Indication name: Progressive
familial intrahepatic cholestasis (PFIC)
Progressive familial intrahepatic
cholestasis (PFIC) – Market outlook, Epidemiology, Market Forecast and
Competitive Landscape Report – 2020 To 2030 (Markets covered: US, Germany,
France, Spain, United Kingdom, Italy, Japan & China)
Progressive familial intrahepatic
cholestasis (PFIC) is a rare inherited condition. In PFIC children are
not able to drain bile from the liver even though the large bile ducts are open
(cholestasis). Mutations in the ATP8B1, ABCB11, and ABCB4 genes cause PFIC1,
PFIC2 and PFIC3 respectively. Some people with PFIC do not have a mutation in
the ATP8B1, ABCB11, or ABCB4 gene. In these cases, the cause of the condition
is unknown.
PFIC can be classified as
PFIC1- may have short stature, deafness,
diarrhea, inflammation of the pancreas (pancreatitis), and low levels of
fat-soluble vitamins (vitamins A, D, E, and K) in the blood. Affected
individuals typically develop liver failure before adulthood. PFIC2- develop
liver failure within the first few years of life. Additionally, affected individuals
are at increased risk of developing a type of liver cancer called
hepatocellular carcinoma. PFIC3- Signs and symptoms usually do not appear until later in infancy
or early childhood; rarely, people are diagnosed in early adulthood
Thelansis estimated PFIC affect 1
in 50,000 to 100,000 people worldwide. PFIC type 1 is much more common in the
Inuit population of Greenland and the Old Order Amish population of the United
States.
Competitive landscape of
Progressive familial intrahepatic cholestasis (PFIC) includes country specific
approved as well as pipeline therapies. Any asset/ product specific designation
or review and Accelerated Approval are being tracked and supplemented with
analyst commentary.
KOLs insights of Progressive
familial intrahepatic cholestasis (PFIC) across 8 MM market from center of
Excellence/ Public/ Private hospitals participated in the study. Insights
around current treatment landscape, epidemiology, clinical characteristics,
future treatment paradigm and Unmet needs.
Progressive familial intrahepatic
cholestasis (PFIC) Market Forecast: Patient Based Forecast Model (MS. Excel
Based Automated Dashboard) which Data Inputs with sourcing, Market Event and
Product Event, Country specific Forecast Model, Market uptake and patient share
uptake, Attribute Analysis, Analog Analysis, Disease burden and pricing
scenario, Summary and Insights.
S. No Asset Company Stage
1 Maralixibat Mirum Pharmaceuticals, Inc. Phase 3
2 A4250 (odevixibat) Albireo Pharma, Inc. Phase 3
Tags: Progressive familial
intrahepatic cholestasis (PFIC), Progressive familial intrahepatic cholestasis (PFIC) market outlook report, Progressive familial intrahepatic cholestasis
(PFIC) competitive
landscape, Progressive familial
intrahepatic cholestasis (PFIC) market forecast, Thelansis, Primary market research, KOL insights
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