Progressive familial intrahepatic cholestasis

Mirum Pharmaceuticals Presents New Data at The Liver Meeting®

Retrieved on: 
Monday, November 13, 2023
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Infectious Diseases, Clinical Trials, Sleep, Hepatology, MDR3, Self-harm, Safety, Hepatocellular carcinoma, Bilirubin, Cholestasis, AASLD, Liver, Disease, Risk, Cholestatic pruritus, Hematology, Quality of life (healthcare), MYO5B, PFIC, Patient, Publication, Pregnancy, Nature, TJP2, Ascending cholangitis, GI, Hippeastrum mirum, Liver support system, Volixibat, ICP, IBAT, Progressive familial intrahepatic cholestasis, FIC, Augmented fifth, Growth, Doctor of Philosophy, Quality of life, AND, Itch, Medication, MCID, HIV disease progression rates, SBA, Chloride, Pharmaceutical industry, Nursing, Medical imaging, Dietary supplement, Boston, BSEP

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced new data from its LIVMARLI and volixibat programs presented at The Liver Meeting®, the American Association for the Study of Liver Diseases (AASLD) annual meeting in Boston, Massachusetts.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced new data from its LIVMARLI and volixibat programs presented at The Liver Meeting®, the American Association for the Study of Liver Diseases (AASLD) annual meeting in Boston, Massachusetts.
  • “PFIC is a chronic, debilitating cholestatic liver disease characterized by elevated bile acids and pruritus so severe that patients can be listed for liver transplant even in the absence of disease progression.
  • These data suggest overall improved liver health with maralixibat treatment in patients with PFIC that can be maintained long-term.
  • No clinically meaningful changes in liver enzyme levels or hematology parameters were observed after volixibat treatment.

Ipsen and Medison Pharma Announce Health Canada Approval of Bylvay™ (odevixibat) for the treatment of pruritus due to Progressive Familial Intrahepatic Cholestasis (PFIC)

Retrieved on: 
Monday, November 13, 2023
Nutrition, Medicine, Progressive familial intrahepatic cholestasis, Health Canada, IPN, Hope, Ageing, Hospital, Medison, PFIC, Patient, Child, Partnership, ADR, Physician, Itch, Hospital for Sick Children, Pharmaceutical industry, Nursing, Odevixibat, Euronext, Gastroenterology, Ipsen

ZUG, Switzerland and PARIS, Nov. 13, 2023 /PRNewswire/ -- Today, Ipsen (Euronext: IPN) (ADR: IPSEY), a global biopharmaceutical company focused on transformative medicines in oncology, rare disease and neuroscience, and Medison Pharma ("Medison"), a global pharma company focused on providing access to highly innovative therapies to patients in international markets, are pleased to announce the Health Canada approval for Bylvay™ (odevixibat) for the treatment of pruritus in patients aged 6 months or older with Progressive Familial Intrahepatic Cholestasis (PFIC), a progressive and life-threatening liver disease.  Bylvay is the first medication approved in Canada for the treatment of pruritus in patients aged six months or older with PFIC.

Key Points: 
  • Bylvay is the first medication approved in Canada for the treatment of pruritus in patients aged six months or older with PFIC.
  • "At Medison, accelerating patients' access to innovative treatments is leading all that we do, so we are pleased and proud to bring Bylvay to patients in Canada," said Gil Gurfinkel, CEO at Medison Pharma.
  • "Until today, surgery was the only treatment option available for PFIC patients," said Mr. Walter Perez, President, Progressive Familial Intrahepatic Cholestasis Advocacy and Resource Network and father of a child living with PFIC.
  • We are extremely pleased that a less invasive treatment option is now available."

