While primary biliary cholangitis (PBC) currently has no cure, several therapies are available to slow the disease’s progression. In recent months, the United States and United Kingdom have approved new therapies to treat PBC.
While the cause of PBC remains unknown, and no treatments have therefore been developed to target the disease’s root cause, current medications aim to improve quality of life and delay liver damage, especially when started in the early stages of PBC.
Following is a roundup of current therapies to treat PBC.
What is PBC?
Primary biliary cholangitis (PBC) is a rare, chronic autoimmune disease that primarily affects the liver. In PBC, the body’s immune system mistakenly attacks the small bile ducts within the liver, causing inflammation and gradual destruction of these ducts. Bile, a substance essential for digesting fats and removing toxins, becomes trapped in the liver, leading to liver damage over time.
Elafibranor
Elafibranor (marketed under the name Iqirvo) is an oral drug that helps to alleviate the symptoms of PBC by reducing the synthesis of bile acid.
Read more about PBC therapies
In June, the U.S. Food and Drug Administration granted accelerated approval for elafibranor to treat PBC, the first country to do so. The FDA based its approval on the ELATIVE clinical trial, which demonstrated the efficacy of treatment of PBC with elafibranor plus ursodiol, a naturally occurring bile acid that helps to replace more toxic bile acids. Ursodiol is also known as ursodeoxycholic acid (UDCA) and is the primary treatment for PBC.
The trial demonstrated that patients treated with elafibranor exhibited greater improvements in levels of bilirubin and alkaline phosphatase (ALP), two biochemical markers of liver function.
In October, the U.K.’s Medicines and Healthcare products Regulatory Agency approved elafibranor’s use in that country, making it the first drug approved in the U.K. for the treatment of PBC.
Elafibranor may be prescribed on its own or in combination with ursodiol, depending on the patient’s tolerance of ursodiol.
Seladelpar
Seladelpar (Livdelzi) is an oral drug that belongs to the same class of drugs as elafibranor. It also works to decrease bile acid synthesis.
In August, the FDA gave accelerated approval to seladelpar to treat PBC on the basis of the RESPONSE trial, which found that 25% of participants who received seladelpar had normal ALP levels after one year of treatment. Seladelpar was also shown to reduce pruritus (itching), a common symptom of PBC.
Like elafibranor, seladelpar may be prescribed alone or in combination with ursodiol.
Obeticholic acid (OCA)
The FDA approved obeticholic acid (Ocaliva) in 2016 to treat PBC. OCA is often used in patients who cannot tolerate ursodiol or in combination with ursodiol among patients who do not have an adequate response to ursodiol alone. OCA regulates bile acid synthesis and reduces inflammation.
In June, the European Commission revoked its approval of OCA because of a report by the European Medicines Agency that the benefits do not outweigh the risks. But in September, the court temporarily suspended the revocation and reinstated OCA access in Europe.
Off-label treatment
Fibrates have been used for the treatment of PBC for many years off-label, meaning they have not been approved specifically to treat PBC. Fibrates work by lowering levels of triglycerides, a type of fat in the blood.
Fibrates may be recommended in some patients with PBC who cannot tolerate ursodiol, as they can also decrease the amount of bile production in the liver. Fenofibrate and bezafibrate are among the most common fibrates prescribed to patients with PBC.
