Latest news with #PrimaryBiliaryCholangitis
Medscape
14 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.
Yahoo
20-05-2025
- Business
- Yahoo
GENFIT to receive a €26.5 million milestone payment following the approval of pricing and reimbursement of Ipsen's Iqirvo® in Italy
Lille (France), Cambridge (Massachusetts, United States), Zurich (Switzerland), May 20, 2025 – GENFIT (Nasdaq and Euronext: GNFT), a biopharmaceutical company dedicated to improving the lives of patients with rare and life-threatening liver diseases, today announced that Ipsen's Iqirvo®1 has been granted pricing and reimbursement in Italy for Primary Biliary Cholangitis (PBC). This major step unlocks a new milestone payment of €26.5 million under our Licensing and Collaboration Agreement with Ipsen2, due upon pricing and reimbursement of Iqirvo® (elafibranor) in three major European markets3, and will allow us to continue progressing our pipeline in Acute On-Chronic Liver Failure (ACLF) and other life-threatening diseases. Milestone payments under the Licensing and Collaboration Agreement with Ipsen are not included in the scope of our royalty financing agreement with HCRx4. ABOUT GENFIT GENFIT is a biopharmaceutical company committed to improving the lives of patients with rare, life-threatening liver diseases whose medical needs remain largely unmet. GENFIT is a pioneer in liver disease research and development with a rich history and a solid scientific heritage spanning more than two decades. Today, GENFIT has built up a diversified and rapidly expanding R&D portfolio of programs at various stages of development. The Company focuses on Acute-on-Chronic Liver Failure (ACLF). Its ACLF franchise includes five assets under development: VS-01, G1090N, SRT-015, CLM-022 and VS-02-HE, based on complementary mechanisms of action using different routes of administration. Other assets target other serious diseases, such as cholangiocarcinoma (CCA), urea cycle disorder (UCD) and organic acidemia (OA). GENFIT's expertise in the development of high-potential molecules from early to advanced stages, and in pre-commercialization, was demonstrated in the accelerated approval of Iqirvo® (elafibranor5) by the U.S. Food and Drug Administration, the European Medicines Agency and the Medicines and Healthcare Regulatory Agency in the UK for Primary Biliary Cholangitis (PBC). Beyond therapies, GENFIT also has a diagnostic franchise including NIS2+® in Metabolic dysfunction-associated steatohepatitis (MASH, formerly known as NASH for non-alcoholic steatohepatitis) and TS-01 focusing on blood ammonia levels. GENFIT is headquartered in Lille, France and has offices in Paris (France), Zurich (Switzerland) and Cambridge, MA (USA). The Company is listed on the Nasdaq Global Select Market and on the Euronext regulated market in Paris, Compartment B (Nasdaq and Euronext: GNFT). In 2021, Ipsen became one of GENFIT's largest shareholders, acquiring an 8% stake in the Company's capital. FORWARD LOOKING STATEMENTS This press release contains certain forward-looking statements, including those within the meaning of the Private Securities Litigation Reform Act of 1995. The use of certain words, such as "believe", "potential", "expect", 'target', 'may', 'will', "should", "could", "if" and similar expressions, is intended to identify forward-looking statements. Although the Company believes its expectations are based on the current expectations and reasonable assumptions of the Company's management, these forward-looking statements are subject to numerous known and unknown risks and uncertainties, which could cause actual results to differ materially from those expressed in, or implied or projected by, the forward-looking statements. These risks and uncertainties include, among others, the uncertainties inherent in research and development, including in relation to safety of drug candidates, cost of, progression of, and results from, our ongoing and planned clinical trials, patient recruitment, review and approvals by regulatory authorities in the United States, Europe and worldwide, of our drug and diagnostic candidates, pricing, approval and commercial success of elafibranor in the relevant jurisdictions, exchange rate fluctuations, and our continued ability to raise capital to fund our development, as well as those risks and uncertainties discussed or identified in the Company's public filings with the AMF, including those listed in Chapter 2 "Risk Factors and Internal Control" of the Company's 2024 Universal Registration Document filed on April 29, 2025 (no. 25-0331) with the Autorité des