Latest news with #UDCA
Medscape
9 hours ago
- Health
- Medscape
Novel Therapies for Primary Biliary Cholangitis
Primary biliary cholangitis (PBC) is a chronic, progressive autoimmune disease characterized by ongoing bile duct inflammation and elevated alkaline phosphatase (ALP). Ursodeoxycholic acid (UDCA) is well established as the initial treatment for PBC. This medication, which helps move bile through the liver, can improve liver function and reduce scarring of the liver. However, UDCA does not appear to provide significant improvement in itching or fatigue. In this ReCAP, Dr Marlyn Mayo of the University of Texas Southwestern Medical Center, Dallas, Texas, discusses novel therapies for PBC in patients not responding to or tolerating UDCA. She notes that obeticholic acid (OCA), the initial second-line therapy approved in 2016, is associated with increased itching, no fatigue improvement, and rare instances of liver worsening — emphasizing the need for safer, more effective second-line options. The approval of PPAR agonists seladelpar and elafibranor in 2024 marked a significant development, according to Dr Mayo. Both compounds demonstrated strong biochemical improvements in their phase 3 trials, effectively lowering ALP levels. A key advantage over OCA is that these PPAR agonists enhance tolerability by alleviating itching and appear to reduce fatigue, a previously untreatable symptom in PBC.
NDTV
11 hours ago
- Health
- NDTV
Celina Jaitley Opens Up About Cholestasis Diagnosis During Pregnancy: All About This Rare Liver Condition
Actress Celina Jaitly opened up about her challenges during pregnancy in a heartfelt note on Instagram. In the caption of the post, she revealed that she was diagnosed with cholestasis, a rare liver condition during pregnancy. She emphasized the seriousness of this condition and how it can affect both the mother and the baby during pregnancy. "Both my pregnancies were overwhelming. I had gestational diabetes, severe SPD, and in both, I developed #choleostasis of Pregnancy, a rare liver condition more common in twin pregnancies. It nearly broke me," she wrote. What is cholestasis? Cholestasis of pregnancy is a liver condition that typically occurs in late pregnancy. Cholestasis of pregnancy can be very uncomfortable and may increase the risk of complications for both the mother and the baby. Pregnancy hormones, genes and some environmental factors can contribute to cholestasis. In cholestasis, the flow of bile juice from the liver to the small intestine reduces or stops. As a result, bile builds up in the liver, and bile acids eventually enter the bloodstream. High levels of bile acids in the blood contribute to some serious symptoms and complications. What are the symptoms of cholestasis? Cholestasis of pregnancy triggers intense itching without a rash. "It starts with itching palms, soles, sleepless nights and a deep fear for your baby's safety. Cholestasis isn't "just a pregnancy itch." It's a warning from your liver that something's wrong," Jaitly added. Syptoms include: Itching, particularly on the hands and feet Dark urine color Pain in the right upper quadrant (RUQ), without gallstones Fatigue Loss of appetite Nausea Treatment for cholestasis during pregnancy Treatment mainly focuses on reducing itching and preventing potential complications. Your doctor may prescribe medication to relieve itching and other discomforts. They will also constantly monitor the baby's health. "I was treated with Ursodeoxycholic Acid (UDCA), which lowers bile acids and eases symptoms. Oat baths and pregnancy-safe topicals also helped," she added. Complications The actress also mentioned the possible complications of cholestasis in the detailed post. She wrote: Cholestasis can have serious impacts- For baby: Preterm birth Fetal distress Meconium-stained fluid Stillbirth (if untreated) For mother: Intense itching Elevated bile acids Risk of preeclampsia Emotional exhaustion "To every mother: If you're pregnant and experiencing persistent itching, especially at night, ask for a bile acid test. Early detection can save lives." "Pregnancy is sacred, but vulnerable that's why I want to keep speaking openly about maternal health. Because silence never saved a mother. But awareness just might... 🤍," she concluded. Cholestasis in pregnancy is more common in the last trimester of pregnancy when hormones are at their peak, but it usually resolves within a few days after delivery.
