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.
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- 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 2024, 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 2024. 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.
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- 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 2024, 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.
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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 2024, 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, 2024, 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.
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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
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