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Progressive familial intrahepatic cholestasis (PFIC) type 2 is a rare genetic disorder that has no curative medication. Key players in the market only provide symptomatic treatment drugs. Thus, the rising R&D activities for the development of new drugs for PFIC type 2 treatment is expected to spur growth of the progressive familial intrahepatic cholestasis (PFIC) type 2 treatment market over the forecast period.
The major treatment drugs used for PFIC type 2 are ursodeoxycholic acid, which is the most prescribed medication for treatment of pruritus associated with the PFIC type 2. Other medications such as cholestyramine, rifampin, and naltrexone are used in combination of therapies when it is required. Furthermore, liver transplant is still the best treatment option for patients with PFIC type 2 and is now being considered a first-line option in many centers, even in patients without evidence of end-stage liver disease. New treatment drugs for PFIC type 2 in pipeline are Odevixibat and Maralixibat.
Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market – Impact of Coronavirus (Covid-19) Pandemic
The patients of progressive familial intrahepatic cholestasis type 2 are expected to receive extra care during the covid-19 pandemic because the liver conditions can be highly comorbid during the covid-19 infection. Many hospitals resumed liver transplantation surgeries, but the need for immunosuppressive medications were highly associated with increased severity of covid-19 infection and death during coronavirus outbreaks. After liver surgery, all patients were managed in a clean ICU and post-transplant ward, and treated with standard triple immunosuppression regimen. The pediatric liver transplant program continued at reduced capacity throughout the pandemic, since SARS-CoV-2 was less prevalent in the pediatric population and there was adequate ICU capacity, and therefore a lower risk of nosocomial infection.
The progressive familial intrahepatic cholestasis type 2 treatment market is estimated to be valued at US$ 29.6 million in 2020 and is expected to exhibit a CAGR of 38.6% during the forecast period (2020-2027).
Figure 1. Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market Share (%) Analysis, By Drug, 2020 - 2027
Increasing R&D activities for progressive familial intrahepatic cholestasis type 2 and increasing approvals from regulatory authorities are expected to drive growth of this market during the forecast period
The increasing research and development of new drugs for PFIC type 2 treatment is expected to spur growth of the progressive familial intrahepatic cholestasis (PFIC) type 2 treatment market over the forecast period. For instance, in August 2020, Mirum Pharmaceuticals, Inc., a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, presented an analysis from its Phase 2 INDIGO study in an oral late-breaker session at the Digital International Liver Congress 2020. The five-year analysis showed that patients with PFIC2, also known as bile salt export pump (BSEP) deficiency, who achieved sBA control on long-term maralixibat treatment have a significant improvement in transplant-free survival.
Furthermore, increasing drug approvals from the regulatory bodies is expected to boost the global progressive familial intrahepatic cholestasis type 2 treatment market growth over the forecast period. For instance, in November 2020, Mirum Pharmaceuticals, Inc., a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, announced that the company’s Marketing Authorization Application (MAA) for its investigational medicine, Maralixibat, for the treatment of patients with progressive familial intrahepatic cholestasis type 2 (PFIC2), also known as bile salt export pump (BSEP) deficiency, was accepted for review (validated) by the European Medicines Agency (EMA). The validation of the application by the EMA confirms that all essential regulatory elements are included in the submission such that the EMA can begin its review.
| Report Coverage | Details | ||
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| Base Year: | 2019 | Market Size in 2020: | US$ 29.6 Mn |
| Historical Data for: | 2017 to 2019 | Forecast Period: | 2020 to 2027 |
| Forecast Period 2020 to 2027 CAGR: | 38.6% | 2027 Value Projection: | US$ 290.8 Mn |
| Geographies covered: |
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| Companies covered: |
Teva Pharmaceutical Industries Ltd., AbbVie, Inc., Glenmark Pharmaceuticals Limited, Par Pharmaceuticals, Inc., Mylan Pharmaceuticals, Inc., Sanofi S.A., Novartis International AG, Akorn, Inc., Albireo Pharma, Inc., and Mirum Pharmaceuticals |
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Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market – Restraints
Progressive familial intrahepatic cholestasis (PFIC) is a rare genetic disorder which has no cure and requires research and development for new treatment drugs. There are no novel or curative therapies for PFIC. Thus, lack of novel therapies is expected to restrain the progressive familial intrahepatic cholestasis (PFIC) type 2 treatment market growth. According to the National Center of Biotechnology Information (NCBI), there are three types of PFIC: PFIC1, PFIC2, and PFIC3. Both PFIC1 and PFIC2 are caused by impaired bile salt secretion due to defects in ATP8B1 encoding the FIC1 protein, and in ABCB11 encoding the bile salt export pump protein (BSEP). Defects in ABCB4, encoding the multi-drug resistant 3 protein (MDR3), impair biliary phospholipid secretion resulting in PFIC3.
Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market – Regional Analysis
North America region is expected to hold dominant position in the global progressive familial intrahepatic cholestasis type 2 treatment market over the forecast period, owing to the increasing clinical trials for PFIC type 2 treatment. For instance, September 2020, Albireo Pharma, Inc. announced positive topline results from PEDFIC 1, a global Phase 3 clinical trial evaluating the efficacy and safety of odevixibat and the largest study ever conducted in PFIC1 and PFIC2. Odevixibat reduced bile acids in both PFIC1 and PFIC2 patients and demonstrated a clinically better results for pruritus.
Moreover, Europe progressive familial intrahepatic cholestasis type 2 treatment market is expected to witness significant growth during the forecast period. Market players are focusing on developing new drugs to broaden their product portfolio, which is expected to boost growth of the market in this region during the forecast period. For instance, on December 08, 2020, Albireo Pharma, Inc. submitted Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) to obtain approval for odevixibat, indicated for the treatment of patients with progressive familial intrahepatic cholestasis (PFIC).
Figure 2. Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market Value (US$ Mn) & Y-o-Y Growth (%), 2016-2027
Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market - Competitive Landscape
Key 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., Novartis International AG, Akorn, Inc., Albireo Pharma, Inc., and Mirum Pharmaceuticals.
Progressive familial intrahepatic cholestasis type 2 (PFIC2) is a rare condition that affects the liver. People with this condition generally develop signs and symptoms during infancy, which may include severe itching, jaundice, portal hypertension (high blood pressure in the vein that provides blood to the liver) and hepatosplenomegaly (enlarged liver and spleen). PFIC2 generally progresses to liver failure in the first few years of life. Affected people also have an increased risk of developing hepatocellular carcinoma (a form of liver cancer). PFIC2 is caused by change (mutations) in the ABCB11 gene and is inherited in an autosomal recessive manner. Treatment options include ursodeoxycholic acid therapy to prevent liver damage, surgery, and/or liver transplantation. There are three known types of PFIC: PFIC1, PFIC2, and PFIC3.
Market Dynamics
Increasing awareness about progressive familial intrahepatic cholestasis type 2 disease is expected to provide more opportunities for players in the progressive familial intrahepatic cholestasis type 2 treatment market in the near future. For instance, on October 3, 2019, Albireo Pharma, Inc., a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, announced its support for PFIC Awareness Day and reaffirmed its commitment to patients and families living with progressive familial intrahepatic cholestasis (PFIC).
However, higher economic growth in low- and middle-income countries has not closed the gap in health spending, and global inequity in health spending has remained largely unchanged. For instance, according to a report on ‘New Perspectives on Global Health Spending for Universal Health Coverage’ by WHO published in 2015, high-income countries with only 16% of the world’s population, account for 80% of global health spending. Conversely, 76% of the world’s population live in middle-income countries, but these account for less than 20% of global health spending. Low-income countries, with more than half a billion people, accounted for less than 1% of the world’s health spending in 2015. Thus, low healthcare spending in low income countries limits such countries for research and development of new drugs for treatment of rare diseases.
Key features of the study:
Detailed Segmentation:
“*” marked represents similar segmentation in other categories in the respective section.
The progressive familial intrahepatic cholestasis type 2 treatment market is expected to exhibit a CAGR of 38.6% during the forecast period (2020-2027).
The market is estimated to be valued at US$ 29.6 million in 2020.
The prominent players operating in the market include Teva Pharmaceutical Industries Ltd., AbbVie, Inc., Glenmark Pharmaceuticals Limited, Par Pharmaceuticals, Inc., Mylan Pharmaceuticals, Inc., Sanofi S.A., Novartis International AG, Akorn, Inc., Albireo Pharma, Inc., and Mirum Pharmaceuticals.
The market is expected to be valued at US$ 290.8 million in 2027.
North America is the major region in the market.
The increasing approvals from regulatory authorities and increasing R&D activities for progressive familial intrahepatic cholestasis type 2 treatment are expected to drive growth of the market.
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