SODIUM PHENYLBUTYRATE MARKET ANALYSIS

The global sodium phenylbutyrate market size is projected to reach US$ 3.02 billion by 2030, from US$ 1.92 billion in 2023, at a CAGR of 6.7% during the forecast period. Sodium phenylbutyrate is a nitrogen-binding drug used to treat urea cycle disorders (UCDs) and maple syrup urine disease (MSUD). UCDs are a group of inherited disorders that affect the body's ability to break down ammonia. MSUD is a rare genetic disorder that causes the body to produce high levels of ammonia.

The growth of the sodium phenylbutyrate market is driven by the increasing incidence of UCDs and MSUD. According to the Centers for Disease Control and Prevention (CDC), the estimated incidence of UCDs is 1 in 8,500 births. Additionally, the rising awareness about the benefits of sodium phenylbutyrate for the treatment of UCDs and MSUD is also driving the growth of the market.

Sodium Phenylbutyrate Market Regional Insights

• North Americais the leading market for sodium phenylbutyrate, with a share of 35% in 2022. The high prevalence of UCDs and MSUD in North America and the presence of a large number of key players in the region are the major factors driving the growth of the market in North America.
• Europeis the second-largest market for sodium phenylbutyrate, with a share of 25% in 2022. The rising awareness about the benefits of sodium phenylbutyrate for the treatment of UCDs and MSUD and the increasing number of product approvals in Europe are the major factors driving the growth of the market in the region.
• Asia Pacific is expected to grow at the highest CAGR during the forecast period, with a share of 20% in 2022. The growth of the market in Asia Pacific is driven by the rising awareness about UCDs and MSUD, the increasing number of product approvals, and the presence of a large number of key players in the region.

Figure 1. Global Sodium Phenylbutyrate Market Share (%), by Region, 2023

Sodium Phenylbutyrate Market Drivers

• Increasing prevalence of urea cycle disorders (UCDs): Sodium phenylbutyrate is commonly used in the treatment of urea cycle disorders, a group of rare genetic disorders that affect the body's ability to remove waste nitrogen. The rising prevalence of UCDs worldwide is driving the demand for sodium phenylbutyrate as a therapeutic option.
• Growing awareness and diagnosis of rare diseases: There has been an increasing focus on the diagnosis and treatment of rare diseases, including urea cycle disorders. Improved awareness and diagnostic capabilities have led to an increase in the number of patients being diagnosed with UCDs, thereby driving the demand for sodium phenylbutyrate.
• Expansion of orphan drug development: Sodium phenylbutyrate is classified as an orphan drug, meaning it is used to treat rare diseases. Governments and regulatory agencies worldwide have implemented supportive policies and incentives to encourage the development and commercialization of orphan drugs. This has facilitated the growth of the sodium phenylbutyrate market.
• Advancements in pharmaceutical research and development: Continuous advancements in pharmaceutical research and development have led to the development of more effective and innovative drugs. This includes the development of sodium phenylbutyrate formulations with improved efficacy, safety, and patient compliance, which contribute to the market growth.

Sodium Phenylbutyrate Market Opportunities

• Expansion into emerging markets: There are significant opportunities for sodium phenylbutyrate manufacturers to expand their presence in emerging markets. Developing regions, such as Asia-Pacific and Latin America, are experiencing rapid urbanization, improved healthcare infrastructure, and increasing awareness of rare diseases. As a result, there is a growing demand for effective treatments like sodium phenylbutyrate in these regions.
• Development of combination therapies: Sodium phenylbutyrate has shown potential as an adjuvant therapy in combination with other medications for the treatment of certain diseases. Research is underway to explore the synergistic effects of sodium phenylbutyrate with other drugs, such as histone deacetylase inhibitors, in various indications, including cancer. The development of combination therapies can open new avenues for sodium phenylbutyrate applications and expand its market potential.
• Expansion of indications: Although sodium phenylbutyrate is primarily used in the treatment of urea cycle disorders, there is ongoing research exploring its efficacy in other diseases. Preclinical and clinical studies are investigating the use of sodium phenylbutyrate in conditions like neurodegenerative disorders, cancer, and metabolic diseases. Successful outcomes in these studies can lead to the expansion of indications for sodium phenylbutyrate, broadening its market opportunities.
• Increasing focus on precision medicine: Precision medicine aims to deliver personalized treatments based on individual characteristics, including genetic makeup and disease profiles. Sodium phenylbutyrate aligns with the principles of precision medicine, as it is used to treat specific genetic disorders. As the field of precision medicine advances, there will be growing opportunities for sodium phenylbutyrate to be integrated into personalized treatment approaches.

