PUBLISHER: Coherent Market Insights | PRODUCT CODE: 1378555
PUBLISHER: Coherent Market Insights | PRODUCT CODE: 1378555
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).
Report Coverage | Report Details | ||
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Base Year: | 2022 | Market Size in 2023: | US$ 78.6 Mn |
Historical Data for: | 2018 to 2021 | Forecast Period: | 2023 - 2030 |
Forecast Period 2023 to 2030 CAGR: | 39.80% | 2030 Value Projection: | US$ 818.8 Mn |
Progressive familial intrahepatic cholestasis refers to a collection of rare genetic disorders due to defective mechanisms of bile secretion. Typically divided into three subtypes, PFIC type 1, PFIC type 2, and PFIC type 3. The condition is usually diagnosed in the early years of life and often presents with signs and symptoms of intrahepatic cholestasis, such as pruritis, dark urine, pale stool, loss of appetite, and fatigue. Mutations in the ABCB11 gene are responsible for PFIC2. The ABCB11 gene provides instructions for making a protein called the bile salt export pump (BSEP). The protein is found in the liver, and its main role is to move bile salts (a component of bile) out of liver cells. Mutations in the ABCB11 gene result in the buildup of bile salts in liver cells, damaging these cells and causing liver disease.
The key players in the pharmaceutical industry are focusing on the development of new drugs and accessing new mechanisms of action to treat progressive familial intrahepatic cholestasis type 2. Thus, the introduction of such new products into the market is expected to drive growth of the global progressive familial intrahepatic cholestasis type 2 treatment market over the forecast period. For instance, on February 14, 2023, Mirum Pharmaceuticals, Inc., a biopharmaceutical company, announced that the company has submitted a supplemental New Drug Application (sNDA) for LIVMARLI (maralixibat) oral solution for the treatment of cholestatic pruritus in patients two months of age and older with progressive familial intrahepatic cholestasis (PFIC). The sNDA submission is based on data from the March PFIC sPhase 3 study of LIVMARLI. MARCH PFIC is the largest randomized trial conducted in PFIC, with 93 patients across a range of genetic PFIC subtypes, including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6 and unidentified mutational status.
Global Progressive Familial Intrahepatic Cholestasis Type 2 Treatment Market Detailed Segmentation: