Phase 3 Odevixibat Clinical Trial Meets Endpoints for PFIC

Recently, biopharmaceutical company Albireo Pharma announced positive results from a Phase 3 clinical trial. The trial, PEDFIC 1, studied the safety, efficacy, and tolerability of odevixibat for patients with progressive familial intrahepatic cholestasis (PFIC) types 1 and 2. Ultimately, the trial was effective. Overall, odevixibat hit both endpoints: reduced serum bile acid responses (sBAs) and lowered itching.


Odevixibat is an investigational ileal bile acid transport inhibitor (IBATi) designed to treat patients with rare pediatric cholestatic liver diseases such as biliary atresia and Alagille syndrome. In fact, Odevixibat already received Orphan Drug designation and inclusion into the PRIME scheme for these conditions. According to Albireo:

After completing digestion, bile acids are reclaimed in the distal part of the small intestine, known as the terminal ileum, by ileal bile acid transporters (IBAT).

However, in patients with PFIC, these bile transporters are defective. Odevixibat works by interrupting bile circulation and helping more bile acids enter the colon. Unlike other therapies, it does not require refrigeration and acts locally, making it a potent and targeted solution.

The Research

The PEDFIC 1 trial included 62 patients ranging in age from 6 months to 15.9 years old. Patients had either PFIC 1 or PFIC 2. During the trial, participants received either 40mg/kg odevixibat or 120 mg/kg odevixibat daily, or a placebo, for 5.5 months. Participants from Cohort 1 are allowed to continue treatment in PEDFIC 2.

Ultimately, the trial was designed to evaluate the safety, tolerability, and efficacy of odevixibat. The endpoints included reducing pruritus (chronic, intense itching) and reducing serum bile acid responses. 53.5% of patients taking odevixibat experienced reduced itching, compared to only 28.7% of those receiving placebos. Additionally, about 1/3 of patients experienced reduced sBAs. The therapy is fairly well-tolerated, with the largest side effect being diarrhea.

If approved, odevixibat would be the first FDA-approved treatment for patients with PFIC. Looking forward, Albireo hopes to submit regulatory filings in 2021.

Progressive Familial Intrahepatic Cholestasis (PFIC)

Progressive familial intrahepatic cholestasis (PFIC) refers to a group of liver disorders that cause impaired bile flow. Resulting from genetic mutations, PFIC is characterized by biliary epithelial transporter defects. When bile accumulates in liver cells, patients experience disruptive symptoms and, more concerningly, liver disease. In most cases, PFIC occurs in children younger than 2 years old. However, in more rare cases, symptom onset occurs in adolescence. Nearly all patients require treatment before age 30. Generally, PFIC leads to liver cirrhosis (scarring) or failure within 10 years.

Symptoms include:

  • Failure to thrive
  • Jaundice (yellowing of the skin)
  • Cholestasis (bile obstruction)
  • Intense itching
  • Vitamin deficiencies
  • Problems with vision
  • Balance issues
  • Spleen or liver enlargement
  • Broken bones
  • Suicidal ideation

Learn more about PFIC.

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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