Primary Sclerosing Cholangitis Market Analysis

Global Primary Sclerosing Cholangitis Market – Efficient Treatment Targeting Orphan Disease

Primary sclerosing cholangitis (PSC) is a condition causing long-term inflammation and scarring (fibrosis) of bile ducts in the liver. These ducts are tubes that allow the liquid (bile) produced by the liver to pass through the intestine allowing fat digestion. This disease predominantly affects men between the age of 25 and 40 years. The damage to the ducts causes accumulation of bile acids, essential components of bile to build up in the liver leading to liver tissue damage, portal hypertension (high blood pressure in the vessels connecting the liver and the gut), and liver failure, and may increase the risk of liver cancer.

Figure 1. Global Primary Sclerosing Cholangitis Market, by Drug

Global Primary Sclerosing Cholangitis Market- Optimistic Scenario:

Currently, over five products are in clinical trials Phase 2 and some of them have shown good results in Phase 2. For instance, ASEOP trial, testing Obeticholic acid (OCA) for primary sclerosing cholangitis, is being conducted by Intercept Pharmaceuticals, Inc. Also, Shire Plc. completed Phase 2 clinical trial for the drug candidate LUM001 in February, 2016.

If these potential molecules pass through the advanced clinical trials phase (i.e. Phase 3) and secure regulatory approval, these would be among the first-to-get-approval for this disease.

Moreover, most of the patients are in developed economies such as Europe and North America where diagnosis percentage and affordability is generally higher compared to emerging economies in regions such as Asia Pacific, which would result in higher adoption rate of novel products with significantly high yearly cost (i.e. more than US$ 50,000 for orphan drugs). Due to the above mentioned factors, the global primary sclerosing cholangitis market is expected to reach US$ 140.4 Mn by 2023.

However, withdrawal of potential therapeutic molecules from late-stage clinical trials have limited the availability of efficient treatment, thus compelling the patient pool to adopt alternative treatment therapies such as bile acid sequestrants, antibiotics, antihistamines, opioid antagonists, and ursodeoxycholic acid (UDCA).

According to a study published by Gastroenterology and Hepatology Journal, 2016, current medical therapies such as ursodeoxycholic acid improves liver enzymes. However, it does not prevent the progression of PSC or reduce the risk of recurrence. Thus, unavailability of approved drugs targeting the condition in practical medical use is a factor hindering growth of the global primary sclerosing cholangitis market.

Increasing incidence of liver failure and bile cancer associated with PSC

Increasing prevalence of diseases associated with PSC such as liver cancer and inflammatory bowel disease is expected to enhance the therapeutic adoption of efficient drugs to manage the condition, thus driving growth of the primary sclerosing cholangitis market. According to the World Journal of Gastroenterology, 2017, inflammatory bowel disease (IBD) is diagnosed in around 50-80% of the patients with PSC, with ulcerative colitis (UC) comprising around 80-90% of these cases.

Furthermore, individuals with PSC are at a greater risk of developing cancer in bile ducts called cholangiocarcinoma, as compared to general populace. According to the National Organization for Rare Disorders (NORD), 2016, around 8-15% of the PSC affected individuals eventually develop cholangiocarcinoma and have enhanced risk of developing gall bladder cancer. Moreover, NORD states that patients with PSC are at a greater risk of developing Crohn’s disease, colon cancer, and immune-mediated disorders such as thyroid disease, Peyronie’s disease, retroperitoneal fibrosis, psoriasis, rheumatoid arthritis, Celiac disease, and others.

Affordable options in comparison with the currently available treatments is expected to increase the adoption of PSC drugs

Currently, liver transplant is the only known cure for primary sclerosing cholangitis and is one of the major causes for liver transplantation in the U.S. However, as per the survey by Mayo Clinic, the disease is expected to recur in the transplanted liver in 27.2% of the patients, thereby demanding efficient and affordable therapeutic solutions to cater to unmet patient needs. According to the United Network for Organ Sharing (UNOS), 2011, estimated bill charges of a liver transplant procedure is around US$ 577,100 in the U.S.

Thus, requirement of providing affordable treatment, owing to high costs associated with liver transplantation coupled with increasing risk factors resulting in the development of serious disorders are major factors that are expected to increase the adoption of PSC drugs, in turn favoring market growth in the near future.

Major players operating in the global primary sclerosing cholangitis market include Acorda Therapeutics, Inc., Gilead Sciences, Inc., NGM Biopharmaceuticals, Inc., Intercept Pharmaceuticals, Inc., Dr. Falk Pharma GmbH, Allergan Plc., Shire Plc., Durect Corporation, Conatus Pharmaceuticals, Inc., Sirnaomics, Inc., and Shenzhen HighTide Biopharmaceutical Ltd.