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  • OC8 Long-term efficacy and safety of odevixibat in patients with progressive familial intrahepatic cholestasis: results with ≥96 weeks of treatment

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OC8 Long-term efficacy and safety of odevixibat in patients with progressive familial intrahepatic cholestasis: results with ≥96 weeks of treatment
  1. RJ Thompson1,
  2. Ö Durmaz2,
  3. T Grammatikopoulos1,3,
  4. A Di Giorgio4,
  5. Q Ni5,
  6. P Stein5,
  7. C Clemson5,
  8. E Sturm6
  1. 1Institute of Liver Studies, King’s College London, London, UK SE5 9RS
  2. 2Istanbul University, Istanbul Faculty of Medicine, Istanbul, Turkey
  3. 3Paediatric Liver, GI, and Nutrition Centre and MowatLabs, King’s College Hospital NHS Trust, London, UK SE5 9RS
  4. 4Paediatric Hepatology, Gastroenterology, and Transplantation, ASST Papa Giovanni XXIII, Bergamo, Italy
  5. 5Albireo Pharma, Inc., Boston, MA, USA
  6. 6Paediatric Gastroenterology and Hepatology, University Children’s Hospital Tübingen, Tübingen, Germany

Abstract

Patients with progressive familial intrahepatic cholestasis (PFIC) may present with elevated serum bile acids (sBAs), growth deficits, and impaired hepatic function. The phase 3 PEDFIC 1 and PEDFIC 2 studies evaluated odevixibat, an ileal bile acid transporter inhibitor, in patients with PFIC. Using pooled data from these studies, we describe key outcomes in patients treated with odevixibat for ≥96 weeks.

PEDFIC 1 (NCT03566238) was a 24-week, randomised, placebo-controlled study in children with PFIC1 and PFIC2. PEDFIC 2 (NCT03659916) is an ongoing, 72-week open-label extension study in patients with any PFIC type; an optional extension period follows PEDFIC 2. This pooled analysis spans from patients’ first dose of odevixibat to 31 January 2022. Outcomes included sBAs, hepatic parameters, growth, and safety.

Of the 111 patients in the pooled population (69 ongoing at data cut-off), 36 had ≥96 weeks’ odevixibat exposure and an sBA measurement at week 96 (36% PFIC1, 61% PFIC2, 3% MYO5B deficiency). At baseline, patients had elevated mean sBA, transaminase, and total bilirubin levels, and impaired growth (table 1). At week 96, mean sBA levels were significantly reduced (P<0.001); no changes were observed in bilirubin levels (table 1). All 36 patients (100%) had treatment-emergent adverse events (TEAEs); most were mild or moderate in severity. No drug-related serious TEAEs were reported.

Odevixibat treatment for ≥96 weeks was associated with improvements in sBAs, transaminase levels, and growth in patients with PFIC. Odevixibat was generally well tolerated.

View this table:
Abstract OC8 Table 1

Outcomes in patients with PFIC treated with odevixibat for ≥96 weeks

Disclosures R. J. Thompson: Albireo and Mirum – Consultant; Generation Bio – Consultant and stock options; Rectify Therapeutics – Consultant and stockholder

Ö. Durmaz: Nothing to disclose

T. Grammatikopoulos: Albireo – Consultant

A. Di Giorgio: Albireo – Travel support and speaker’s fee

Q. Ni, P. Stein, and C. Clemson: Albireo – Employment

E. Sturm: Albireo and Mirum – Consultant and research support; Univar – Consultant; Orphalan – Speaker’s fee

This study was sponsored by Albireo. Medical writing and editorial assistance were provided by Peloton Advantage, LLC, an OPEN Health company, and were funded by Albireo Pharma, Inc.

https://doi.org/10.1136/flgastro-2023-bspghan.8

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