NICE recommends Albireo’s Bylvay (odevixibat) for all PFIC types
pharmafile | February 23, 2022 | News story | Research and Development |
NICE has issued guidance that recommends Bylvay (odevixibat) for the treatment of all types of progressive familial intrahepatic cholestasis (PFIC) in people aged six months and older.
Bylvay is a potent, non-systemic ileal bile acid transport inhibitor (IBATi), administered as a once-daily treatment. This recommendation means it will be funded for use within 90 days in the NHS in England, Wales, Northern Ireland.
PFIC is the name given to a group of conditions in which liver cells do not properly release bile. This leads to a build-up of bile inside the liver cell, known as cholestasis. This can severely damage the liver, and liver cells can start to die and become replaced with scar tissue. This is a rare disorder which affects roughly one in 50,000.
“It’s incredibly gratifying to see children with PFIC gain access to the first ever non-surgical treatment option for a disease that causes tremendous suffering, in many cases leading to cirrhosis and liver failure within the first 10 years of life,” said Richard Thompson, Professor of Molecular Hepatology at King’s College London and principal investigator of PEDFIC 1 & PEDFIC 2 studies. “Odevixibat represents an important advance for patients, allowing us to respond to urgent treatment needs with a drug option that can provide clinically meaningful benefits and the ability to reduce the disease burden for patients and families who otherwise face PEBD surgery or liver transplant.”
“Children living with PFIC have an incurable devastating disease that causes pruritus which was treated with liver transplantation. Until the recommendation from NICE, we’ve had no approved drug options to address this devastating disease,” commented Professor Deirdre Kelly, Consultant Paediatric Hepatologist, Liver Unit, Birmingham Women’s and Children’s Hospital and University of Birmingham. “My colleagues and I are delighted that we now have an effective non-surgical treatment option to reduce the burden of this disease on children and families and improve the quality of their lives.”
Lina Adams
