In mid-May 2023, Pharmaceutical Technology reported that a new combination treatment had earned Orphan Drug designation. This combo? Obeticholic acid (OCA) and bezafibrate. Together, they represent a potential therapeutic option for primary biliary cholangitis (PBC), a chronic liver disease that causes bile duct damage. 

Orphan Drug designation is granted by the FDA to treatments that have the potential to change the rare disease landscape. These treatments may help to prevent, diagnose, or treat a rare condition, which is defined as any condition affecting fewer than 200,000 people in the country. This status also comes with a number of benefits and incentives; if you’d like to learn more, check out this helpful FDA primer!

Ocaliva, an obeticholic acid treatment from Intercept Pharmaceuticals, was already approved for PBC nearly seven years ago. However, the company is now working to determine whether a fixed-dose combination of OCA and bezafibrate, a PPAR-α agonist which works to reduce cholesterol and triglycerides, could be even more effective. Outside of PBC, Intercept Pharmaceuticals is also looking at OCA as a treatment for nonalcoholic steatohepatitis (NASH).

Currently, Intercept Pharmaceuticals is performing interim analyses of Phase 2 clinical trials evaluating this combination. Data should be released by the end of June 2023. 

About Primary Biliary Cholangitis (PBC)

As described above, PBC is a kind of chronic liver disease characterized by slow, but progressive, bile duct damage. The bile ducts are located in the liver; bile, which aids in digestion, flows through these ducts. When the ducts become damaged, bile is unable to flow out. Bile builds up and causes scarring, inflammation, and even liver failure in later stages. The exact cause of PBC is unknown, though many people consider it an autoimmune disease. Others believe that environmental and hereditary factors may play a role. 

Primary biliary cholangitis is more common in females than males. It is also more common in people between ages 30-60 and those with a family history of this disease. Early symptoms of PBC may include severe fatigue, persistent itching on the bottoms of the feet, dry mouth, joint and muscle pain, skin darkening, and small white bumps under the skin around the eyes. As the disease progresses, additional symptoms may appear:

• Jaundice (yellowing of the skin, eyes, and mucous membranes)
• Underactive thyroid
• Chronic diarrhea
• Swelling of the lower extremities
• Abdominal ascites (distention due to fluid buildup) 
• Internal bleeding in the stomach and throat
• Weak and brittle bones
• Unintentional weight loss
• Upper right abdominal pain
• Enlarged spleen

Anti-itch medication, vitamin supplementation, and medications like Ocaliva and Actigall are currently used to treat PBC. There is no cure.