US rare liver disease-focussed biotech Albireo Pharma (Nasdaq: ALBO) said today that the UK’s National Institute for Health and Care Excellence (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. With this final recommendation under the Highly Specialized Technologies (HST) pathway, Bylvay will be funded for use within 90 days in the National Health Service (NHS) in England, Wales and Northern Ireland.
“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,” he added.
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