Albireo's Bylvay Wins Positive Opinion from EMA For Rare Pediatric Liver Disease

European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended granting marketing authorization to Albireo Pharma Inc’s ALBO Bylvay (odevixibat) for the treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) in patients aged six months or older.
PFIC is a rare, life-threatening liver disease, and only about half of the children affected by the disease survive beyond the age of 10 years.
Results from a Phase 3 trial showed a significant reduction in serum bile acids accompanied by a substantial reduction in pruritus in those treated with odevixibat.
“These results were maintained in an ongoing, long-term open-label follow-up study. Hepatic parameters and fibrosis scores were improving or were stable for the duration of the study (max. 72 weeks). However, more data are needed to determine if odevixibat can delay disease progression and the need for liver transplantation. The CHMP, therefore, requested a registry-based efficacy study as a follow-up,” EMA said.
Price Action: ALBO shares are up 6.28% at $33.45 during the market session on the last check Friday.