Health
FDA approves new drug to treat autoimmune liver disease: ‘Giant step forward’
A new drug to control liver disease has gotten the green light.
The U.S. Food and Drug Administration (FDA) has approved the French drugmaker Ipsen’s medication Iqirvo (elafibranor).
The drug, an 80 mg tablet administered 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 of the liver.
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Without the active bile ducts, acids can then leak into the nearby tissue and cause liver damage or failure, according to the National Institutes of Health (NIH).
The U.S. Food and Drug Administration (FDA) has approved the French drugmaker Ipsen’s medication Iqirvo (elafibranor). (iStock)
The disease typically involves chronic inflammation along with a stagnant build-up of bile and toxins known as cholestasis, which can lead to irreversible scarring of the liver and ultimately destroy the bile ducts.
Although PBC is considered a rare condition, it can often go undetected, some health experts said.
“PBC is likely to be way underdiagnosed,” Dr. Douglas Dieterich, M.D., director of the Institute for Liver Medicine at Mount Sinai Health System in New York City, told Fox News Digital.
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“Many people — mostly women — have elevated liver enzymes that can be easily diagnosed with a simple blood test called the AMA.”
Patients typically experience severe fatigue and significant itching called pruritus.
If the disease is not treated or an individual does not respond to current therapies, it can lead to liver failure, the need for a liver transplant or even early death, according to experts.
PBC is a disease in which the immune system attacks and destroys the small bile ducts of the liver. Without the active bile ducts, acids can then leak into the nearby tissue and cause liver damage or failure. (iStock)
PBC is diagnosed with a blood test that measures liver enzymes.
One common test analyzes the patient’s alkaline phosphatase (ALP), an enzyme that helps detect liver or bone disease.
Another blood test to diagnose PBC measures antimitochondrial antibodies (AMAs), which are positive in approximately 95% of patients with the condition, according to several liver experts.
Patient welcomes more treatment options
One New York patient with PBC told Fox News Digital that she did not know she had a liver disease until her primary care physician performed routine blood work and noticed that her liver enzymes were elevated.
Meredith S., who withheld her last name for privacy reasons, was referred to a hepatologist, whom she credits for saving her life.
“It is distressing to know that your body is fighting itself and you can’t figure out how to stop it.”
“I was feeling tired, but attributed it to working and studying at school,” she told Fox News Digital.
“I was completely surprised to find out I had a liver disease and learned it was PBC.”
She went on, “My doctor performed a liver biopsy and I had significant scarring of my liver in my 30s, even though I didn’t drink alcohol.”
Patients have expressed relief at having another treatment option for liver disease. (iStock)
Meredith S. said she is glad there are more treatment options available, and hopes for more awareness and research of PBC.
“It is distressing to know that your body is fighting itself and you can’t figure out how to stop it,” she told Fox News Digital.
Addressing an ‘unmet need’
Dieterich of New York City, who is also a professor of medicine at the Icahn School of Medicine at Mount Sinai, told Fox News Digital that this newly approved medication “is a giant step forward in the treatment of PBC.”
He noted, “This is a giant step forward in the treatment of PBC. Up to now, there has been only one drug available to add to the urso, which is the basis for PBC treatment. Now there are two.”
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The existing drug, ursodeoxycholic acid (UDCA) — commonly called ursodial or “urso” — is a naturally occurring bile acid that has been used to treat liver disease for decades.
The newly approved Iqirvo (elafibranor) is intended to be used in combination with UDCA in patients who are not responding to the first medication on its own, or can be used on its own for people who cannot tolerate UDCA.
The disease typically involves chronic inflammation along with a stagnant build-up of bile and toxins known as cholestasis, which can lead to irreversible scarring of the liver and ultimately destroy the bile ducts. (iStock)
“For a significant number of people living with PBC, available treatments do not control the condition and may exacerbate symptoms of PBC,” said Christelle Huguet, executive vice president and head of research and development at Ipsen, in a press release.
“Iqirvo demonstrated statistically significant improvements in biochemical response compared to UDCA alone. Iqirvo is therefore a much-needed treatment option and the first new medicine for PBC in nearly a decade.”
Primary biliary cholangitis affects approximately 100,000 people in the U.S.
The accelerated approval for Iqirvo was based on positive results from the Phase III ELATIVE trial, which showed reduced levels of the alkaline phosphatase enzyme, which is elevated in people with liver disease.
The study, published in The New England Journal of Medicine, included 161 patients with PBC who were inadequately responding to treatment with UDCA or could not tolerate that medication.