marchés financiers ("AMF"), which is available on GENFIT's website ( and the AMF's website ( and those discussed in the public documents and reports filed with the U.S. Securities and Exchange Commission ("SEC"), including the Company's 2024 Annual Report on Form 20-F filed with the SEC on April 29, 2025 and subsequent filings and reports filed with the AMF or SEC or otherwise made public, by the Company. In addition, even if the results, performance, financial position and liquidity of the Company and the development of the industry in which it operates are consistent with such forward-looking statements, they may not be predictive of results or developments in future periods. These forward-looking statements speak only as of the date of publication of this press release. Other than as required by applicable law, the Company does not undertake any obligation to update or revise any forward-looking information or statements, whether as a result of new information, future events or otherwise. CONTACTS GENFIT | InvestorsTel: +33 3 2016 4000 | investors@ GENFIT | MediaStephanie Boyer – Press relations | Tel: +333 2016 4000 | GENFIT | 885 Avenue Eugène Avinée, 59120 Loos - FRANCE | +333 2016 4000 | 1 Elafibranor is marketed and commercialized in the U.S by Ipsen under the trademark Iqirvo®2 3 Iqirvo® has already been granted pricing and reimbursement in the UK and in Germany4 Attachment GENFIT to receive a €26.5 million milestone payment following the approval of pricing and reimbursement of Ipsen's Iqirvo® in ItalyError in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
08-04-2025
- Health
- Yahoo
Edinburgh woman's 'menopause symptoms' turned out to be chronic liver disease
An Edinburgh woman who thought her liver disease symptoms was the menopause has opened up on her shocking diagnosis. Jo McCardle, 52, said she went through a 'really dark time' after she was told she had Primary Biliary Cholangitis - a liver disease that can get progressively worse over time. She first noticed symptoms back in 2022 including fatigue and brain fog. She also wasn't articulate and stopped paying attention to detail. READ MORE: Heartbreak for family in Edinburgh hospital after 'squint' in baby's eye is rare cancer READ MORE: Worried East Lothian locals 'can't have visitors' due to hoarding neighbour Speaking to Edinburgh Live, the Global Medical Affairs Manager said she put her feelings down to menopause before a blood test determined her diagnosis. She is now on lifetime medication but said she will not let the disease 'define her'. Jo added: "It has been such a journey for me. At the time, I had an incident at work and I had to go to A&£. "I had my bloods taken and they said there was something wrong with my blood results and I was referred to my GP. They phoned me after and said I had something serious going on but I didn't know what it was. "I was referred to a liver specialist and it was a really dark time for me. I didn't want to tell family everything that was going on. I did look up a lot of things but I didn't know what was happening - I just knew it was going to be a bad outcome. "Around three or four months later, I got the liver test to check the condition. I was relieved I wasn't dying but I did feel really ashamed at first. I remember feeling tired a lot, I had a lot of brain fog and I was forgetting things. "I didn't fell well at all and realised I wasn't paying attention to detail anymore. I just thought it was menopause I was going through." Jo was able to catch the disease at a good stage and is thankful her liver was in a treatable condition. She continued: "Luckily, although I have the condition it was picked up at a good stage and if it hadn't been, it could have been really damaged. It was pure luck I got the blood test done at work to pick it up. "This is just my normal now and I don't want to be defined by this - I want to live my life. It has really been a journey for me but it's made me appreciate everything a lot more. Join Edinburgh Live's Whatsapp Community hereand get the latest news sent straight to your messages. "Having the disease doesn't mean your life is over - I still have a very functioning job role and there is hope after the diagnosis of a long term condition. PBC is a lifelong condition associated with severe fatigue and persistent, debilitating itch – but can worsen over time and cause liver failure.. New medication Iqirvo has recently been approved which could help those impacted across Scotland. Around 1,900 people and 9 in 10 people living with PBC are woman, with most menopausal age around 40-60 years.
