Odevixibat

Odevixibat is a reversible inhibitor of the ileal bile acid transporter (IBAT). It decreases the reabsorption of bile acids (primarily the salt forms) from the terminal ileum .

• BYLVAY is an ileal bile acid transporter (IBAT) inhibitor indicated for: Progressive Familial Intrahepatic Cholestasis (PFIC) the treatment of pruritus in patients 3 months of age and older with progressive familial intrahepatic cholestasis (PFIC). ( 1.1 ) Limitation of Use : BYLVAY is not recommended in a subgroup of PFIC type 2 patients with specific ABCB11 variants resulting in non-functional or complete absence of the bile salt export pump protein.

• IBAT inhibitors, including BYLVAY, are contraindicated in patients with prior or active hepatic decompensation events (e.g., variceal hemorrhage, ascites, hepatic encephalopathy) [see Warnings and Precautions (5.1) ] . Patients with prior or active hepatic decompensation events (e.g., variceal hemorrhage, ascites, hepatic encephalopathy).contraindicated

Recommended Dosage PFIC: Patients 3 months and older: 40 mcg/kg taken orally once daily. ( 2.1 ) If there is no improvement in pruritus after 3 months, the dosage may be increased in 40 mcg/kg increments up to 120 mcg/kg once daily, not to exceed a daily dosage of 6 mg/day. ( 2.1 ) ALGS: Patients 12 months and older: 120 mcg/kg taken orally once daily. ( 2.2 ) Preparation and Administration Instructions Administer BYLVAY in the morning with a meal. ( 2.4 ) Do not crush or chew capsules. ( 2.4 ) See full prescribing information for preparation and administration instructions. ( 2.4 ) 2.1 Recommended Dosage for Progressive Familial Intrahepatic Cholestasis (PFIC) in Patients Aged 3 Months and Older The recommended dosage of BYLVAY is 40 mcg/kg taken orally once daily in the morning with a meal. Table 1 below shows the recommended once daily dosage by body weight. If there is no improvement in pruritus after 3 months, the dosage may be increased in 40 mcg/kg increments up to 120 mcg/kg once daily not to exceed a daily dosage of 6 mg/day. BYLVAY oral pellets are intended for use by patients weighing less than 19.5 kilograms. BYLVAY capsules are intended for use by patients weighing 19.5 kilograms or above. Table 1.

Hepatoxicity : Obtain baseline liver tests and monitor patients frequently for the first 6 to 8 months after starting therapy, and as clinically indicated thereafter during treatment. If liver test abnormalities or signs of clinical hepatitis occur, consider dose reduction or treatment interruption. For persistent or recurrent liver test abnormalities relative to baseline, discontinue BYLVAY. Monitor patients with compensated cirrhosis or portal hypertension more frequently. Permanently discontinue BYLVAY if hepatic decompensation occurs. ( 2.3 , 5.1 ) Diarrhea : Treat dehydration. Treatment interruption or discontinuation may be required for persistent diarrhea. ( 5.2 ) Fat-Soluble Vitamin (FSV) Deficiency : Obtain baseline levels and monitor during treatment. Supplement with FSV if deficiency is observed. If FSV deficiency persists or worsens despite FSV supplementation, consider discontinuing BYLVAY treatment. Fracture: Consider interrupting BYLVAY treatment. Supplement with FSV if indicated. Bleeding: Interrupt treatment with BYLVAY. Optimize treatment of FSV deficiency and consider restarting BYLVAY once the patient is clinically stable. ( 5.3 ) 5.1 Hepatoxicity BYLVAY treatment is associated with a potential for drug-induced liver injury (DILI). In the PFIC and ALGS trials, treatment-emergent elevations of liver tests or worsening of liver tests occurred. Of the six patients who experienced DILI, two underwent liver transplant. Obtain baseline liver tests because some ALGS and PFIC patients have abnormal liver tests at baseline.

The following adverse reactions are discussed in greater detail in other sections of the label: Hepatotoxicity [ see Warnings and Precautions (5.1) ] Diarrhea [see Warnings and Precautions (5.2) ] Fat-Soluble Vitamin Deficiency [see Warnings and Precautions (5.3) ] PFIC: Most common adverse reactions (>2%) are liver test abnormalities, diarrhea, abdominal pain, vomiting, and fat-soluble vitamin deficiency. ( 6.1 ) ALGS: Most common adverse reactions (>5%) are diarrhea, abdominal pain, hematoma, and decreased weight. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Ipsen Biopharmaceuticals, Inc. at 1-855-463-5127, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. PFIC Clinical Studies Trial 1 is a randomized, double-blind, placebo-controlled, 24-week study of two dose levels of BYLVAY (40 mcg/kg and 120 mcg/kg) administered once daily [see Clinical Studies (14.1) ] . Sixty-two patients were randomized (1:1:1) to receive one of the following: BYLVAY 40 mcg/kg/day (n=23), BYLVAY 120 mcg/kg/day (n=19), or Placebo (n=20). Table 3 summarizes the frequency of adverse reactions reported in ≥2% and at a rate greater than placebo in patients treated with BYLVAY in Trial 1.

