PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS TYPE 2 TREATMENT MARKET ANALYSIS

The global progressive familial intrahepatic cholestasis type 2 treatment market is estimated to be valued at US$ 78.6 Million in 2023 and is expected to exhibit a CAGR of  40.56% during the forecast period (2023-2030).

Analysts’ Views on Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market:

Currently, many key players in the market are looking for a novel treatment for progressive familial intrahepatic cholestasis type 2. This rare genetic disorder affects the liver's ability to transport bile acids, leading to progressive liver damage. Researchers are exploring innovative approaches, such as gene therapy and targeted drug development, to address the underlying cause of the disease and provide effective treatment options for patients. These approaches aim to restore or enhance the liver's ability to transport bile acids, thereby slowing down or halting the progression of liver damage. Additionally, clinical trials are being conducted to evaluate the safety and efficacy of these potential treatments, offering hope for patients and their families.

Figure 1. Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market Share (%), By Drug, 2023

Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market– Drivers

• New generation treatment approach for progressive familial intrahepatic cholestasis type 2: Progressive familial intrahepatic cholestasis type 2 is one of the diseases in which the liver cannot properly release bile into the digestive system. And, currently, the treatment for progressive familial intrahepatic cholestasis type 2 is not very effective, due to which researchers and market key players are looking for new generation of solutions that can solve many unmet needs. They are focused on developing innovative therapies and exploring potential breakthroughs. The potential breakthroughs include gene therapy, which aims to correct the genetic mutations responsible for the disease. Additionally, researchers are investigating the use of small-molecule drugs that can target specific pathways involved in bile release, offering hope for more effective treatments in the future.

Figure 2. Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market Share (%), By Region, 2023

Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market- Regional Analysis

Among regions, North America is estimated to hold a dominant position in the global progressive familial intrahepatic cholestasis type 2 treatment market over the forecast period. North America is estimated to hold 41.3% of the market share in 2023. The global progressive familial intrahepatic cholestasis type 2 treatment market is expected to witness significant growth in the near future, driven by the increasing prevalence of progressive familial intrahepatic cholestasis type 2.

Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market– Impact of Coronavirus (COVID-19) Pandemic

Since the COVID-19 virus outbreak in December 2019, the disease has spread to over 100 countries across the globe, and the World Health Organization (WHO) declared it a public health emergency on January 30, 2020.

COVID-19 affected the economy in three main ways: by directly affecting the production and demand of drugs, by creating disruptions in distribution channels, and through its financial impact on firms and financial markets. Due to nationwide lockdowns, several countries, such as China, India, Saudi Arabia, the U.A.E., Egypt, and others, faced problems with the transportation of things from one place to another.

Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market Segmentation:

The global progressive familial intrahepatic cholestasis type 2 treatment market is segmented into  drug, distribution channel, and region.

• By drug, the market is segmented into ursodeoxycholic acid, cholestyramine, rifampicin, late stage pipeline drugs, and others. Out of which, ursodeoxycholic acid is expected to hold a dominant position in the global progressive familial intrahepatic cholestasis type 2 treatment market during the forecast period, and this is attributed to the increase of ursodeoxycholic acid for the making of drugs.
• By distribution channel, the market is segmented into hospital pharmacies, retail pharmacies, and online pharmacies. Out of which, hospital pharmacies is expected to dominate the market over the forecast period, and this is due to an increase in the number of patients, and hospitals only purchase their medications from hospital pharmacies to prevent any treatment blunders.
• Among all the segmentation, the drug segment is expected to dominate the market over the forecast period, and this is attributed to increasing use of drug for the effective treatment or disease management.

Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market- Cross Sectional Analysis:

Key players are focusing on presenting clinical trials for the progressive familial intrahepatic cholestasis type 2, which could be expected to boost demand for progressive familial intrahepatic cholestasis type 2 treatment market in the North America region. For instance, on May 17, 2023, Mirum Pharmaceuticals, Inc., a biopharmaceutical company, announced that the company will present data at the upcoming 55th Annual European Society for Pediatric, Gastroenterology, Hepatology, and Nutrition (ESPGHAN) Annual Meeting, taking place in Vienna, Austria. The company will present data from its LIVMARLI (maralixibat) oral solution clinical studies in Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC).

Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market: Key Developments

On June 12, 2023, Ipsen, a global, mid-sized biopharmaceutical company, announced that the company will present data from across its growing rare liver disease portfolio at the European Association for the Trial of Liver (EASL) Congress 2023 in Vienna, Austria. These include seven abstracts on new clinical data being presented on Bylvay (odevixibat) when used in patients with progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS). The Bylvay abstracts give more insight into the efficacy and safety profile of the therapy when applied to subgroups of both paediatric and adult PFIC patients. The data report on outcomes includes event-free survival, reduction in serum bile acids (sBAs), pruritus, and other quality of life outcomes in long-term clinical trials and real-world settings.

Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market: Key Trends

• Positive clinical trials result in progressive familial intrahepatic cholestasis type 2: The positive clinical trial results of progressive familial intrahepatic cholestasis type 2 are one major factor in market growth over the forecast period. In addition to the positive clinical trial results, the increasing prevalence of progressive familial intrahepatic cholestasis type 2 worldwide is expected to contribute significantly to market growth. Furthermore, the rising adoption of advanced diagnostic techniques and therapeutic options for managing this condition is anticipated to drive market expansion even further. Moreover, the growing awareness among healthcare professionals and patients about the importance of early diagnosis and treatment of progressive familial intrahepatic cholestasis type 2 is likely to fuel market demand. Additionally, government initiatives aimed at improving healthcare infrastructure and providing better access to healthcare services are expected to support market growth for this condition.

Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market: Restraint

• Less research and development towards progressive familial intrahepatic cholestasis type 2: Less research and development towards progressive familial intrahepatic cholestasis type 2 is one of the market-restraining factors. This causes less awareness and knowledge about the potential of progressive familial intrahepatic cholestasis type 2 in the market. As a result, there may be limited investment in developing effective treatments and therapies for this condition. Additionally, the lack of awareness and knowledge may also lead to delayed diagnosis and inadequate support for patients living with progressive familial intrahepatic cholestasis type 2. This can have significant implications for patient outcomes and quality of life. Additionally, the lack of funding for research and development may impede efforts to better understand the underlying causes of progressive familial intrahepatic cholestasis type 2, which would slow down efforts to find a treatment or more effective management methods for this condition.
• Counterbalance: The market is anticipated to grow over the forecast period as a result of key players concentrating on developing advanced products that are safe and affordable.

Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market - Key Players

Major players operating in the global progressive familial intrahepatic cholestasis type 2 treatment market include Teva Pharmaceutical Industries Ltd., AbbVie, Inc., Glenmark Pharmaceuticals Limited, Par Pharmaceuticals, Inc., Mylan Pharmaceuticals, Inc., Sanofi S.A., Mylan N.V., Novartis International AG, Akorn, Inc., Albireo Pharma, Inc., Mirum Pharmaceuticals, Jadeite Medicines Inc., and Ipsen Pharma.

Definition: Progressive familial intrahepatic cholestasis (PFIC) is a class of chronic cholestasis disorders that comprises a variety of genetic diseases. These conditions begin in infancy and usually progress to cirrhosis within the first decade of life. The average age at onset is 3 months, although some patients do not develop jaundice until later, even as late as adolescence. PFIC can progress rapidly and cause cirrhosis during infancy or may progress relatively slowly with minimal scarring well into adolescence. Few patients have survived into the third decade of life without treatment.