Odevixibat

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."

Ipsen delivers solid sales growth in the first nine months of 2023 and confirms its full-year guidance

Retrieved on: 
Thursday, October 26, 2023
FDA, American Association for the Study of Liver Diseases, FY, Food, Progression-free survival, International, Heart, Hepatology, Committee, ADR, Rare disease, GMT, European Directive on Traditional Herbal Medicinal Products, CER1, Commerce, Arbitral tribunal, News, Symantec Endpoint Protection, European Medicines Agency, Alagille syndrome, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Research, ALGS, Cabozantinib, AB toxin, Webcast, Partnership, IPN, U.S. FDA, EMA, Primary biliary cholangitis, Committee for Medicinal Products for Human Use, Ipsen, Patient, YTD, AASLD, Fine chemical, Pharmaceutical industry, Cryptocurrency, Euronext, Neuroscience, Oncology, Odevixibat

Our portfolio has performed well across the three therapy areas, driven by strengthened commercial execution and the results of our external-innovation strategy.

Key Points: 
  • Our portfolio has performed well across the three therapy areas, driven by strengthened commercial execution and the results of our external-innovation strategy.
  • Based on the solid sales momentum, today we are confirming our guidance for the full year.
  • This was despite a positive opinion from the Committee for Medicinal Products for Human Use in July 2023.
  • The Company is planning to host a capital-markets event, starting at 12.30pm GMT on 7 December 2023 in London.

Ipsen updates on E.U. Marketing Authorisation Application for odevixibat in Alagille syndrome

Retrieved on: 
Monday, October 23, 2023
Ageing, Incidence, CHMP, Itch, Committee, ADR, European Directive on Traditional Herbal Medicinal Products, European Medicines Agency, PFIC, Development, COMP, Phase, Medicine, IPN, EMA, Cholestatic pruritus, Nutrition, Biliary atresia, Committee for Medicinal Products for Human Use, Safety, European Society for Medical Oncology, Ipsen, Odevixibat, Patient, Diagnosis, Pharmaceutical industry, Euronext, Research, Alagille syndrome, ALGS

This is despite a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) in July 2023.

Key Points: 
  • This is despite a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) in July 2023.
  • Orphan designation has a strong influence on the reimbursement mechanisms and access for patients to medicines in some countries in the E.U.
  • “We are disappointed with the opinion of the COMP as the Orphan Medicinal Product Regulation aims to stimulate research and development for rare diseases.
  • In the trial, there were no patient discontinuations and 96% of patients rolled over into the open-label extension trial.

Ipsen delivers solid H1 2023 results and upgrades its full-year guidance

Retrieved on: 
Thursday, July 27, 2023
IPN, Heart, Cholestatic pruritus, Maintenance, CHC, FY, Ascending cholangitis, Civil service commission, ADR, Prescription Drug User Fee Act, Fibrodysplasia ossificans progressiva, Ursodeoxycholic acid, Account, Ipsen, Galderma, Bank statement, Oxaliplatin, COMP, Rare disease, New Drug Application, European Directive on Traditional Herbal Medicinal Products, Research, Committee, EMA, ICC, Primary biliary cholangitis, Report, FOP, Patient, Committee for Medicinal Products for Human Use, PDUFA, Webcast, Consumer, Growth, Ageing, Alagille syndrome, Indoor Living, FDA, Regulation of tobacco by the U.S. Food and Drug Administration, Food, European Commission, Safety, CHMP, Pharmaceutical industry, Medical device, Cryptocurrency, ALGS, Oncology, Neuroscience, Odevixibat, Euronext

The performance in the first half of the year is reflected in today’s upgrade to our full-year guidance.

Key Points: 
  • The performance in the first half of the year is reflected in today’s upgrade to our full-year guidance.
  • The Company’s auditors performed a limited review of the H1 2023 condensed consolidated financial statements.
  • Ipsen intends to publish its year-to-date and third-quarter sales update on 26 October 2023.
  • The performance shown in this announcement covers the six-month period to 30 June 2023 (H1 2023) and the three-month period to 30 June 2023 (Q2 2023), compared to six-month period to 30 June 2022 (H1 2022) and the three-month period to 30 June 2022 (Q2 2022), respectively, unless stated otherwise.

Ipsen provides update on E.U. Marketing Authorization Application for Bylvay® for cholestatic pruritus in patients with Alagille syndrome  

Retrieved on: 
Friday, July 21, 2023
IPN, Maintenance, Ascending cholangitis, ADR, COMP, Skin, Quality of life, Biliary atresia, EMA, U.S. FDA, Committee, Incidence, Development, European Commission, Patient, Itch, Committee for Medicinal Products for Human Use, European Medicines Agency, Cholestasis, Liver, Ageing, Congress, European Society of Neurogastroenterology and Motility, Phase, PFIC, Cholestatic pruritus, CHMP, Pharmaceutical industry, ALGS, Nutrition, Ipsen, Alagille syndrome, Odevixibat, Euronext, Research

This negative COMP opinion prevents the retention of orphan-drug status in Bylvay’s marketing authorization in ALGS and might delay a final European Commission decision.

