According to the National Institute of Health (NIH), (PFIC) is a disorder that causes progressive liver disease, which typically leads to liver failure in which liver cells are unable to secrete a digestive fluid called bile. The buildup of bile in liver cells causes liver disease in affected individuals. The average age at onset is three months, although some patients do not develop jaundice until later, even as late as adolescence. PFIC can progress rapidly and cause cirrhosis during infancy or may progress relatively slowly with minimal scarring into adolescence. Few patients survive into the third decade of life without treatment.
DelveInsight's "Progressive Familial Intrahepatic Cholestasis (PFIC) Market Insights, Epidemiology, and Market Forecast 2030" reports deliver an in-depth understanding of the disease, historical and forecasted epidemiology, the pipeline insight as well as the Progressive Familial Intrahepatic Cholestasis (PFIC) market size and shares analysis in the seven major markets (7MM) (i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom) and Japan. The report also covers emerging drugs, current treatment practices, Progressive Familial Intrahepatic Cholestasis (PFIC) market shares of the individual therapies, a detailed current Progressive Familial Intrahepatic Cholestasis (PFIC) treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying potential of the market.
Progressive Familial Intrahepatic Cholestasis (PFIC) Market Key Facts
- As per the Progressive Familial Intrahepatic Cholestasis (PFIC) Organization, PFIC affects <1 in 100,000 people worldwide with recently discovered new genes, such as TJP2, NRIH4, and MYO5B.
- The United Kingdom-based study titled “Systematic review of progressive familial intrahepatic cholestasis” conducted by Baker et al. found that the incidence of intrahepatic cholestasis was 1 per 18,000 live births. The researchers also referred to a study in which infants and children (2–18 years) with cholestasis were 12–13% with genetically diagnosed PFIC.
- According to a French study by Davit-Spraul et al., titled “Progressive familial intrahepatic cholestasis”, highlighted that the estimated incidence varies between 1 per 50,000 and 1 per 100,000 births in PFIC patients. It was also found that PFIC1 and PFIC2 represented two out of three cases of total PFIC, whereas PFIC3 was one case per three cases.
- As per a study titled “Progressive familial intrahepatic cholestasis: diagnosis, management, and treatment” by Gunaydin et al. stated that PFICs were responsible for about 10–15% of children with cholestatic liver diseases with the total number of cases from the two subtype Prevalent populations (PFIC1 and PFIC2) reported were less than 200. PFIC-3 subtype incident population was even rarer with a reported case number of less than 20.
- A study conducted by Jacquemin titled, “Progressive familial intrahepatic cholestasis” stated that the main clinical manifestations of PFIC include cholestasis, pruritus, and jaundice. PFIC patients usually develop fibrosis and end-stage liver disease before adulthood.
Progressive Familial Intrahepatic Cholestasis (PFIC) Market Trends
Progressive Familial Intrahepatic Cholestasis (PFIC) market size is expected to increase in the coming years owing to the rise in the number of prevalent cases in the 7MM.
The Progressive Familial Intrahepatic Cholestasis (PFIC) market analysis section of the report helps to understand the current and forecasted Progressive Familial Intrahepatic Cholestasis (PFIC) market trends and growth by analyzing the impact of current therapies on the market, unmet needs, drivers, and barriers. The report gives complete detail of Progressive Familial Intrahepatic Cholestasis (PFIC) Market Size and Shares of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, and view of the key opinion leaders.
The pipeline of Progressive Familial Intrahepatic Cholestasis is not robust. However, owing to the positive outcomes of some of the key players the PFIC market is expected to show positive growth in the coming years.
Key Companies in the Progressive Familial Intrahepatic Cholestasis Market include Albireo, Mirum, Pharmaceuticals, Vivet Therapeutics, and others. Progressive Familial Intrahepatic Cholestasis Therapies covered in the report include Maralixibat, A4250 (Odevixibat), VTX-802, and VTX-803, and many more.
Progressive Familial Intrahepatic Cholestasis (PFIC) Epidemiology Forecast
The Progressive Familial Intrahepatic Cholestasis (PFIC) epidemiology section covers insights about historical and current Progressive Familial Intrahepatic Cholestasis (PFIC) patient pool and forecasted trends for every seven major countries (i.e., the United States, EU5 countries (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
It helps to recognize the causes of current and forecasted Progressive Familial Intrahepatic Cholestasis (PFIC) Epidemiology trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides the diagnosed patient pool and their trends, along with assumptions undertaken.
Progressive Familial Intrahepatic Cholestasis (PFIC) Epidemiology Segmentation
Prevalent Population of PFIC
Type-specific Prevalent Population of PFIC
Diagnosed and Treatable Cases of PFIC
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