Ipsen and Medison Pharma Announce Health Canada Approval of Bylvay™ (odevixibat) for the treatment of pruritus due to Progressive Familial Intrahepatic Cholestasis (PFIC)

Retrieved on: 
Monday, November 13, 2023
Nutrition, Medicine, Progressive familial intrahepatic cholestasis, Health Canada, IPN, Hope, Ageing, Hospital, Medison, PFIC, Patient, Child, Partnership, ADR, Physician, Itch, Hospital for Sick Children, Pharmaceutical industry, Nursing, Odevixibat, Euronext, Gastroenterology, Ipsen

ZUG, Switzerland and PARIS, Nov. 13, 2023 /PRNewswire/ -- Today, Ipsen (Euronext: IPN) (ADR: IPSEY), a global biopharmaceutical company focused on transformative medicines in oncology, rare disease and neuroscience, and Medison Pharma ("Medison"), a global pharma company focused on providing access to highly innovative therapies to patients in international markets, are pleased to announce the Health Canada approval for Bylvay™ (odevixibat) for the treatment of pruritus in patients aged 6 months or older with Progressive Familial Intrahepatic Cholestasis (PFIC), a progressive and life-threatening liver disease.  Bylvay is the first medication approved in Canada for the treatment of pruritus in patients aged six months or older with PFIC.

Key Points: 
  • Bylvay is the first medication approved in Canada for the treatment of pruritus in patients aged six months or older with PFIC.
  • "At Medison, accelerating patients' access to innovative treatments is leading all that we do, so we are pleased and proud to bring Bylvay to patients in Canada," said Gil Gurfinkel, CEO at Medison Pharma.
  • "Until today, surgery was the only treatment option available for PFIC patients," said Mr. Walter Perez, President, Progressive Familial Intrahepatic Cholestasis Advocacy and Resource Network and father of a child living with PFIC.
  • We are extremely pleased that a less invasive treatment option is now available."

Mirum Pharmaceuticals Showcases Five New LIVMARLI (maralixibat) Presentations at the EASL Congress

Retrieved on: 
Friday, June 23, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, Chloride, ALGS, Maintenance, EASL, Hippeastrum mirum, Cholestasis, SBA, Sleep, Research, Doctor of Philosophy, MRX, Medication, Congress, HIV disease progression rates, Patient, Concentration ratio, Bilirubin, Itch, Progressive familial intrahepatic cholestasis, IBAT, Alagille syndrome, Ageing, European Association for the Study of the Liver, PFIC, Safety, Pharmaceutical industry, Medical imaging, Fertilizer, Surveying, Toy, Liver

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced data presented during the European Association for the Study of the Liver (EASL) Congress 2023, taking place in Vienna, Austria.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced data presented during the European Association for the Study of the Liver (EASL) Congress 2023, taking place in Vienna, Austria.
  • The congress presentations feature new analyses from the LIVMARLI® (maralixibat) oral solution MARCH-PFIC study as well as the long-term extension study, MARCH-ON.
  • Additionally, data showing LIVMARLI’s benefit in adult patients with Alagille syndrome (ALGS) was presented during EASL.
  • All presentations are available in the Publications and Presentations section on Mirum’s website.

Virion Therapeutics Presents VRON-0200 Preclinical Data Showing Direct Correlations Between Intrahepatic CD8+ T cells and Hepatitis B Virus Declines (HBV) at EASL 2023

Retrieved on: 
Wednesday, June 21, 2023
EASL, American Association for the Study of Liver Diseases, Vaccination, Cancer, AAV, Virus, Congress, Progressive familial intrahepatic cholestasis, Hepatology, Blood, Mouse, Viral load, GD, European Association for the Study of the Liver, Therapy, HBV, Vaccine

These data were recognized as one of the Top 77 posters (out of 1,700 plus presentations) by the meeting organizers.

Key Points: 
  • These data were recognized as one of the Top 77 posters (out of 1,700 plus presentations) by the meeting organizers.
  • Chronic HBV infection impairs CD8+ T cell functions resulting in the loss of virus control.
  • That makes VRON-0200 so promising," said Hildegund Ertl, MD, Professor, Vaccine & Immunotherapy Center, at The Wistar Institute in Philadelphia PA and Scientific Founder of Virion Therapeutics.
  • The above presentation is available via the EASL-ILC Meeting Site at: https://www.easlcongress.eu , and on Virion's website at https://www.viriontx.com/science/presentations-publications/ .

