Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market Analysis

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.

Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market Report Coverage

Report Coverage	Details
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:	North America: U.S. and Canada Latin America: Brazil, Argentina, Mexico, and Rest of Latin America Europe: Germany, U.K., Spain, France, Italy, Russia, and Rest of Europe Asia Pacific: China, India, Japan, Australia, South Korea, ASEAN, and Rest of Asia Pacific Middle East: GCC, Israel, and Rest of Middle East Africa: South Africa, North Africa, and Central Africa
Segments covered:	By Drug: Ursodeoxycholic acid, Cholestyramine, Rifampicin, Late Stage Pipeline Drugs, Others. By Distribution Channel: Hospital Pharmacies, Retail Pharmacies, Online Pharmacies.
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
Growth Drivers:	Increasing approvals from regulatory authorities Increasing R&D activities for progressive familial intrahepatic cholestasis type 2 treatment
Restraints & Challenges:	Lack of evidences associated with the treatment coronavirus disease (COVID 19)

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.