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  • The FDA has granted accelerated approval to a novel drug for the treatment of a rare autoimmune disease that damages the liver.
  • Primary biliary cholangitis (PBC) occurs when the immune system attacks the bile ducts, leading to liver damage and other serious health outcomes.
  • Treatment options for PBC are limited and frequently not well-tolerated by patients.

The U.S. Food and Drug Administration(FDA) has just approved a first-in-class drug for the treatment of a rare autoimmune liver disease.

On June 10th, the FDA granted accelerated approval to the drug Iqirvo (elafibranor) to treat primary biliary cholangitis (PBC).

PBC is a rare autoimmune liver condition in which the small bile ducts in the liver are inflamed and damaged, causing bile and toxins to build up.

As the bile collects in the liver, it damages the surrounding tissue, which can lead to severe health problems like cirrhosis of the liver and destruction of the bile ducts.

Prior treatment options for PBC were limited and frequently not well-tolerated by patients. The definitive treatment for PBC requires a liver transplant. Experts say the approval of Iqirvo will meet a currently unmet need and help to expand options for patients.

“This is an exciting time for new therapies for the treatment of PBC. I am optimistic that the approval of elafibranor will allow more patients with PBC to have an improved prognosis as there will be additional options to provide for second line therapy to help achieve biochemical remission,” Aparna Goel, MD, a Clinical Associate Professor of Medicine, specializing in gastroenterology and hepatology at Stanford Medicine, told Healthline.

Iqirvo helps to treat PBC through several pathways, including promoting bile acid transportation, modulating bile production, and decreasing inflammation.

“Iqirvo is the first treatment advance for PBC in 8 years and is the very first in a new class of medicines called PPARs. PPARs are designed with the aim of targeting the underlying PBC disease,” a representative for Ipsen Biopharmaceuticals, the developer of Iqirvo, told Healthline.

The FDA granted the drug accelerated approval following a promising phase 3 trial, the results of which were published in the New England Journal of Medicine in November, 2023.

That trial involved 161 patients who had inadequate response to or unacceptable side effects with ursodeoxycholic acid, the current primary treatment for PBC.

“First line treatment for PBC is ursodeoxycholic acid. This has been the standard of care for many years. However, at least 60% of patients are either intolerant to or have an incomplete response to ursodeoxycholic acid,” said Goel.

About 67% of the patients in the trial received Iqirvo, while the rest received a placebo. After 52 weeks, half the individuals taking Iqirvo achieved a “statistically significant treatment benefit,” compared to only 4% of those taking the placebo.

Patients taking Iqirvo did experience some side effects during the trial. The most common side effects included abdominal pain, diarrhea, nausea, and vomiting.

Rhabdomyolysis, a serious and potentially fatal muscle-wasting disease, occurred in one individual taking Iqirvo.

“It will be important to monitor for any signs of muscle injury (rhabdomyolysis ) and kidney function as patients are started on this medication. As with any new medication, the long-term safety and efficacy will need to be carefully monitored,” said Goel.

PBC is a rare autoimmune disease. According to the latest statistics from the National Institutes of Health (2014), PBC affects about 58 in every 100,000 women and 15 in every 100,000 men in the United States. However, like other rare diseases, the exact number of people who have PBC isn’t entirely clear. Like other autoimmune conditions, PBC is also far more common in women than in men.

Overall, women account for about 80% of all cases of autoimmune disease.

In individuals with PBC, the immune system mistakenly attacks the bile ducts of the liver, causing inflammation and damage. Bile is an acidic fluid formed in the liver that is used in digestion. It is transported by the bile ducts to other organs like the small intestines where it can be used to break down food material.

Damaged bile ducts can not effectively transport bile, a condition known as cholestasis. As bile builds up in the liver, it can cause scarring (fibrosis), which can lead to irreversible, advanced cirrhosis. A patient may eventually require a liver transplant due to chronic liver damage.

PBC can be hard to diagnose as it begins with mild symptoms, including tiredness and itchy skin. As it progresses, other more severe symptoms like abdominal pain and joint pain may develop. In its final stages, highly visible symptoms may occur like darkening of skin color, the presence of fatty deposits under the skin, and jaundice.

The cause of PBC is still not well understood, but is believed to be the result of a combination of genetic and environmental factors that lead to the development of an autoimmune condition.

“Several environmental triggers have been associated with ‘activating’ the disease including toxins, viruses and several bacteria. As knowledge of the disease increases, appropriate recognition and testing of the disease is also increasing which is likely to change the prevalence estimates,” said Goel.

This month, the FDA granted accelerated approval to Iqirvo (elafibranor) for the treatment of primary biliary cholangitis (PBC).

PBC is a rare autoimmune disease in which the bile ducts are damaged, leading to inflammation and scarring of the liver.

Current treatment options for PBC are limited and often not well-tolerated. Experts say the new medication is promising, but will need to be monitored for continued safety.