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.
For more Health articles, visit www.foxnews/health
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 solution may be on the horizon for the 30 million people in the U.S. who suffer from sleep apnea.
In a Finland study, a new breathing device showed promising results in reducing symptoms of the disorder, according to researchers.
The device, called WellO2, uses resistance training to strengthen throat muscles and steam breathing to humidify airways.
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In the small study conducted by University of Turku’s Sleep Research Center, 25 participants with mild to moderate sleep apnea used the device at home twice a day for three months, breathing in and out 30 times per session.
At the end of the study period, the participants reported increased respiratory muscle strength, reduced nighttime breathing interruptions, improved sleep quality and decreased insomnia, according to a press release.
In a Finland study, a new breathing device, shown at right, has shown promising results in reducing symptoms of the disorder, according to researchers. (iStock/WellO)
Snoring occurrences were also reduced.
The findings were presented on June 5 at the Nordic Lung Congress 2024 in Helsinki, Finland.
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The study will now be peer-reviewed by the scientific congress, then will be published later this year, according to the researchers.
The device, called WellO2, uses resistance training to strengthen throat muscles, plus steam breathing to humidify airways. (WellO2)
“Currently, there are no available technologies or medications to treat sleep apnea,” lead author Dr. Usame Al-Rammahi, a doctoral researcher of pulmonary diseases and clinical allergology at the University of Turku, told Fox News Digital.
“Our first data suggest that respiratory muscle training may be the answer that not only alleviates symptoms, but also addresses the underlying cause of OSA by strengthening muscles.”
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CPAP (continuous positive airway pressure) is currently the most common treatment for sleep apnea — but about a third of patients struggle to adapt to it, Al-Rammahi noted.
WellO2 is designed to be used as an add-on therapy with CPAP.
CPAP (continuous positive airway pressure) is right now the most common treatment for sleep apnea, yet about a third of patients struggle to adapt to it. (iStock)
“Even though CPAP should be used as needed, WellO2 training can be used as an additional therapy, since it addresses underlying reasons and may lessen CPAP adverse effects by moistening the airways,” Al-Rammahi told Fox News Digital.
Limitations of the study
This is a preliminary report and research is currently ongoing, Al-Rammahi said.
The study also did not include a control group.
“There is no quick fix for respiratory rehabilitation.”
“Nonetheless, given that the study compared their results to each participant’s personal findings, those persons were the controls,” Al-Rammahi said.
“Despite the lack of a control group, the findings remain important. Qualified questionnaires were used, and polysomnography (PSG) was a thorough and trustworthy procedure that provided substantial results.”
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Dr. Chelsie Rohrscheib, a neuroscientist and sleep specialist at Wesper in New York, was not involved in the study but commented on the device’s potential.
“Sleep apnea is primarily caused by obstructions in the upper airway that block the flow of oxygen into the lungs,” she said.
Approximately 30 million people in the U.S. suffer from sleep apnea. (iStock)
“This device is designed to strengthen the muscles of the upper airway and reduce these obstructions from occurring,” she also told Fox News Digital.
While the device was shown to offer “some improvement” when tested in a small population of patients, Rohrscheib noted that use of the device was only correlated with “moderate improvement” in the number of respiratory events.
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“That means it’s not guaranteed to work for every patient or to completely eliminate your sleep apnea symptoms,” she said.
“Further, the device was not tested in patients with severe sleep apnea — thus, patients should not rely on this device alone for treatment for their sleep apnea without the guidance of a sleep physician.”
“Patients should not rely on this device alone for treatment for their sleep apnea without the guidance of a sleep physician,” a sleep expert said. (WellO2 )
Katri Lindberg, a respiratory specialist nurse in Finland who works with WellO2 as a breathing expert, said she recommends WellO2 training regularly for individuals who want to stop snoring or for whom CPAP devices are not suitable for treating sleep apnea.
“The research results are only preliminary findings from a small study, but they provide strong indications that we have found a rehabilitation tool that can now rehabilitate the symptoms of a large group of people worldwide, rather than merely alleviating them,” Lindberg told Fox News Digital.
“The study also indicates that there is no quick fix for respiratory rehabilitation. To remove symptoms and keep them at bay, regular, although relatively small, amounts of respiratory training are needed.”
Kidney transplants are not uncommon — the kidney is actually the most frequently transplanted organ in the U.S. — but a Chicago man recently received his in a highly unusual way.
John Nicholas, 28, was awake during the entire procedure, which was performed on May 24 at Northwestern Medicine Hospital. He was discharged the very next day.