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Researchers found that 51% of the patients who received the elafibranor had a biochemical response, compared to only 4% who received a placebo.
After 52 weeks, the patients who were treated with elafibranor showed normalized liver enzymes, compared to 15% of patients in the placebo group.
The study, which was published in The New England Journal of Medicine, included 161 patients with PBC. (iStock)
“Data from the pivotal Phase III ELATIVE clinical trial demonstrated that Iqirvo is an effective second-line treatment for patients with PBC with favorable benefit and risk data,” Dr. Kris Kowdley, a primary investigator on the ELATIVE study and the director at Liver Institute Northwest, Washington, said in a news release.
“The approval of Iqirvo will allow health care providers in the U.S. to address an unmet need, with the potential to significantly reduce ALP levels for our patients with PBC,” he added.
Continued approval is contingent upon further studies showing improved survival or prevention of liver decomposition, the FDA report noted.
Potential side effects and limitations
Some reported side effects of Iqirvo included muscle pain, rhabdomyolysis, myopathy, fractures, weight gain and drug-induced liver injury, according to the FDA report.
Some reported side effects of Iqirvo included muscle pain, rhabdomyolysis, myopathy, fractures, weight gain and drug-induced liver injury, according to the FDA report. (iStock)
Also noted was the potential risk to the fetus in pregnant patients, based on animal study data.
The FDA cautioned health care providers to ensure that patients are not pregnant prior to starting the medication.
Iqirvo is also not recommended in patients who have advanced stages of cirrhosis.
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Primary biliary cholangitis affects some 100,000 people in the United States, according to the drugmaker Ipsen.
It is a lifelong disease that can result in liver failure if left untreated, according to experts.
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A new study suggests that middle-aged men may be more vulnerable to faster biological aging, potentially linked to exposure to “forever chemicals.”
The research, published in the journal Frontiers in Aging, examined how perfluoroalkyl and polyfluoroalkyl substances, more commonly known as PFAS, could impact aging at the cellular level.
PFAS are synthetic chemicals commonly used in nonstick cookware, food packaging, water-resistant fabrics and other consumer products, the study noted.
Their chemical structure makes them highly resistant to breaking down, allowing them to accumulate in water, soil and the human body.
Chinese researchers analyzed blood samples from 326 adults enrolled in the U.S. National Health and Nutrition Examination Survey between 1999 and 2000.
A new study suggests that middle-aged men could face accelerated biological aging at the cellular level due to exposure to PFAS. (iStock)
The researchers measured levels of 11 PFAS compounds in participants’ blood and used DNA-based “epigenetic clocks” — tools that analyze chemical changes to DNA to estimate biological age — to determine how quickly their bodies were aging at the cellular level, the study stated.
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Two compounds, perfluorononanoic acid (PFNA) and perfluorooctanesulfonamide (PFOSA), were detected in 95% of participants.
Higher concentrations of those chemicals were associated with faster biological aging in men of certain age groups, but not in women.
“People should not panic.”
The compounds most strongly linked to accelerated aging were not the PFAS chemicals that typically receive the most public attention, the researchers noted.
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“The associations were strongest in adults aged 50 to 64, particularly in men,” Dr. Xiangwei Li, professor at Shanghai Jiao Tong University School of Medicine and the study’s corresponding author, told Fox News Digital.
“While this does not establish that PFAS cause aging, it suggests that these widely present ‘forever chemicals’ may be linked to molecular changes related to long-term health and aging.”
The study found that two of the compounds were detected in 95% of participants, and higher levels were linked to faster biological aging in men ages 50–64. (iStock)
Midlife may represent a more sensitive biological period, when the body becomes more vulnerable to age-related stressors, according to the researchers.
Lifestyle factors, such as smoking, may influence biological aging markers, potentially increasing vulnerability to environmental pollutants.
While Li said “people should not panic,” she does recommend looking for reasonable ways to reduce exposure.
That might mean checking local drinking water reports, using certified water filters designed to reduce PFAS, and limiting the use of stain- or grease-resistant products when alternatives are available.
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Meaningful reductions in PFAS exposure will likely depend on broader regulatory action and environmental cleanup efforts, Li added.
The researchers noted that midlife could be a particularly sensitive stage, when the body is more susceptible to stressors associated with aging. (iStock)
Study limitations
The researchers outlined several important limitations of the research, including that the findings show an association, but do not prove that PFAS directly causes accelerated aging.
