A new drug has been approved to curb liver disease.
The U.S. Food and Drug Administration (FDA) has announced that French pharmaceutical company Ipsen Medicine Ikirubo (Elafibranor).
The drug, available as an 80 mg tablet taken orally once daily, is intended to treat an autoimmune cholestatic liver disease called primary biliary cholangitis (PBC).
What is PBC?
PBC is a disease in which the immune system attacks and destroys the small bile ducts in the liver.
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When the bile ducts don’t function, acid can leak into nearby tissue, causing liver damage and failure, according to the National Institutes of Health (NIH).
The U.S. Food and Drug Administration (FDA) has approved the drug Iquirvo (elafibranor), made by French pharmaceutical company Ipsen. (iStock)
The disease usually involves chronic inflammation and a stagnant buildup of bile and toxins, called cholestasis, which can form irreversible scarring in the liver and eventually destroy the bile ducts.
Although PBC is considered a rare disease, it often goes undetected. Health Professionals Said.
“PBC is likely diagnosed quite late,” Douglas Dietrich, MD, director of the Liver Medicine Institute at Mount Sinai Health System in New York City, told Fox News Digital.
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“Many people, mainly women, have elevated liver enzymes, which can be easily diagnosed with a simple blood test called an AMA.”
Patients usually Extreme fatigue and intense itching called pruritus.
If the disease is not treated or does not respond to current treatments, it can lead to liver failure. Liver transplantation Experts say it could even lead to premature death.
PBC is a disease in which the immune system attacks and destroys the small bile ducts in the liver. When the bile ducts no longer function, acid can leak into nearby tissues, causing liver damage and liver failure. (iStock)
PBC is diagnosed by blood tests that measure liver enzymes.
One common test analyzes a patient’s status for alkaline phosphatase (ALP), an enzyme that helps detect liver and bone disease.
Another blood test to diagnose PBC measures antimitochondrial antibodies (AMA), which several liver experts say test positive in about 95% of people with PBC.
Patients Welcome More Treatment Options
A PBC patient in New York told Fox News Digital that he didn’t know he had liver disease. Doctor I had some routine blood work done and noticed that my liver enzymes were elevated.
Meredith S., who asked not to give her last name to protect her privacy, was referred to a liver specialist who she credits with saving her life.
“It’s hard when your body is fighting itself and you don’t know how to stop it.”
“I was feeling tired, but I thought it was because of work and school,” she told Fox News Digital.
“When I found out I had liver disease and it was PBC, I was really surprised.”
She continued: “Doctors performed a liver biopsy and found that I had significant scarring in my liver, even though I was in my 30s and didn’t drink alcohol.”
Patients express relief that there is now a new treatment option for their liver disease. (iStock)
Meredith S. said she is pleased to see more treatment options available and hopes there will be more awareness and research into PBC.
“It’s hard when your body is fighting itself and you don’t know how to stop it,” she told Fox News Digital.
Addressing “unmet needs”
Dieterich, a New York City-based professor of medicine at the Mount Sinai School of Medicine, told Fox News Digital that the newly approved drug is “a major step forward in the treatment of PBC.”
“Previously there was only one drug available, now there are two,” he said.
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The existing drug, ursodeoxycholic acid (UDCA), commonly known as ursodial or “urso,” is a naturally occurring bile acid that has been used to treat liver diseases for decades.
The newly approved Iquirvo (elafibranor) is intended for use in combination with UDCA for patients who do not respond to the first agent alone, but can also be used alone for patients who cannot tolerate UDCA.
The disease usually involves chronic inflammation and a stagnant buildup of bile and toxins, called cholestasis, which can form irreversible scarring in the liver and eventually destroy the bile ducts. (iStock)
“For many people with PBC, currently available treatments do not control the disease and may worsen PBC symptoms,” Christelle Huguette, executive vice president and head of research and development at Ipsen, said in a press release.
“Iqirvo demonstrated a statistically significant improvement in biochemical response compared to UDCA alone. Iqirvo is therefore a long-awaited treatment option and the first new drug for PBC in almost a decade.”
Primary biliary cholangitis affects approximately 100,000 people in the United States.
The accelerated approval of Ikirubo Phase III ELATIVE studyIt was shown to reduce levels of the alkaline phosphatase enzyme, which is elevated in patients with liver disease.
The study, published in the New England Journal of Medicine, included 161 patients with PBC who had not responded adequately to treatment with UDCA or who could not tolerate the drug.
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The researchers found that 51 percent of patients who received elafibranor experienced a biochemical response compared with just 4 percent of those who received a placebo.
After 52 weeks, liver enzymes normalized in 15% of patients receiving elafibranor compared with 15% of patients in the placebo group.
The study, published in the New England Journal of Medicine, involved 161 patients with PBC. (iStock)
“Data from the pivotal Phase 3 ELATIVE clinical trial demonstrate that Iqirvo is an effective second-line treatment for patients with PBC with favorable benefit-risk data,” Dr. Chris Cowdrey, principal investigator of the ELATIVE study and director of the Northwest Liver Institute in Washington, said in a news release.
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“The approval of Iquirvo will enable U.S. healthcare providers to address an unmet need, potentially significantly reducing ALP levels in patients with PBC,” he added.
Continued approval depends on further studies showing improved survival and prevention of liver decay, according to the FDA report.
Potential Side Effects and Limitations
Reported side effects of Ikirubo include: muscle painAccording to the FDA report, side effects reported include rhabdomyolysis, myopathy, bone fractures, weight gain, and drug-induced liver damage.
According to the FDA report, reported side effects of Iquirovo included muscle pain, rhabdomyolysis, myopathy, bone fractures, weight gain, and drug-induced liver injury. (iStock)
Potential risks to the fetus were also noted. Pregnant patientsBased on animal test data.
The FDA warned health care professionals to make sure their patients are not pregnant before starting the medication.
Iqirvo is also not recommended for patients with advanced cirrhosis.
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According to pharmaceutical company Ipsen, primary biliary cholangitis affects about 100,000 people in the United States.
Experts say it is a lifelong disease that can lead to liver failure if left untreated.
