PRIMARY BILIARY CHOLANGITIS TREATMENT MARKET ANALYSIS

The global primary biliary cholangitis treatment market is anticipated to witness substantial growth during the forecast period (2023-2030). It is projected to expand from US$ 650 million in 2023 to reach US$ 1,174.1 million by 2030, exhibiting a compound annual growth rate (CAGR) of 8.8%. This growth can be attributed to various factors including the increasing prevalence of primary biliary cholangitis. Primary Biliary Cholangitis (PBC) is a chronic autoimmune disease that damages the small bile ducts (cholangiocytes) in the liver.

Many people with PBC have no symptoms in the early stages. As the disease progresses, signs of biliary disease begin to appear. The earliest and most common symptoms of people with PBC are fatigue and itchy skin. These symptoms affect different people to different degrees. They can occur later or earlier in the course of the disease, and they can be mild to severe at any stage.

Analysts’ Views on the Global Primary Biliary Cholangitis Treatment Market:

The global primary biliary cholangitis treatment market is expected to witness significant growth over the forecast period. The rising prevalence of primary biliary cholangitis worldwide is the major factor driving the growth of the market. Other key drivers include the growing aging population, increasing approvals of novel drug therapies, and rising healthcare spending in developing nations. North America currently dominates the market and is expected to continue its dominance in the coming years. This can be attributed to the well-developed healthcare infrastructure and increasing Primary Biliary Cholangitis (PBC) disease incidence in the region. Meanwhile, the Asia Pacific region is likely to emerge as the fastest growing market due to rising healthcare awareness, improving healthcare facilities, and large population base.

However, lack of disease awareness among people and healthcare providers, high cost of treatment therapies and long approval time for new drugs may negatively impact the market growth. Stringent regulatory policies for drug approval procedures in different countries can also hamper sales.

Nevertheless, ongoing R&D activities for developing advanced treatment options with better efficacy and minimal side effects provide lucrative opportunities for market players. In addition, increasing healthcare expenditure of governments worldwide on rare disease treatment creates a favorable market landscape. Partnerships between pharmaceutical companies and clinical research organizations are also contributing to the growth of the global primary biliary cholangitis treatment market.

Primary Biliary Cholangitis Treatment Market Regional Insights

• North America: The North America region, including the U.S. and Canada, has a well-established healthcare infrastructure and has a strong presence of leading key players, such as Intercept Pharmaceuticals, Inc., GSK plc., Bristol-Myers Squibb and Company, and Enanta Pharmaceuticals, focused on the research and development of primary biliary cholangitis treatment. The market for primary biliary cholangitis treatment in North America is expected to be significant, driven by the increasing research and development activities. For instance, in November 2022, Intercept Pharmaceuticals, Inc., a biopharmaceutical company, announced that results from two analyses assessing the potential of obeticholic acid (OCA) to improve outcomes for patients with primary biliary cholangitis (PBC).
• Europe: Europe is another region with a strong focus on the development of novel therapeutics for PBC. Countries, such as Germany, France, and the U.K., have robust healthcare pharmaceutical companies, which contribute to the demand for therapeutics for PBC. The market in Europe is expected to experience steady growth due to the increasing prevalence of primary biliary cholangitis (PBC).
• Asia Pacific: The Asia Pacific region presents significant growth opportunities for the primary biliary cholangitis treatment market. Rapidly developing countries, such as China, India, etc., are witnessing increased healthcare spending and increasing prevalence of liver diseases.

