle disorders treatment market during the forecast period. Favorable government policies promoting rare disease research, rising healthcare expenditures, and increasing patient awareness are driving the market growth in this region. In addition, the presence of top hospitals and availability of skilled physicians will help boost early diagnosis and management of UCD in the Southwest states.
Get More Insights On, U.S. Urea Cycle Disorders Treatment Market
About Author:
Ravina Pandya, Content Writer, has a strong foothold in the market research industry. She specializes in writing well-researched articles from different industries, including food and beverages, information and technology, healthcare, chemical and materials, etc. (https://www.linkedin.com/in/ravina-pandya-1a3984191)