Mirum Pharmaceuticals to Present New LIVMARLI Data at the 2023 EASL Congress

Retrieved on: 
Wednesday, June 7, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, Abstract, EASL, Hospital for Sick Children (Toronto), Sleep, Publication, Medication, Patient, Itch, Bilirubin, Progressive familial intrahepatic cholestasis, Hospital, European Association for the Study of the Liver, Congress, Safety, Pharmaceutical industry, Vaccine

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that it will present data at the upcoming International Liver Congress of the European Association for the Study of the Liver taking place June 21-24, 2023 in Vienna, Austria.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that it will present data at the upcoming International Liver Congress of the European Association for the Study of the Liver taking place June 21-24, 2023 in Vienna, Austria.
  • Data from Mirum’s studies of LIVMARLI® (maralixibat) oral solution include oral and poster presentations:
    Abstract OS-072: Maralixibat leads to significant reductions in bilirubin for patients with Progressive Familial Intrahepatic Cholestasis: Data from MARCH-PFIC
    Oral presentation during Abstract Session 2: Immune-mediated and cholestatic diseases
    Poster presentation during Poster Session: Immune-mediated and cholestatic diseases
    Author: Dr. Binita Kamath, The Hospital for Sick Children (SickKids), Toronto, Ontario, Canada
    WED-252: Maralixibat leads to significant reductions in pruritus and improvements in sleep for children with Progressive Familial Intrahepatic Cholestasis: Data from MARCH-PFIC
    Poster presentation during Poster Session: Immune-mediated and cholestatic disease
    WED-282: Analysis of safety in maralixibat-treated participants with Progressive Familial Intrahepatic Cholestasis: Data from MARCH-PFIC
    The abstracts are available on the EASL website and during the congress, the presentations will be available within the Publications and Presentations section on Mirum’s website.

Ipsen to present new Bylvay® (odevixibat) data at annual ESPGHAN congress, showcasing commitment to furthering treatment for rare cholestatic liver diseases

Retrieved on: 
Wednesday, May 17, 2023
IPN, ALGS, American Gastroenterological Association, Ascending cholangitis, ADR, Diarrhea, Ipsen, Progressive familial intrahepatic cholestasis, Safety, Sleep, NYU, New York University Grossman School of Medicine, Medical school, Medicine, Alagille syndrome, Research and development, Development, Liver transplantation, Cell biology, Augmented fifth, Medical University of Innsbruck, Itch, NYU Langone Health, Poster, European Society of Neurogastroenterology and Motility, Abstract, News, Pharmaceutical industry, Nursing, Nutrition, Pediatrics, Odevixibat, Euronext, Patient, Research, PFIC

The six data presentations, made up of four oral, one poster, and one e-poster, consistently demonstrate the efficacy and tolerability of investigational Bylvay® (odevixibat) for the potential treatment of cholestatic liver disease patients with Alagille syndrome (ALGS) and the treatment of patients with progressive familial intrahepatic cholestasis (PFIC).

Key Points: 
  • The six data presentations, made up of four oral, one poster, and one e-poster, consistently demonstrate the efficacy and tolerability of investigational Bylvay® (odevixibat) for the potential treatment of cholestatic liver disease patients with Alagille syndrome (ALGS) and the treatment of patients with progressive familial intrahepatic cholestasis (PFIC).
  • In PFIC, longer native liver survival suggests there may be a disease-modifying benefit to this treatment,” said Dr. Howard Mayer, Executive Vice President and Head of Research and Development, Ipsen.
  • Discoveries from a study with real-world data exploring diarrhea and quality of life issues in PFIC1 patients post liver transplant and the impact of Bylvay usage.
  • Follow Ipsen on Twitter via @IpsenGroup and keep up to date with ESPGHAN news and updates by using the hashtag #ESPGHAN23.

Albireo Presents Data on Greater Efficacy in PFIC with Earlier Bylvay® Treatment at NASPGHAN

Retrieved on: 
Thursday, October 13, 2022
Quality of life, Institute of Liver and Biliary Sciences, Caregiver, Disease, Liver, Liver disease, Growth, Itch, Cholestasis, Parent, Society, Albireo, Sleep, PICTURE, Nasdaq, Augmented fifth, Child–Pugh score, Genetic disorder, PFIC, Transplant, Adult, GLOBE, Progressive familial intrahepatic cholestasis, Poster, Dietary supplement, Pharmaceutical industry, Gastroenterology, Hepatology, Patient

BOSTON, Oct. 13, 2022 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (Nasdaq: ALBO), a rare disease company developing novel bile acid modulators to treat pediatric and adult liver diseases, today announced the presentation of new data at the NASPGHAN Annual Meeting being held in Orlando, Florida from October 12-15, 2022. A new analysis of pooled data from the landmark Phase 3 PEDFIC 1 and PEDFIC 2 trials demonstrates that Bylvay (odevixibat) reduces serum bile acids and pruritus in children with progressive familial intrahepatic cholestasis (PFIC) who have varying levels of baseline hepatic impairment. Importantly, the analysis shows that significantly more patients with mild hepatic impairment at baseline had a serum bile acid response, suggesting children may benefit from earlier treatment with Bylvay.