Figure 1. Global Primary Biliary Cholangitis Treatment Market Share (%), By Region, 2023

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Primary Biliary Cholangitis Treatment Market Drivers:

• Increasing Prevalence of Primary Biliary Cholangitis: The rising prevalence of primary biliary cholangitis across the globe is one of the major factors fueling the growth of the global primary biliary cholangitis treatment market. With changing lifestyles and increased life expectancy, and liver diseases are on the rise which require effective treatment options. Primary biliary cholangitis treatment have emerged as a revolutionary approach to develop more targeted and efficient drugs to treat such complex diseases. For instance, in June 2021, according to a report published by National Center for Biotechnology Information (NCBI), the pooled point-prevalence rate of primary biliary cholangitis was 22.27 cases per 100,000 inhabitants.
• Growing Adoption of Novel Drugs: Primary biliary cholangitis has traditionally been treated with ursodeoxycholic acid (UDCA) as the first-line therapy. However, with close to 40% of patients not responding adequately to UDCA, there was a considerable unmet need for alternative treatment options. This has prompted drug manufacturers to focus on developing novel precision therapies targeting specific disease pathways involved in PBC. In the last 5 years alone, three new drugs have been approved by regulatory agencies like the U.S. FDA and European Medicines Agency (EMA) - obeticholic acid (Ocaliva), fibric acid derivatives such as bezafibrate (Pemptor), and interleukin-12/23 inhibitors like mirikizumab (Kineret). Compared to conventional treatments, these novel drugs have demonstrated higher rates of biochemical response and incomplete biochemical response in clinical trials. For instance, in November 2020, GSK plc., a multinational pharmaceutical and biotechnology company, announced further progression in its R&D pipeline with the presentation of data for linerixibat, an investigational product for the potential treatment of cholestatic pruritus in patients with primary biliary cholangitis (PBC).

Primary Biliary Cholangitis Treatment Market Opportunities:

• Increasing adoption of Combination Therapy: Combination therapy holds significant potential to drive the growth in the global primary biliary cholangitis treatment market. As per medical experts, combination of two or more treatment options may help achieve better clinical response for patients suffering from primary biliary cholangitis (PBC). PBC is an autoimmune disease where there is slow destruction of smaller bile ducts inside the liver over a period of time. Current treatment options, such as ursodeoxycholic acid (UDCA) and obeticholic acid, provide relief to some extent but not all patients respond equally to single drug therapies. Medical reports from the World Health Organization (WHO) suggest that maximum 50-60% patients respond to UDCA, leaving remainder with inadequate response. This is where combination therapies can make a difference. Combining UDCA with other immunomodulatory drugs holds promise. Recent clinical studies show that combining UDCA with fibric acid derivatives, such as bezafibrate, led to improved biochemical, serological, and histological responses in UDCA partial responders or non-responders. Similarly, combining UDCA and monoclonal antibodies targeting specific inflammatory pathways demonstrated enhanced therapeutic benefits over monotherapies. Ongoing phase 3 clinical trials evaluating safety and efficacy of UDCA in combination with immunomodulators like Budesonide and combination of Obeticholic acid with other agents are encouraging. Positive outcomes from such combination therapy trials can go a long way in better management of PBC and improving patient outcomes. This will likely fuel the increased adoption of combination drug regimens among physicians and healthcare providers for PBC treatment. Growing clinical evidence on benefits of combination approach is expected to drive higher treatment uptake beyond existing patient pools, thereby accelerating future growth prospects of the global primary biliary cholangitis treatment market over the forecast period.

Primary Biliary Cholangitis Treatment Market Trends:

• Strategic Alliance and Collaboration between Companies: Strategic alliances and collaborations between companies have become increasingly common in the global primary biliary cholangitis treatment market in recent years. By joining forces, companies are able to pool together their complementary strengths like research expertise, clinical trial networks, manufacturing capabilities, and marketing reach to accelerate drug development and better serve patients. For instance, on January 8, 2023, CymaBay Therapeutics, Inc., a clinical-stage biopharmaceutical company, announced that it has entered into a collaboration and license agreement with Kaken Pharmaceutical Co., Ltd., a pharmaceutical company, for the development and commercialization in Japan of CymaBay’s investigational drug, seladelpar for the treatment of primary biliary cholangitis. Increasing strategic alliances like these indicate companies recognize that collaborating is essential to address the challenges of effectively researching, developing, and commercializing new treatment options for rare diseases like primary biliary cholangitis on a global scale. It ultimately benefits patients by accelerating the availability of novel therapies.

