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Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market: Introduction

  • Progressive familial intrahepatic cholestasis (PFIC) type 2 is a heterogeneous group of autosomal recessive disorder that occurs during childhood and disturbs the bile formation. This disorders is present with cholestasis at the hepatocellular origin. Major clinical manifestations involve pruritus, cholestasis, and jaundice. Patients suffering from progressive familial intrahepatic cholestasis (PFIC) type 2 usually develop fibrosis along with end-stage liver disease before adulthood.
  • The Progressive Familial Intrahepatic Cholestasis Type 2 treatment market can be segmented based on drug type, distribution channels, end-user, and region. Based on drug type, the market can be categorized into cholestyramine, ursodeoxycholic acid, rifampicin & others. Based on end-user, the market can be split into hospitals, specialty clinics, ambulatory surgical centers, and others. In terms of distribution channel, the Progressive Familial Intrahepatic Cholestasis Type 2 treatment market can be classified into hospital pharmacies, retail pharmacies, and ecommerce.

Opportunities of Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market

  • Rise in research and development of novel drugs for progressive familial intrahepatic cholestasis (PFIC) type 2 treatment is projected to boost the market during the forecast period. For instance, in August 2020, Mirum Pharmaceuticals, Inc., a biopharmaceutical company focused on the development of novel therapies for debilitating liver diseases, presented an overview from its phase 2 indigo study at the International Liver Congress 2020. The five-year analysis represented that patients with Progressive familial intrahepatic cholestasis (PFIC) type 2, also called as bile salt export pump (BSEP) deficiency, who achieved long-term maralixibat treatment have a significant enhancement in the transplant-free survival.
  • Increase in drug approvals from regulatory bodies is anticipated to fuel the global progressive familial intrahepatic cholestasis type 2 treatment market during the forecast period. For instance, in November 2020, Mirum Pharmaceuticals, Inc., 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), received approval for review from the European Medicines Agency (EMA).
  • Progressive familial intrahepatic cholestasis (PFIC) type 2 is a rare genetic disorder that has no basic treatment and requires R & D for novel treatment drugs. There is no curative treatment for Progressive familial intrahepatic cholestasis (PFIC) type 2. Hence, lack of novel therapies is anticipated to hamper the progressive familial intrahepatic cholestasis (PFIC) type 2 treatment market.

North America to Capture Major Share of Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market

  • North America region is anticipated to hold a notable share of the global progressive familial intrahepatic cholestasis type 2 treatment market during the forecast period, due to an increase in clinical trials intervention for progressive familial intrahepatic cholestasis type 2 treatment. For instance, in September 2020, Albireo Pharma, Inc. announced positive topline results from PEDFIC 1, a global Phase 3 clinical trial evaluating the safety of odevixibat conducted for PFIC1 and PFIC2. Odevixibat reduce bile acids in both PFIC1 and PFIC2 patients and demonstrated a clinically proven better results for pruritus.
  • However, the progressive familial intrahepatic cholestasis type 2 treatment market in Europe is anticipated to expand at a rapid pace during the forecast period. Additionally, key market players are focused on the development of novel drugs to broaden their product portfolio, which is anticipated to fuel the progressive familial intrahepatic cholestasis type 2 treatment market in the region during the forecast period. For instance, in December 08, 2020, Albireo Pharma, Inc. submitted Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) to obtain derive approval for odevixibat, useful for the treatment of patients suffering with progressive familial intrahepatic cholestasis (PFIC) type 2.

Key Players Operating in Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market

The global progressive familial intrahepatic cholestasis type 2 treatment market is highly fragmented, with the presence of various key players. A large number of manufacturers hold a major share in their respective regions. Major players operating in the global progressive familial intrahepatic cholestasis type 2 treatment market are:

  • 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.
  • Mirum Pharmaceuticals

Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market: Research Scope

Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market, by Drug Type

  • Cholestyramine
  • Ursodeoxycholic Acid
  • Rifampicin
  • Others

Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market, by Distribution Channel

