New Medicines

Primary sclerosing cholangitis


New molecular entity
Gilead Sciences
Gilead Sciences

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Jul 20Has orphan drug status in EU [3].


Non-steroidal agonist of the Farnesoid X receptor (FXR); FXR is the primary regulator of bile acid synthesis and plays important roles in glucose and lipid metabolism
PSC is a rare disease with a prevalence of 0.2-16 per 100,000. PSC commonly affects males with a median age at diagnosis of 35 years. PSC accounts for 10% of all UK liver transplants [1].
Primary sclerosing cholangitis

Trial or other data

Dec 20PIII PRIMIS study (NCT03890120) still recruiting with estimated primary completion date of Aug 23 [4]
Jul 20PIII PRIMIS study (NCT03890120) to evaluate whether cilofexor reduces the risk of fibrosis progression among non-cirrhotic adults with PSC starts. 400 adults will be recruited from sites including the US & EU (not UK). Primary outcome is proportion of participants with progression of liver fibrosis at week 96; collection of these data is due to complete Aug 22 [2].