Key Points: 
  • Additional data analyses presented at the meeting show that Bylvay reduced pruritus in patients regardless of their baseline pruritus scores.
  • These new data provide important evidence that treating children with Bylvay earlier in their disease course could result in greater efficacy, said Jan Mattsson, Ph.D., Chief Scientific Officer and Head of R&D at Albireo.
  • Furthermore, the data we are presenting reinforce the medicines efficacy in patients with mild pruritus, suggesting patients benefit from beginning treatment at the first sign of itching.
  • As a result of risks and uncertainties that Albireo faces, the results or events indicated by any forward-looking statement may not occur.

Albireo Presenting New Bylvay™ (odevixibat) Data at NASPGHAN 2021

Retrieved on: 
Monday, December 13, 2021
UDCA, Sleep, Cholestasis, Liver disease, Nasdaq, Albireo, Ursodeoxycholic acid, Safety, Alagille syndrome, Disease, Quality of life, Data, Ageing, Progressive familial intrahepatic cholestasis, Natural history, GLOBE, Augmented fifth, Society, Pharmaceutical industry, Silviculture, PFIC, Patient, Itch

BOSTON, Dec. 13, 2021 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company developing novel bile acid modulators, will be presenting five posters on Bylvay (odevixibat) at the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) meeting being held December 12–18. Data will be presented that show evidence of correlations in serum bile acid reductions and long-term improvements in pruritus and sleep in patients with progressive familial intrahepatic cholestasis (PFIC); reductions in serum bile acids and improvements in pruritus with and without concomitant ursodeoxycholic acid (UDCA) and/or rifampicin use; and the efficacy and safety of Bylvay in patients with PFIC and prior partial external biliary diversion (PEBD). Data on disease burden and natural history of PFIC will be presented to describe the clinical characteristics of the disease. There will also be a poster presentation on the Phase 3 double-blind, randomized, placebo-controlled ASSERT study of Bylvay in Alagille syndrome (ALGS). Bylvay is a potent, non-systemic ileal bile acid transport inhibitor (IBATi) that is approved in the U.S. for the treatment of pruritus in patients 3 months of age and older in all types of PFIC and in Europe for the treatment of all types of PFIC in patients aged 6 months or older.

Key Points: 
  • Data on disease burden and natural history of PFIC will be presented to describe the clinical characteristics of the disease.
  • There will also be a poster presentation on the Phase 3 double-blind, randomized, placebo-controlled ASSERT study of Bylvay in Alagille syndrome (ALGS).
  • Safety and tolerability were comparable in patients using vs not using UDCA and/or rifampicin with Bylvay.
  • As a result of risks and uncertainties that Albireo faces, the results or events indicated by any forward-looking statement may not occur.

MULTIMEDIA UPDATE - Albireo Announces FDA Approval of Bylvay™ (odevixibat), the First Drug Treatment for Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)

Retrieved on: 
Tuesday, July 20, 2021
Medical specialties, Hepatology, Progressive familial intrahepatic cholestasis, PFIC, Cholestasis, Primary biliary cholangitis, Odevixibat, Liver, Itch, Cholestatic pruritus, Cholestasis facies

– Only once-daily drug indicated for the treatment of pruritus in PFIC –

Key Points: 
  • Albireo is launching Bylvay immediately to accelerate availability for the patients and families impacted by PFIC.
  • There are an estimated 100,000 patients with cholestatic liver disease without an approved drug treatment.
  • Bylvay is the first drug treatment approved in the U.S. for the treatment of pruritus in all subtypes of progressive familial intrahepatic cholestasis (PFIC).
  • Progressive familial intrahepatic cholestasis (PFIC) is a rare disorder that causes progressive, life-threatening liver disease.