  • Retail Pharmacies
  • Hospital Pharmacies
  • E-Commerce

Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market, by End Users

  • Hospitals
  • Specialty clinics
  • Ambulatory surgical centers
  • Others

Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market, by Region

  • North America
    • U.S.
    • Canada
  • Europe
    • Germany
    • France
    • U.K.
    • Italy
    • Spain
    • Rest of Europe
  • Asia Pacific
    • China
    • Japan
    • India
    • Australia & New Zealand
    • Rest of Asia Pacific
  • Latin America
    • Brazil
    • Mexico
    • Rest of Latin America
  • Middle East &Africa
    • GCC Countries
    • South Africa
    • Rest of Middle East & Africa

This study by TMR is all-encompassing framework of the dynamics of the market. It mainly comprises critical assessment of consumers' or customers' journeys, current and emerging avenues, and strategic framework to enable CXOs take effective decisions.

Our key underpinning is the 4-Quadrant Framework EIRS that offers detailed visualization of four elements:

  • Customer Experience Maps
  • Insights and Tools based on data-driven research
  • Actionable Results to meet all the business priorities
  • Strategic Frameworks to boost the growth journey

The study strives to evaluate the current and future growth prospects, untapped avenues, factors shaping their revenue potential, and demand and consumption patterns in the global market by breaking it into region-wise assessment.

The following regional segments are covered comprehensively:

  • North America
  • Asia Pacific
  • Europe
  • Latin America
  • The Middle East and Africa

The EIRS quadrant framework in the report sums up our wide spectrum of data-driven research and advisory for CXOs to help them make better decisions for their businesses and stay as leaders.

Below is a snapshot of these quadrants.

1. Customer Experience Map

The study offers an in-depth assessment of various customers’ journeys pertinent to the market and its segments. It offers various customer impressions about the products and service use. The analysis takes a closer look at their pain points and fears across various customer touchpoints. The consultation and business intelligence solutions will help interested stakeholders, including CXOs, define customer experience maps tailored to their needs. This will help them aim at boosting customer engagement with their brands.

2. Insights and Tools

The various insights in the study are based on elaborate cycles of primary and secondary research the analysts engage with during the course of research. The analysts and expert advisors at TMR adopt industry-wide, quantitative customer insights tools and market projection methodologies to arrive at results, which makes them reliable. The study not just offers estimations and projections, but also an uncluttered evaluation of these figures on the market dynamics. These insights merge data-driven research framework with qualitative consultations for business owners, CXOs, policy makers, and investors. The insights will also help their customers overcome their fears.

3. Actionable Results

The findings presented in this study by TMR are an indispensable guide for meeting all business priorities, including mission-critical ones. The results when implemented have shown tangible benefits to business stakeholders and industry entities to boost their performance. The results are tailored to fit the individual strategic framework. The study also illustrates some of the recent case studies on solving various problems by companies they faced in their consolidation journey.

4. Strategic Frameworks

The study equips businesses and anyone interested in the market to frame broad strategic frameworks. This has become more important than ever, given the current uncertainty due to COVID-19. The study deliberates on consultations to overcome various such past disruptions and foresees new ones to boost the preparedness. The frameworks help businesses plan their strategic alignments for recovery from such disruptive trends. Further, analysts at TMR helps you break down the complex scenario and bring resiliency in uncertain times.

The report sheds light on various aspects and answers pertinent questions on the market. Some of the important ones are:

1. What can be the best investment choices for venturing into new product and service lines?

2. What value propositions should businesses aim at while making new research and development funding?

3. Which regulations will be most helpful for stakeholders to boost their supply chain network?

4. Which regions might see the demand maturing in certain segments in near future?

5. What are the some of the best cost optimization strategies with vendors that some well-entrenched players have gained success with?

6. Which are the key perspectives that the C-suite are leveraging to move businesses to new growth trajectory?

7. Which government regulations might challenge the status of key regional markets?

8. How will the emerging political and economic scenario affect opportunities in key growth areas?

9. What are some of the value-grab opportunities in various segments?

10. What will be the barrier to entry for new players in the market?

Note: Although care has been taken to maintain the highest levels of accuracy in TMR’s reports, recent market/vendor-specific changes may take time to reflect in the analysis.

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Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market

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