Doctors administered a spinal anesthesia shot — similar to what is used for a Cesarean section — instead of general anesthesia, according to a press release from the hospital.
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“This is the first case at Northwestern Medicine where a patient was awake during an entire kidney transplant procedure and went home the next day, basically making this an outpatient procedure,” said Satish Nadig, M.D., PhD, transplant surgeon and director of the Northwestern Medicine Comprehensive Transplant Center, in the release.
“Inside the operating room, it was an incredible experience being able to show a patient what their new kidney looked like before placing it inside the body,” he added.
John Nicholas, the patient, is pictured with his surgeons in the operating room after completion of the kidney transplant. (Northwestern Medicine)
“It was incredibly simple and uneventful.”
This type of “awake transplant” could reduce surgical risks and shorten the length of the patient’s hospitalization, doctors say.
“It was an incredible experience, being able to show a patient what their new kidney looked like before placing it inside the body.”
It could also increase access to care for patients who are considered high-risk or have phobias surrounding general anesthesia.
Ideal candidate
Nicholas’ surgery took about two hours, according to the hospital.
He was considered an ideal patient for awake surgery due to his young age, his otherwise good health and his “eagerness to participate,” per the release.
John Nicholas is pictured awake during his kidney transplant in late May. (Northwestern Medicine)
“It was a pretty cool experience to know what was happening in real time and be aware of the magnitude of what they were doing,” said Nicholas in a statement to Northwestern.
“At one point during surgery, I recall asking, ‘Should I be expecting the spinal anesthesia to kick in?’ They had already been doing a lot of work and I had been completely oblivious to that fact. Truly, no sensation whatsoever.”
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Although Nicholas did receive sedation for comfort, he said he was still aware of what was going on.
“Especially when they called out my name and told me about certain milestones they had reached,” he noted.
Nicholas, center, is pictured after surgery with Dr. Vicente Garcia Tomas, his anesthesiologist, at left, and Dr. Nadig, transplant surgeon, at right. (Northwestern Medicine)
Nicholas walked out of the hospital on May 25, the day after surgery.
With typical kidney transplants, the recipient remains in the hospital for two to three days.
Saved by a friend
Nicholas’ kidney issues began at age 16 when he was diagnosed with Crohn’s disease, the release stated.
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After a few years of managing the condition with medication, his kidney function worsened and he required a transplant.
Nicholas’ mother originally planned to donate her kidney, but a breast cancer diagnosis prevented her from doing so, the hospital relayed.
Nicholas, at left, is pictured with his donor and best friend, Pat Wise, after surgery. (Northwestern Medicine)
It was Nicholas’ best friend from elementary school, 29-year-old Pat Wise in Alexandria, Virginia, who ultimately donated the life-saving kidney.
‘Another tool in our toolbelt’
Dr. Marc Siegel, clinical professor of medicine at NYU Langone Medical Center and a Fox News medical contributor, was not involved in the surgery but shared his input.
“My opinion is that in most cases, spinal anesthesia presents a reasonable alternative for those at high risk of general anesthesia complications,” Siegel told Fox News Digital.
Nicholas is pictured awake with his care team during his kidney transplant surgery. (Northwestern Medicine)
“Having said that, general anesthesia would be preferred whenever possible for major surgery like this,” said Dr. Siegel, “because control of the airway and breathing is essential, and is much easier if the patient is asleep and on the ventilator.”
The doctors at Northwestern credit the patient for helping to advance the field of transplant medicine.
Nicholas, left, and donor Wise are pictured at a press conference after the surgery. (Northwestern Medicine)
“When John agreed to be the first known patient at Northwestern Medicine to undergo an awake kidney transplant and be discharged home the next day, he knew the benefits outweighed the risks, and … he’s now helping to move the field of transplantation forward,” said Vinayak Rohan, M.D., transplant surgeon at Northwestern Memorial Hospital, in the release.
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“He is an extremely compliant patient who was in tune with his body and willing to push the envelope,” stressing that the patient had faith in the doctors and they, in turn, had faith in him.
Nicholas, center, is pictured with his entire care team. “He’s now helping to move the field of transplantation forward,” a surgeon said about the patient. (Northwestern Medicine)
The hospital now plans to establish the AWAKE Program (Accelerated Surgery Without General Anesthesia in Kidney Transplantation) for patients who want or need to pursue surgery without general anesthesia.
For more Health articles, visit www.foxnews/health
“It really opens up a whole new door and is another tool in our toolbelt for the field of transplantation,” Nadig added.
Fox News Digital reached out to Northwestern Medical for additional input.
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