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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The American Cancer Society (ACS) has updated its guidelines for colorectal cancer screening.
The organization released the update in its flagship journal on Wednesday, noting that the new recommendations “re-affirm” that adults at average risk should be screened for colorectal cancer at age 45 and continue through 75, for those with a life expectancy greater than 10 years.
In addition to the standard colonoscopy, the ACS also recommends that patients receive a blood-based screening test in a doctor’s office, which is designed to detect tumor DNA in the blood.
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The ACS also suggested an at-home screening option that looks for hidden blood and molecular markers in stool samples.
These new guidelines reflect recent advancements in disease detection, as well as a “critical shift in public health strategy to expand screening options and lower barriers to access,” the ACS stated in a press release.
The ACS recommends blood-based testing and at-home stool sampling as options for colorectal cancer screening. (iStock)
Dr. Robert Smith, senior vice president of early cancer detection science at the American Cancer Society and senior author of the report, wrote in a statement that colorectal cancer should be emphasized as a “highly preventable disease as much as a treatable one.”
“By offering more screening tools in our guideline update, more eligible adults will be able to participate in lifesaving colorectal cancer testing, helping to close the screening gap and catch more cancers at an earlier, treatable stage,” he added.
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According to the ACS, colorectal screening “dramatically improves survival,” as studies show early-stage detection yields a five-year survival rate of more than 90% in the U.S.
About one in three American adults are eligible for colorectal cancer screening but have not been tested, although ACS research marks colorectal cancer as the top cancer killer of adults under 50.
Colorectal cancer is the top cancer killer of adults under the age of 50, according to the ACS. (iStock)
People at a high risk of colorectal cancer may need to begin screening before age 45 or be screened more often, the ACS added. Those over 85 years old should no longer be screened for colorectal cancer, per the guidelines.
Dr. William Dahut, chief scientific officer at the ACS, commented that “no matter which test you choose, what’s most important is to get screened, and that includes underserved, rural and minority populations.”
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These guideline changes follow a surge in colorectal cancer diagnoses in younger individuals. Recent ACS research revealed a 50% relative increase in diagnoses in adults aged 45 to 49 from 2021 to 2022.
Dr. Aparna Parikh, medical director of the Center for Young Adult Colorectal Cancer at the Mass General Cancer Center, who is not affiliated with the ACS, shared that experts don’t “entirely understand why” cases are on the rise.
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“But it seems to be an interplay of a person’s risk factors, overall makeup and early exposures,” she previously told Fox News Digital. “[Those] include dietary exposures, environmental exposures and possible antibiotic exposures, as well as lifestyle factors in the right host.”
Changes in bowel habits are the primary red flag that should raise the suspicion of colorectal cancer. (iStock)
Another recent ACS study found that drinking heavily and consistently over an adult’s lifetime could lead to a higher risk of colorectal cancer.
Other known risk factors include family history, obesity, smoking, a diet high in red and processed meats, inflammatory bowel disease, and a personal history or family history of polyps.
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While there may be no symptoms of colorectal cancer before diagnosis, especially in the early stages, certain symptoms should not be overlooked, experts say.
Dr. Eitan Friedman, PhD, an oncologist and founder of The Suzanne Levy-Gertner Oncogenetics Unit at the Sheba Medical Center in Israel, confirmed to Fox News Digital that changes in bowel habits are the primary red flag that should raise the suspicion of colorectal cancer.
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Other symptoms include fatigue as a result of anemia, stomach pain or abdominal discomfort, rectal bleeding or blood in the stool, weakness and unexplained weight loss.
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The sudden death of Kyle Busch has drawn attention to a rare but devastating medical progression: when pneumonia escalates into fatal sepsis.
An ER doctor spoke with Fox News Digital about how sepsis can trigger a rapid health decline.
“Sepsis is actually not a specific disease or diagnosis, but rather the syndrome that occurs when the body has certain abnormal findings and a presumed infection,” said Dr. Kenneth J. Perry, a South Carolina-based emergency medicine physician.
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The markers of sepsis include elevated white blood cell counts, a high or low temperature, and elevated heart and respiratory rates, according to Perry. Because of this, a patient with pneumonia is often already technically septic by definition.
In the wake of Kyle Busch’s sudden passing, there is a focus on the rapid decline from pneumonia to fatal sepsis. (Getty; iStock)
While many people assume a worsening infection means bacteria are multiplying uncontrollably, it often has more to do with the body’s internal environment.
“It is often not the bacteria itself that is causing the specific decline,” Perry said. “In most cases, it is a cascade of inflammatory processes that are set in motion by the infection.”
When this inflammation spirals out of control, the body moves from having a manageable infection into severe sepsis. This is when otherwise healthy people can rapidly deteriorate.
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“The concerning thing that can happen with any individual … is that sepsis can then lead to low blood pressure, worsening vital signs and organ damage,” Perry said.
“As multiple organs fail, it becomes very difficult for the medical team to treat and can sometimes lead ultimately to death.”
“The medical evaluation provided to the Busch Family concluded that severe pneumonia progressed into sepsis, resulting in rapid and overwhelming associated complications,” the family shared in a statement. (James Gilbert/Getty Images)
It is very unlikely to have pneumonia and not have any symptoms, according to Perry. Early signs can mimic a severe flu, including fevers, chills, a productive cough, and chest or back pain in cases where the lung is infected.
When sepsis begins to take hold, time becomes the most critical factor. “We have known for a number of years that early antibiotic therapy is beneficial in the treatment of sepsis,” Perry said.
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If you or a loved one are managing an infection at home, the doctor says the following red flags mean you should bypass the clinic and head straight to the emergency room.
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- Shortness of breath or difficulty breathing
- A racing heart rate or fever that continues to worsen even after starting treatment
- Severe chest pain associated with a productive cough
The slide into sepsis is, in most cases, a cascade of inflammatory processes that are set in motion by the infection, the doctor said. (iStock)
While cases like Busch’s are tragic, Perry stressed that this shouldn’t cause widespread panic. Most patients with pneumonia do very well with standard oral antibiotics.
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The NASCAR star’s rapid decline underscores the importance of medical vigilance and “having a primary care physician with whom you have a good relationship,” according to the ER doctor.
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“Monitoring symptoms while having easy access to primary care is a very beneficial and appropriate plan for most patients,” he added.
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