Hans India
18 hours ago
- Health
- Hans India
Celina Jaitly opens up about battling cholestasis and other medical conditions during pregnancy
Actress Celina Jaitly opened up about the health challenges she faced during her twin pregnancy, shedding light on a lesser-discussed aspect of motherhood. In a heartfelt revelation on Instagram, she shared that she battled gestational diabetes, cholestasis, and other medical conditions while expecting. By speaking candidly about her experience, Celina hopes to create more awareness around high-risk pregnancies and offer support to women navigating similar journeys. Sharing a series of her photos with her kids, the 'No Entry' actress wrote, '#maternalhealth - When the Itch Could Have Taken My Babies: My Battle With Cholestasis I was born with a rare genetic gift, a tendency to hyper ovulate. I had two spontaneous twin pregnancies, a trait passed down through generations of women in my family. I later learned my mother too had carried twins after my brother & me, babies she tragically lost after a fall in Ranikhet. Even my great-great-grandmother died from complications during a twin pregnancy.' 'Both my pregnancies were overwhelming. I had gestational diabetes and severe SPD, and in both, I developed #choleostasis of Pregnancy, a rare liver condition more common in twin pregnancies. It nearly broke me. It starts with itching palms, soles, sleepless nights and a deep fear for your baby's safety. Cholestasis isn't 'just a pregnancy itch.' It's a warning from your liver that something's wrong. After my wonderful gynaecologist diagnosed me, I was referred to a hepatologist a Jordanian Dr who held my hand in a Dubai hospital and said a prayer. A man of science and faith. That moment of compassion stayed with me and reminded me of how important compassion is to a patient.' Celina Jaitly also revealed that cholestasis during pregnancy is a liver condition that can have significant effects on both the baby and the mother. For the baby, it increases the risk of preterm birth, fetal distress, and the presence of meconium-stained amniotic fluid, which can lead to complications during delivery. In severe cases, if left untreated, cholestasis may even result in stillbirth. For the mother, the condition often causes intense itching—especially on the hands and feet—due to the build-up of bile acids in the bloodstream. The actress also mentioned that she was treated with Ursodeoxycholic Acid (UDCA), a medication commonly prescribed to lower bile acid levels and alleviate the discomfort associated with cholestasis.
Associated Press
28-04-2025
- Health
- Associated Press
Health Canada approves IQIRVO® as a first-in-class dual-peroxisome proliferator-activated receptor treatment for primary biliary cholangitis
IQIRVO® (elafibranor) is the first new medicine approved in nearly a decade for the treatment of rare cholestatic liver disease Primary Biliary Cholangitis MISSISSAUGA, ON, April 28, 2025 /CNW/ - Ipsen Biopharmaceuticals Canada Inc. today announced Health Canada has issued a Notice of Compliance with Conditions (NOC/c) approving IQIRVO® (elafibranor) for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA.1 PBC is a chronic, rare autoimmune liver disease where the immune system attacks the liver causing slow, progressive damage to the bile ducts.2,3 Canada has one of the highest rates of PBC incidence and prevalence worldwide, affecting 318 individuals for every million Canadians (approximately 13,000 people).4,5 PBC is a lifelong condition that can worsen over time if not effectively treated, leading to liver transplant and in some cases, premature death.6 'The treatment landscape for PBC has not seen advancement in nearly a decade, making IQIRVO® a significant step forward in filling an unmet need for Canadian patients,' said Dr. Mark G. Swain, Professor of Medicine, Cal Wenzel Family Foundation Chair in Hepatology. 'If left untreated, PBC can progress and lead to liver failure, which is why second line options are needed. Based on the Phase III ELATIVE® trial, IQIRVO® offers the potential to significantly reduce serum ALP levels, providing patients with an effective second line treatment option.' IQIRVO® is a first-in-class oral, once-daily peroxisome proliferator-activated receptor (PPAR) agonist. The accelerated approval by Health Canada was based on data from ELATIVE® trial, a randomized, placebo-controlled, phase III study that assessed alkaline phosphatase (ALP) and bilirubin as a composite biochemical surrogate endpoint.7 In the study, IQIRVO® demonstrated a statistically significant treatment benefit with 51% of patients on IQIRVO® achieving a biochemical response compared with 4% on the placebo arm for a 47% treatment difference regardless of baseline ALP.8 Biochemical response was defined as ALP less than 1.67 Upper Limit of Normal (ULN), an ALP decrease of greater than or equal to 15% from baseline and total bilirubin (TB) ≤ ULN at week 52.9 'PBC is a rare, slowly progressive autoimmune liver disease and because it is somewhat 'invisible' it may be misunderstood and dismissed by friends, family and even the medical community,' said Gail Wright, President of Canadian PBC Society. 