“The study is cross-sectional, meaning exposure and aging markers were measured at the same time, so we cannot determine causality,” Li told Fox News Digital.
The study was also relatively small, limited to 326 adults age 50 or older, which means the findings may not apply to younger people or broader populations.
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Researchers measured PFAS levels using data collected between 1999 and 2000, and today’s exposure patterns may differ.
Li added that while PFAS is known to persist in the environment and the body, these results should be validated through larger, more recent studies that follow participants over time.
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A drug that has long been used to treat seizures has shown promise as a potential means of Alzheimer’s prevention, a new study suggests.
The anti-seizure medication, levetiracetam, was first approved by the FDA in November 1999 under the brand name Keppra as a therapy for partial-onset seizures in adults. The approval has since expanded to include children and other types of seizures.
Northwestern University researchers recently found that levetiracetam prevented the formation of toxic amyloid beta peptides, which are small protein fragments in the brain that are commonly seen in Alzheimer’s patients.
The medication was found to prevent the formation of amyloid-beta 42 in both animal models and cultured human neurons, according to the study findings, which were published in Science Translational Medicine.
The effect was also seen in post-mortem human brain tissue obtained from individuals with Down syndrome, who are at high risk for Alzheimer’s disease.
The medication was found to prevent the formation of amyloid-beta 42 in both animal models and cultured human neurons. (iStock)
“While many of the Alzheimer’s drugs currently on the market, such as lecanemab and donanemab, are approved to clear existing amyloid plaques, we’ve identified this mechanism that prevents the production of the amyloid‑beta 42 peptides and amyloid plaques,” said corresponding author Jeffrey Savas, associate professor of behavioral neurology at Northwestern University Feinberg School of Medicine, in a press release.
“Our new results uncovered new biology while also opening doors for new drug targets.”
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The brain is better able to avoid the pathway that produces toxic amyloid‑beta 42 proteins in younger years, but the aging process gradually weakens that ability, Savas noted.
“This is not a statement of disease; this is just a part of aging. But in brains developing Alzheimer’s, too many neurons go astray, and that’s when you get amyloid-beta 42 production,” he said.
The effect was also seen in post-mortem human brain tissue obtained from individuals with Down syndrome, who are at high risk for Alzheimer’s disease. (iStock)
That then leads to tau (“tangles”) — abnormal clumps of protein inside brain neurons — which can kill brain cells, trigger neuroinflammation and lead to dementia.
In order for levetiracetam to function as an Alzheimer’s blocker, high-risk patients would have to start taking it “very, very early,” Savas said — up to 20 years before elevated amyloid-beta 42 levels would be detected.
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“You couldn’t take this when you already have dementia, because the brain has already undergone a number of irreversible changes and a lot of cell death,” the researcher noted.
The researchers also did a deep dive into previous human clinical data to determine whether Alzheimer’s patients who were taking the anti-seizure drug had slower cognitive decline. They reported that the patients in that category had a “significant delay” in the span from cognitive decline to death compared to those not taking the drug.
“This analysis supports the positive effect of levetiracetam to slow the progression of Alzheimer’s pathology,” the researcher said. (iStock)
“Although the magnitude of change was small (on the scale of a few years), this analysis supports the positive effect of levetiracetam to slow the progression of Alzheimer’s pathology,” Savas said.
Looking ahead, the research team aims to find people who have genetic forms of Alzheimer’s to participate in testing, Savas said.
Limitations and caveats
The study had several limitations, including that it relied on animal models and cultured cells, with no human trials conducted.
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Because the study was observational in nature, it can’t prove that the medication caused the prevention of the toxic brain proteins, the researchers acknowledged.
Savas noted that levetiracetam “is not perfect,” cautioning that it breaks down in the body very quickly.
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The team is currently working to create a “better version” that would last longer in the body and “better target the mechanism that prevents the production of the plaques.”
“You couldn’t take this when you already have dementia, because the brain has already undergone a number of irreversible changes and a lot of cell death.”
The medication’s common documented side effects include drowsiness, weakness, dizziness, irritability, headache, loss of appetite and nasal congestion.
It has also been linked to potential mood and behavior changes, including anxiety, depression, agitation and aggression, according to the prescribing information. In rare cases, it could lead to severe allergic reactions, skin reactions, blood disorders and suicidal ideation.
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Funding for the study was provided by the National Institutes of Health and the Cure Alzheimer’s Fund.
Fox News Digital reached out to the drug manufacturer and the researchers for comment.
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