This Market Spotlight report covers the Primary Sclerosing Cholangitis (PSC) market, comprising key pipeline drugs and upcoming events, clinical trials, probability of success, and a 10-year disease prevalence forecast.

Key Takeaways

• Datamonitor Healthcare estimates that in 2021, there were 250,400 prevalent cases of primary sclerosing cholangitis (PSC) worldwide among adults aged 30 years and over, and forecasts that number to increase to 277,300 prevalent cases by 2028.
• The majority of industry-sponsored drugs in active clinical development for PSC are in Phase II, with just a single product each in Phase I and Phase III.
• Therapies in the pipeline for PSC focus on a wide variety of targets. The majority of pipeline drugs in development are administered via the oral route, with two products being tested in an intravenous formulation.
• High-impact upcoming events for drugs in the PSC space comprise topline Phase II trial results for CM-101, topline Phase IIa trial results for PLN-74809, and topline Phase IIb trial results for volixibat.
• The overall likelihood of approval of a Phase I autoimmune/immunology-other asset is 18.2%, and the average probability a drug advances from Phase III is 66.1%. Drugs, on average, take 10.2 years from Phase I to approval, compared to 9.6 years in the overall autoimmune/immunology space.
• The distribution of clinical trials across Phase I–IV indicates that the majority of trials for PSC have been in the early and midphases of development, with 82% of trials in Phase I–II, and only 18% in Phase III.
• The US leads in terms of the number of PSC clinical trials globally, while the UK leads the major European markets.
• Gilead Sciences has four completed trials and one ongoing trial in PSC.
• Gilead Sciences and Takeda are the only companies to have conducted Phase III trials for PSC.