'The impact of PBC on day-to-day life can be devastating, especially for those PBC patients that suffer from severe fatigue and debilitating itch. Unrelenting symptoms affect their ability to work and carry out tasks of daily living, which can result in social isolation, anxiety and depression. The Canadian PBC Society welcomes this approval underscoring the importance of having access to well-tolerated and effective treatment options for Canadians living with PBC that potentially slow disease progression.' PBC is the most common autoimmune liver disease, primarily affecting women aged 35-60, and impacting 1 in 1,000 women over the age of 40.10 Men are also susceptible but to a much lesser degree, with a ratio of one affected man for every nine affected women.11 'For many people living with PBC, the existing treatment does not control the condition, said Ed Dybka, General Manager, Ipsen Biopharmaceuticals Canada Inc. 'IQIRVO® is a first-in-class treatment and this approval underscores Ipsen's commitment to being a leader in developing innovations that address unmet needs for Canadians living with rare disease.' About the Phase III ELATIVE® Trial12 The ELATIVE® trial is a multi-centre, randomized double-blind, placebo-controlled Phase III clinical trial (n=161) that evaluated the efficacy and safety of IQIRVO® 80mg once daily plus UDCA (n=108) versus placebo plus UDCA (n=53). IQIRVO® or placebo was administered in combination with UDCA in 95% of patients and as monotherapy in 5% of patients who were unable to tolerate UDCA. The 52-week study was completed by 92% of participants with 97% of those who completed the study continuing in an extension study. The results were published in the New England Journal of Medicine.13 About IQIRVO® IQIRVO® (pronounced EYE-KER-VO) is an oral, once-daily, peroxisome proliferator-activated receptor (PPAR) agonist indicated for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.16 While the mechanism is not well understood, pharmacological activity that is potentially relevant to IQIRVO® therapeutic effects includes inhibition of bile acid synthesis through activation of PPAR-alpha and PPAR-delta.17 IQIRVO® has been approved by the Food and Drug Administration (FDA) in the United States, the European Medicines Agency (EMA) in Europe and the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA). The Health Canada approval of IQIRVO® further strengthens Ipsen's portfolio of treatments for rare cholestatic liver diseases available to patients in Canada. IQIRVO® is an 80 mg tablet administered orally once daily.18 To support access to IQIRVO® for eligible individuals in Canada, the IPSEN CARES ® patient support program is available as a resource to people living with PBC and their caregivers to provide educational support and address coverage, access and reimbursement questions (1-855-215-2288). IQIRVO® has been granted a Notice of Compliance with Conditions (NOC/c) and Health Canada has provided market authorization to this product on the condition that Ipsen carries out additional clinical trials to verify the anticipated benefit within an agreed upon time frame.19 The confirmatory trial for IQIRVO®, ELFIDENCE, is ongoing. For more safety and prescribing information, please refer to the IQIRVO® product monograph. About Ipsen in North America Ipsen is a global biopharmaceutical company focused on innovation and specialty care. At Ipsen, we focus our resources, investments and energy on discovering, developing and commercializing medicines in three key therapeutic areas – Oncology, Rare Disease and Neuroscience. Our North American operations are located in Cambridge, Massachusetts, one of the company's three global hubs. Based in the heart of Kendall Square, our fully integrated biopharmaceutical business includes Commercial, Research & Development, Manufacturing, and Business Development. Combined with our Canadian headquarters in Mississauga, Ontario, and other locations, we employ approximately 600 people in North America. For more information, please visit ELATIVE is a registered trade-mark of Genfit, used under licence by Ipsen Biopharmaceuticals Canada Inc. IQIRVO is a registered trade-mark of Genfit S.A., used under licence by Ipsen Biopharmaceuticals Canada Inc. Ipsen Cares is a registered trade-mark of Ipsen S.A. References SOURCE Ipsen Biopharmaceuticals Canada Inc.