Pregnancy: Based on animal data, may cause cardiac malformations ( 8.1 ) 8.1 Pregnancy Risk Summary Limited human data on the use of BYLVAY in pregnant women are insufficient to establish a drug-associated risk of major birth defects, miscarriage, or adverse developmental outcomes. Based on findings from animal reproduction studies, BYLVAY may cause cardiac malformations when a fetus is exposed during pregnancy. In pregnant rabbits treated orally with odevixibat during organogenesis, an increased incidence of malformations in fetal heart, great blood vessels, and other vascular sites occurred at all doses; maternal systemic exposure at the lowest dose was 2.1 times the maximum recommended dose (see Data ) . Odevixibat may inhibit the absorption of fat-soluble vitamins. FSV are essential for normal fetal growth and development. Monitor pregnant patients for FSV deficiency and supplement as needed. Increased supplementation of FSVs may be needed during pregnancy [see Warnings and Precautions (5.3) and Clinical Considerations ] . Consider the woman's need for BYLVAY, the potential drug-related risks to the fetus, and the potential adverse outcomes from untreated maternal PFIC and ALGS. The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. In the U.S.

Metabolism

In pediatric patients with PFIC, 6 months to 17 years of age who received BYLVAY 40 mcg/kg or 120 mcg/kg once daily with food in the morning, the measurable odevixibat concentrations ranged from 0.06 to 0.72 ng/mL, and odevixibat concentrations were below the limit of quantification (0.05 ng/mL) in the majority of plasma samples. In pediatric patients with ALGS who received BYLVAY 120 mcg/kg once daily with food in the morning, the measurable odevixibat concentrations ranged from 0.05 to 3.4 ng/mL.

• Jul 20, 2021NDANDA215498Ipsen

1. 1Diarrhoea7725%
2. 2Pruritus7725%
3. 3Off Label Use5016%
4. 4Drug Ineffective3311%
5. 5Liver Transplant247.8%
6. 6Product Dose Omission Issue216.8%
7. 7Abdominal Pain206.5%
8. 8Abdominal Pain Upper196.2%
9. 9Blood Bilirubin Increased175.5%
10. 10Hepatic Enzyme Increased165.2%
11. 11Vomiting154.9%
12. 12Bile Acids Increased144.6%
13. 13Alanine Aminotransferase Increased134.2%
14. 14Nausea134.2%
15. 15Aspartate Aminotransferase Increased113.6%

The 10 most recently updated of 13 ClinicalTrials.gov registrations naming Odevixibat as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Odevixibat Outcomes in Patients With PFIC Versus an External Control Cohort (OvEC-PFIC)
  Active not recruiting · Observational · 200 enrolled · Ipsen
  NCT07497724updated 2026-05-28
• A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in South Korea
  Recruiting · Observational · 10 enrolled · Ipsen
  NCT07185919updated 2026-05-28
• A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment
  Recruiting · Observational · 30 enrolled · Ipsen
  NCT06850038updated 2026-05-28
• Odevixibat Pregnancy and Lactation Surveillance Program: A Study to Evaluate the Safety of Odevixibat During Pregnancy and/or Lactation
  Recruiting · Observational · 20 enrolled · Ipsen
  NCT06258902updated 2026-05-28
• Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome
  Recruiting · Phase 3 · Interventional · 70 enrolled · Albireo, an Ipsen Company
  NCT05035030updated 2026-05-28
• Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)
  Active not recruiting · Phase 3 · Interventional · 254 enrolled · Albireo, an Ipsen Company
  NCT04336722updated 2026-05-28
• An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia
  Enrolling by invitation · Phase 3 · Interventional · 180 enrolled · Albireo, an Ipsen Company
  NCT05426733updated 2026-05-28
• A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in China
  Recruiting · Observational · 20 enrolled · Ipsen
  NCT07588880updated 2026-05-15
• A Study to Observe the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment
  Not yet recruiting · Observational · 30 enrolled · Ipsen
  NCT07585097updated 2026-05-13
• Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC
  Completed · Phase 3 · Interventional · 116 enrolled · Albireo, an Ipsen Company
  NCT03659916updated 2026-04-30

Frequently asked questions

How does Odevixibat work?

Odevixibat is a reversible inhibitor of the ileal bile acid transporter (IBAT). It decreases the reabsorption of bile acids (primarily the salt forms) from the terminal ileum .

What is Odevixibat used for?

According to FDA labeling, Odevixibat carries indications including: BYLVAY is an ileal bile acid transporter (IBAT) inhibitor indicated for: Progressive Familial Intrahepatic Cholestasis (PFIC) the treatment of pruritus in patients 3 months of age and older with progressive familial intrahepatic cholestasis (PFIC). ( 1.1 ) Limitation of Use : BYLVAY is not recommended in a subgroup of PFIC type 2 patients with specific ABCB11 variants resulting in non-functional or complete absence of the bile salt export pump protein.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Odevixibat?

Odevixibat is classified as Other drugs for bile therapy, Ileal Bile Acid Transporter Inhibitor, Ileal Bile Acid Transporter Inhibitors, Bile Secretion Alteration.

What are the brand names for Odevixibat?

Odevixibat is marketed under brand names including Bylvay.

What are the contraindications for Odevixibat?

Odevixibat labeling lists contraindications including: IBAT inhibitors, including BYLVAY, are contraindicated in patients with prior or active hepatic decompensation events (e.g., variceal hemorrhage, ascites, hepatic encephalopathy) [see Warnings and Precautions (5.1) ] . Patients with prior or active hepatic decompensation events (e.g., variceal hemorrhage, ascites, hepatic encephalopathy).. Always consult the full prescribing information and a clinician.