Key Points: 
  • This negative COMP opinion prevents the retention of orphan-drug status in Bylvay’s marketing authorization in ALGS and might delay a final European Commission decision.
  • for a second orphan indication for Bylvay, the treatment of pruritus in patients with ALGS.
  • More than 90% of patients were pruritus responders (≥ one point change at any time during 24 weeks).
  • In June 2023, Bylvay was also approved in the U.S. for the treatment of cholestatic pruritus in patients from 12 months of age with ALGS.

U.S. FDA approves Bylvay® for patients living with cholestatic pruritus due to Alagille syndrome

Retrieved on: 
Tuesday, June 13, 2023
IPN, ALGS, Cholestatic pruritus, Apache iBATIS, Ascending cholangitis, ADR, Ipsen, European Medicines Agency, European Directive on Traditional Herbal Medicinal Products, Biliary atresia, Trial of the century, EMA, Committee, Incidence, Development, MD, Patient, Physician, Hepatology, Child, Itch, Committee for Medicinal Products for Human Use, Division, NYU Langone Health, Cholestasis, Liver, Ageing, Hospital, Phase, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, PFIC, Food, Disease, Pharmaceutical industry, Alagille syndrome, Odevixibat, Euronext, Gastroenterology, Research

Bylvay was approved as the first drug treatment option for patients living with cholestatic pruritus due to progressive familial intrahepatic cholestasis (PFIC) in the U.S., and for the treatment of PFIC in Europe, in 2021.

Key Points: 
  • Bylvay was approved as the first drug treatment option for patients living with cholestatic pruritus due to progressive familial intrahepatic cholestasis (PFIC) in the U.S., and for the treatment of PFIC in Europe, in 2021.
  • More than 90% of patients were pruritus responders (≥ 1 point change at any time during 24 weeks).
  • No patients discontinued the study and 96% of patients rolled over into the open-label extension study.
  • In a third indication, the rare pediatric cholestatic liver disease, biliary atresia, Bylvay is in late-stage development with the Phase III BOLD trial.

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.

Ipsen completes acquisition of Albireo, expanding the scope of its Rare Disease portfolio

Retrieved on: 
Thursday, March 2, 2023
Apache iBATIS, Rare, Albireo, Biliary atresia, CVR, Alagille syndrome, Ageing, Hepatology, PSC, Patient, Ascending cholangitis, Itch, ALGS, PDUFA, PFIC, Primary sclerosing cholangitis, ADR, Liver disease, Priority review, Therapy, BA, EMA, NTCP, Acquisition, IPN, Pharmaceutical industry, Vaccine, Ipsen, Euronext, Phase, Odevixibat

The acquisition enriches Ipsen’s Rare Disease portfolio, with promising therapeutics for pediatric and adult rare cholestatic-liver diseases, innovative pipeline potential, as well as scientific and commercial capabilities.

Key Points: 
  • The acquisition enriches Ipsen’s Rare Disease portfolio, with promising therapeutics for pediatric and adult rare cholestatic-liver diseases, innovative pipeline potential, as well as scientific and commercial capabilities.
  • “The acquisition of Albireo will greatly strengthen our portfolio in rare diseases,” said David Loew, Chief Executive Officer of Ipsen.
  • “I am excited to welcome new colleagues to Ipsen, who led the innovation on the development of novel bile acid modulators, like Bylvay, to treat rare liver diseases in children and adults.
  • As part of the transaction, Ipsen has also acquired A3907 and A2342, two clinical-stage assets in Albireo’s pipeline.

FDA Grants June 15, 2023 PDUFA Date to Albireo for Bylvay® in Alagille Syndrome

Retrieved on: 
Tuesday, February 14, 2023
ALGS, Hepatology, PFIC, Life, New Drug Application, JAG1, Physician, Sleep, Shanda, Diagnosis, PDUFA, Safety, EAP, Bile, Priority review, Patient, Incidence, Liver, Liver disease, EMA, Biliary atresia, FOC, Food, Skeleton, NOTCH2, Microsoft Access, Apache iBATIS, Heart, Primary biliary cholangitis, FDA, Itch, U.S. FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Quality of life, Central nervous system, Prescription Drug User Fee Act, Ageing, Pharmaceutical industry, Alagille syndrome, Odevixibat, EU

With completed submissions for Bylvay in the U.S. and EU for use in patients with ALGS, the Company anticipates approvals in mid-2023.

Key Points: 
  • With completed submissions for Bylvay in the U.S. and EU for use in patients with ALGS, the Company anticipates approvals in mid-2023.
  • Over 90% of patients were pruritus responders and Bylvay was generally well tolerated; the overall incidence of treatment emergent adverse events (TEAEs) was similar to placebo.
  • “As an advocate for Alagille families, it is great to know physicians may have another drug treatment option for the debilitating pruritus that affects so many Alagille patients,” said Roberta Smith, President, Alagille Syndrome Alliance.
  • Alagille syndrome, or ALGS, is a rare, multisystem genetic disorder that the Company estimates impacts 25,000 people globally.