Cision Canada
28-04-2025
- Health
- Cision Canada
Health Canada approves IQIRVO® as a first-in-class dual-peroxisome proliferator-activated receptor treatment for primary biliary cholangitis Français
IQIRVO ® (elafibranor) is the first new medicine approved in nearly a decade for the treatment of rare cholestatic liver disease Primary Biliary Cholangitis MISSISSAUGA, ON, April 28, 2025 /CNW/ - Ipsen Biopharmaceuticals Canada Inc. today announced Health Canada has issued a Notice of Compliance with Conditions (NOC/c) approving IQIRVO ® (elafibranor) for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA. 1 PBC is a chronic, rare autoimmune liver disease where the immune system attacks the liver causing slow, progressive damage to the bile ducts. 2,3 Canada has one of the highest rates of PBC incidence and prevalence worldwide, affecting 318 individuals for every million Canadians (approximately 13,000 people). 4,5 PBC is a lifelong condition that can worsen over time if not effectively treated, leading to liver transplant and in some cases, premature death. 6 "The treatment landscape for PBC has not seen advancement in nearly a decade, making IQIRVO ® a significant step forward in filling an unmet need for Canadian patients," said Dr. Mark G. Swain, Professor of Medicine, Cal Wenzel Family Foundation Chair in Hepatology. "If left untreated, PBC can progress and lead to liver failure, which is why second line options are needed. Based on the Phase III ELATIVE ® trial, IQIRVO ® offers the potential to significantly reduce serum ALP levels, providing patients with an effective second line treatment option." IQIRVO ® is a first-in-class oral, once-daily peroxisome proliferator-activated receptor (PPAR) agonist. The accelerated approval by Health Canada was based on data from ELATIVE ® trial, a randomized, placebo-controlled, phase III study that assessed alkaline phosphatase (ALP) and bilirubin as a composite biochemical surrogate endpoint. 7 In the study, IQIRVO ® demonstrated a statistically significant treatment benefit with 51% of patients on IQIRVO ® achieving a biochemical response compared with 4% on the placebo arm for a 47% treatment difference regardless of baseline ALP. 8 Biochemical response was defined as ALP less than 1.67 Upper Limit of Normal (ULN), an ALP decrease of greater than or equal to 15% from baseline and total bilirubin (TB) ≤ ULN at week 52. 9 "PBC is a rare, slowly progressive autoimmune liver disease and because it is somewhat 'invisible' it may be misunderstood and dismissed by friends, family and even the medical community," said Gail Wright, President of Canadian PBC Society. "The impact of PBC on day-to-day life can be devastating, especially for those PBC patients that suffer from severe fatigue and debilitating itch. Unrelenting symptoms affect their ability to work and carry out tasks of daily living, which can result in social isolation, anxiety and depression. The Canadian PBC Society welcomes this approval underscoring the importance of having access to well-tolerated and effective treatment options for Canadians living with PBC that potentially slow disease progression." PBC is the most common autoimmune liver disease, primarily affecting women aged 35-60, and impacting 1 in 1,000 women over the age of 40. 10 Men are also susceptible but to a much lesser degree, with a ratio of one affected man for every nine affected women. 11 "For many people living with PBC, the existing treatment does not control the condition, said Ed Dybka, General Manager, Ipsen Biopharmaceuticals Canada Inc. "IQIRVO ® is a first-in-class treatment and this approval underscores Ipsen's commitment to being a leader in developing innovations that address unmet needs for Canadians living with rare disease." About the Phase III ELATIVE ® Trial 12 The ELATIVE ® trial is a multi-centre, randomized double-blind, placebo-controlled Phase III clinical trial (n=161) that evaluated the efficacy and safety of IQIRVO ® 80mg once daily plus UDCA (n=108) versus placebo plus UDCA (n=53). IQIRVO ® or placebo was administered in combination with UDCA in 95% of patients and as monotherapy in 5% of patients who were unable to tolerate UDCA. The 52-week study was completed by 92% of participants with 97% of those who completed the study continuing in an extension study. The results were published in the New England Journal of Medicine. 