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At Intercept, our work is motivated by our desire to help patients and families who struggle with chronic liver diseases and need better treatment options.

PSC

Overview of PSC

  • PSC is a disease of unknown etiology characterized by progressive inflammation, fibrosis, and stricture formation in liver ducts
  • Many patients are asymptomatic, even with advanced disease
  • Common symptoms include pruritus and jaundice
  • The disease is strongly associated with inflammatory bowel disease (IBD)—about 5% of patients with ulcerative colitis will have PSC
  • Up to 70% of patients with PSC also have IBD, most commonly ulcerative colitis
  • PSC may be detected as an incidental finding of a raised serum alkaline phosphatase (ALP) level
  • Despite treatment, prognosis is poor for this progressive disease, with median survival of 12 years without a liver transplant

Natural history of PSC

In a study of 126 patients with PSC

  • 16% of patients were asymptomatic
  • Median follow-up from time of presentation was 5.8 years
  • More patients had liver transplants (21%) than patients who died of liver-related disease (16%)
  • Estimated median survival to these end points was 12 years; cholangiocarcinoma was found in 8 patients and in 23% of those undergoing liver transplant
  • Asymptomatic patients had milder disease than symptomatic patients
  • On multivariate analysis, the following independent prognostic factors were found:
    • Hepatomegaly
    • Splenomegaly
    • Elevated serum ALP
    • Advanced histological stage
    • Increasing age

Epidemiology

  • Rare in the general population
  • Data from Olmsted County (USA) in the year 2000 identified 20.9 cases of PSC per 100,000 of the population in men and 6.3 per 100,000 in women

Symptoms of PSC

  • Pruritus
  • Jaundice
  • Fatigue
  • Many patients are asymptomatic, even with advanced disease

Potential complications of PSC

Outcomes may include, but are not limited to

  • IBD
    • Ulcerative colitis
  • Carcinoma of the colon

Identifying patients with PSC

  • Male:female ratio of PSC is 2:1
  • 70% of all patients are male
  • 0.7% risk in first-degree relatives of patients with PSC, rising to 1.5% in siblings of affected individuals

Diagnosis of PSC

  • Mean age of diagnosis is 40 years of age for men and 45 for women
  • Main tests used for diagnosis are magnetic resonance cholangiography and endoscopic cholangiography

Treatment of PSC

  • American Association for the Study of Liver Disease guidelines for PSC recommend endoscopic retrograde cholangiography to exclude dominant stricture in patients with increases in serum bilirubin and/or worsening pruritus
    • Guidelines also recommend antimicrobial therapy with correction of bile duct obstruction in dominant strictures to effectively resolve cholangitis
  • In patients with recurrent bacterial cholangitis, prophylactic long-term antibiotics are recommended
    • However, patients with refractory bacterial cholangitis should be evaluated for liver transplant
  • European Association for the Study of the Liver guidelines for PSC recommend use of UDCA for treatment of PSC
    • UDCA may also have potential to prevent colonic neoplasia in patients with PSC and underlying IBD
  • A multitude of medications, including tacrolimus, methotrexate, corticosteroids, and azathioprine, have been used in PSC without consistent effect
  • Treatment with high-dose UDCA (20 to 30 mg/kg per day in divided doses) may offer some chemoprotective effect against colon cancer and may help to decrease symptoms, and biochemical and histological progression of PSC

Development of OCA for PSC

  • Intercept plans to further investigate OCA for the treatment of PSC in a Phase 2 trial

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