PEDFIC 1 Data Shows Odevixibat Benefits for PFIC

During the Association for the Study of Liver Diseases (AASLD) Liver Meeting, which took place from November 13 to 16, biopharmaceutical company Albireo Pharma (“Albireo) announced data from the Phase 3 PEDFIC 1 clinical trial. During the trial, researchers evaluated the safety, efficacy, and tolerability of odevixibat for patients with progressive familial intrahepatic cholestasis (PFIC). In addition to reducing serum bile acids (sBAs) and itching, odevixibat also helped improve liver function and patient outcomes.


Developed to treat a variety of rare liver diseases in pediatric patients, odevixibat is an investigational non-systemic ideal bile acid transport inhibitor (IBATi). Due to its lack of refrigeration, this drug offers an easier and more accessible option for many patients. Outside of being studied for PFIC, odevixibat is also currently being studied in 2 clinical trials for biliary atresia. It has received Orphan Drug, Rare Pediatric Disease, and Fast Track designations for the treatment of PFIC. However, in both Europe and America, it also received Orphan Drug designation to odevixibat for the treatment of biliary atresia, primary biliary cholangitis, and Alagille syndrome.

In the AASLD meeting, researchers from Albireo presented data from the Phase 3 PEDFIC 1 trial, as well as the PEDFIC 2 extension study. Throughout the two, researchers discovered:

  • Odevixibat was relatively safe and well-tolerated. While some side effects did occur, the most common side effect was diarrhea, which was not considered severe.
  • Both 40 and 120 µg/kg/day odevixibat reduced sBAs and itching when compared to a placebo. The treatment worked quickly to remove symptoms. Researchers noticed sustained symptom reduction over a 24 week (5.5 month) period.
  • Continued sBA and itching reductions continued throughout the extension period, which went up to 48 weeks (11 months).
  • Odevixibat helped patients combat failure to grow, showing an improvement in both height and weight.
  • 93% of patients enrolled in the trial continued odevixibat treatment.
  • Even patients with different subsets of PFIC, such as PFIC 1 or 3, all experienced improvements thanks to this therapy.

Ultimately, Albireo presented this data in late-breakers at the AASLD TLMdX under abstract numbers #LO4 and #LP19.

Progressive Familial Intrahepatic Cholestasis (PFIC)

Progressive familial intrahepatic cholestasis is a rare liver condition caused by biliary epithelial transporter defects. Gene mutations cause PFIC to develop. Normally, biliary epithelial transporters help regulate and transport bile out of the liver. However, when damaged or defective, bile accumulates in the liver. Ultimately, this causes issues with liver function, cirrhosis, and liver disease. Often, PFIC leads to liver failure within 10 years. Typically, PFIC affects children, with the onset occurring before age 2 on average; many patients require treatment prior to 30 years old. Symptoms include:

  • Intense itching
  • Cholestasis — bile obstruction
  • Jaundice (yellowing of the skin and eyes)
  • Failure to thrive
  • Malnutrition and vitamin deficiencies
  • Vision loss
  • Broken bones
  • Poor balance
  • Suicidal ideation
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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