13 The ELATIVE ® trial demonstrated that IQIRVO ® had a statistically significant treatment benefit with 51% of patients on IQIRVO ® achieving a biochemical response compared with 4% on the placebo arm, a treatment difference of 47% (95% CI 32, 57; p<0.0001) regardless of baseline ALP. Biochemical response was defined as ALP less than 1.67 Upper Limit of Normal (ULN), an ALP decrease of greater than or equal to 15% from baseline and total bilirubin (TB) ≤ ULN at week 52. ALP normalization at week 52 was a key secondary endpoint with 15% of IQIRVO ® -treated patients demonstrating normalization versus 0% placebo (p=0.002). The significant biochemical response to IQIRVO ® was further supported by data demonstrating reductions from baseline in ALP levels were sustained through week 52 and response was rapid, seen as early as Week 4 in the IQIRVO ® group. The most commonly reported (>10%) adverse drug reactions associated with IQIRVO ® in more than 10% of study participants (n=108) were abdominal pain (11%), diarrhea (11%), nausea (11%) and vomiting (11%). 14 These were non-serious and mild to moderate in severity. The most common adverse drug reaction leading to treatment discontinuation was blood CPK increased (3.7%). 15 About IQIRVO ® IQIRVO ® (pronounced EYE-KER-VO) is an oral, once-daily, peroxisome proliferator-activated receptor (PPAR) agonist indicated for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. 16 While the mechanism is not well understood, pharmacological activity that is potentially relevant to IQIRVO ® therapeutic effects includes inhibition of bile acid synthesis through activation of PPAR-alpha and PPAR-delta. 17 IQIRVO ® has been approved by the Food and Drug Administration (FDA) in the United States, the European Medicines Agency (EMA) in Europe and the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA). The Health Canada approval of IQIRVO ® further strengthens Ipsen's portfolio of treatments for rare cholestatic liver diseases available to patients in Canada. IQIRVO ® is an 80 mg tablet administered orally once daily. 18 To support access to IQIRVO ® for eligible individuals in Canada, the IPSEN CARES ® patient support program is available as a resource to people living with PBC and their caregivers to provide educational support and address coverage, access and reimbursement questions (1-855-215-2288). IQIRVO ® has been granted a Notice of Compliance with Conditions (NOC/c) and Health Canada has provided market authorization to this product on the condition that Ipsen carries out additional clinical trials to verify the anticipated benefit within an agreed upon time frame. 19 The confirmatory trial for IQIRVO ®, ELFIDENCE, is ongoing. For more safety and prescribing information, please refer to the IQIRVO ® product monograph. About Ipsen in North America Ipsen is a global biopharmaceutical company focused on innovation and specialty care. At Ipsen, we focus our resources, investments and energy on discovering, developing and commercializing medicines in three key therapeutic areas – Oncology, Rare Disease and Neuroscience. Our North American operations are located in Cambridge, Massachusetts, one of the company's three global hubs. Based in the heart of Kendall Square, our fully integrated biopharmaceutical business includes Commercial, Research & Development, Manufacturing, and Business Development. Combined with our Canadian headquarters in Mississauga, Ontario, and other locations, we employ approximately 600 people in North America. For more information, please visit ELATIVE is a registered trade-mark of Genfit, used under licence by Ipsen Biopharmaceuticals Canada Inc. IQIRVO is a registered trade-mark of Genfit S.A., used under licence by Ipsen Biopharmaceuticals Canada Inc. Ipsen Cares is a registered trade-mark of Ipsen S.A. SOURCE Ipsen Biopharmaceuticals Canada Inc.