Sodium Phenylbutyrate Market Trends

• Increasing research and development activities: There is a significant focus on research and development activities related to sodium phenylbutyrate. This includes efforts to develop new formulations, optimize dosing regimens, explore combination therapies, and investigate additional indications for sodium phenylbutyrate. The aim is to enhance the therapeutic outcomes, improve patient compliance, and expand the market potential of sodium phenylbutyrate.
• Rise in strategic collaborations and partnerships: Collaborations and partnerships between pharmaceutical companies, research institutions, and healthcare organizations are becoming increasingly common. These collaborations help in sharing expertise, resources, and data to accelerate drug development and commercialization. In the sodium phenylbutyrate market, such collaborations facilitate clinical trials, expand market reach, and enhance product offerings.
• Growing demand for orphan drugs: Orphan drugs, including sodium phenylbutyrate, are medications used to treat rare diseases. There is a rising demand for orphan drugs globally, driven by increasing awareness, improved diagnosis rates, and supportive regulations and incentives. The sodium phenylbutyrate market is benefiting from this trend, as it is a key treatment option for urea cycle disorders, which are classified as rare diseases.
• Emphasis on patient-centric approaches: There is a growing focus on patient-centric approaches in healthcare, and this trend is also influencing the sodium phenylbutyrate market. Patient advocacy groups, regulatory agencies, and healthcare providers are prioritizing patient preferences, needs, and experiences. This includes personalized medicine approaches, patient support programs, and initiatives to improve medication access and affordability for sodium phenylbutyrate.

Sodium Phenylbutyrate Market Restraints

• Limited awareness and underdiagnosis: Despite efforts to increase awareness of rare diseases and their treatments, including urea cycle disorders, there remains a lack of awareness among healthcare professionals and the general public. This leads to underdiagnosis and delays in initiating appropriate treatment with sodium phenylbutyrate. Limited awareness and underdiagnosis act as a restraint on the market's growth potential.
• High treatment costs: The cost of sodium phenylbutyrate treatment can be high, especially for individuals without adequate insurance coverage or in regions with limited healthcare resources. The high treatment costs may restrict access to sodium phenylbutyrate for patients, particularly in developing countries or for those without sufficient financial means, thus posing a restraint on market growth.
• Stringent regulatory requirements: The regulatory approval process for sodium phenylbutyrate and other orphan drugs can be complex and time-consuming. Meeting the stringent regulatory requirements, including demonstrating safety and efficacy through rigorous clinical trials, can pose challenges and delay market entry. These regulatory barriers act as restraints on the timely availability and commercialization of sodium phenylbutyrate.
• Side effects and safety concerns: While sodium phenylbutyrate is generally well-tolerated, it can still cause side effects, including gastrointestinal disturbances, neurotoxicity, and metabolic abnormalities. Safety concerns associated with sodium phenylbutyrate may limit its use in certain patient populations or require close monitoring, thereby affecting market acceptance and uptake.

Recent Developments

New product launches

• There have been a few new product launches for sodium phenylbutyrate in recent years. In 2018, the FDA approved Olpruva™ (sodium phenylbutyrate) as an oral suspension for the treatment of patients with urea cycle disorders (UCDs). Olpruva™ is a first-in-class, more palatable formulation of sodium phenylbutyrate that is better tolerated by patients.
• In 2020, Hikma Pharmaceuticals launched a generic version of sodium phenylbutyrate tablets in the United States. This generic version is available in 500 mg and 1000 mg strengths.
• In 2021, Acella Pharmaceuticals launched a generic version of sodium phenylbutyrate powder for oral suspension in the United States. This generic version is available in a 0.94 gram/gram strength.

Acquisition and partnerships

• In 2021, Hikma Pharmaceuticals acquired the rights to market sodium phenylbutyrate in the United States from Salix Pharmaceuticals.
• In 2022, Alvogen acquired the rights to market sodium phenylbutyrate in Europe from Orphan Europe.
• In 2022, CSL Behring entered into a partnership with Oryzon Genomics to develop and commercialize sodium phenylbutyrate for the treatment of urea cycle disorders.

Figure 2. Global Sodium Phenylbutyrate Market Share (%), by application type, 2023

Top companies in Sodium Phenylbutyrate Market

• Horizon Therapeutics plc
• Ammonett Pharma LLC
• Medison Pharma Ltd.
• Modern Dietetics Inc.
• Synageva BioPharma Corp.
• Medison Pharma Ltd.
• Swedish Orphan Biovitrum AB (Sobi)
• Entera Health, Inc.
• Dipharma Francis S.r.l.
• Ucyclyd Pharma, Inc.

*Definition: The Sodium Phenylbutyrate market refers to the global market for the pharmaceutical product Sodium Phenylbutyrate. Sodium Phenylbutyrate is a medication used for the treatment of certain genetic disorders, such as urea cycle disorders and sickle cell disease. It works by reducing the levels of toxic substances in the body and has been approved for use in various countries. The market encompasses the manufacturing, distribution, and sales of Sodium Phenylbutyrate and is driven by factors such as disease prevalence, patient awareness, government initiatives, and advancements in drug delivery systems.