Primary Biliary Cholangitis Treatment Market Restraints:

• High Cost of Treatment: The high cost of treatment is one of the major factors restraining the growth of the global primary biliary cholangitis treatment market. Primary biliary cholangitis is a chronic and progressive liver disease that damages the smallest bile ducts in the liver over time. This causes bile buildup that can lead to scarring and damage to the liver. At present, there is no cure for primary biliary cholangitis and the treatment options mainly focus on managing the symptoms and slowing the progression of liver damage. The first line of treatment includes ursodeoxycholic acid tablets which need to be taken lifelong. While this drug helps improve liver tests and symptoms for many patients, its high cost poses a challenge. For instance, in February 2021, according to a study published by the National Center for Biotechnology Information (NCBI), the mean all-cause inpatient medical cost over a mean follow-up period of 22.3 months was US$ 3,905 per patient per month. An estimated 66% of this cost was due to PBC. This high out-of-pocket expenditure acts as a deterrent for proper treatment compliance. Some patients may omit doses or stop treatment due to affordability issues, which negatively impacts disease management in the long run.

Figure 2. Global Primary Biliary Cholangitis Treatment Market Share (%), By Treatment Type, 2023

Recent Developments

Primary Biliary Cholangitis Treatment Market

• On June 30 2023, Ipsen Pharma, a biopharmaceutical company, and GENFIT, a biopharmaceutical group, announced positive topline data from the pivotal ELATIVE Phase III trial. In the trial the efficacy and safety of elafibranor, an investigational dual α, δ PPAR agonist, is being assessed for the treatment of patients with the rare cholestatic liver disease, primary biliary cholangitis (PBC), who have an inadequate response or intolerance to the current standard of care therapy, ursodeoxycholic acid (UDCA).
• In January 2022, COUR Pharmaceuticals, a biotechnology company developing novel immune-modifying nanoparticles (CNPs) to treat immune disorders, announced that the U.S. Food and Drug Administration (FDA) granted Fast Track Designation to the company's investigational therapy, CNP-104 for the treatment of primary biliary cholangitis.

Agreements and collaborations

• In December 2021, Ipsen Pharma and GENFIT, a biopharmaceutical group, announced that the companies have entered into a long-term strategic partnership for global collaboration between the two companies. The agreement gives Ipsen Pharma exclusive worldwide license to develop, manufacture, and commercialize GENFIT’s investigational treatment elafibranor, for people living with Primary Biliary Cholangitis (PBC).
• In November 2021, Ironwood Pharmaceuticals, Inc., a healthcare company, announced that it is expanding its pipeline by entering into a collaboration and license option agreement with COUR Pharmaceuticals, a biotechnology company. This agreement gives Ironwood an option to acquire an exclusive license to develop and commercialize, in the U.S., COUR’s investigational therapy, CNP-104 (the “License”), which if successful, could transform the treatment of primary biliary cholangitis.

Top Companies in the Primary Biliary Cholangitis Treatment Market

• Intercept Pharmaceuticals, Inc.
• Highlight Therapeutics, S.L.
• GSK plc.
• Bristol-Myers Squibb and Company
• Enanta Pharmaceuticals
• NW Biotherapeutics
• Merck & Co Inc.
• Ipsen Pharma
• Johnson & Johnson
• GENFIT
• Ironwood Pharmaceuticals, Inc.
• Novartis AG
• COUR Pharmaceuticals
• Kaken Pharmaceutical Co., Ltd.

Definition:  Primary biliary cholangitis is a chronic disease in which the small bile ducts in the liver become inflamed and are eventually destroyed. When there are no bile ducts, bile builds up and causes liver damage. Over time, this damage can lead to liver scarring, cirrhosis, and eventually liver failure.