This site is intended for U.S. residents only

Bylvay (odevixibat) 200, 400, 600, 1200 mcg capsules logo

Bylvay is FDA-approved for cholestatic pruritus in patients (≥12 months of age) with Alagille syndrome (ALGS)1

Explore the ALGS data from the ASSERT trial—part of the largest phase 3 clinical trial program in cholestatic pruritus of ALGS and PFIC to date

All images are actor portrayals.
Teenage boy with Alagille syndrome (ALGS) playing with a model airplane pushing through the Bylvay logo cutout.

Bylvay reduced cholestatic pruritus early and over time1-3

Change in Scratching Score: ASSERT 1 Through ASSERT 2 Week 24

Scratching was assessed in both ASSERT 1 and ASSERT 2. Chart shows a reduction for those on Bylvay as early as week 4.

Reductions in cholestatic pruritus were seen as early as week 4 and sustained through week 482,3

Primary endpoint: Change in cholestatic pruritus from baseline to month 6 (weeks 21-24). ASSERT 2 is an ongoing open-label extension trial. Data shown are interim data.

A girl with progressive familial intrahepatic cholestasis (PFIC) enjoying the beach in her orange dress, highlighted by the Bylvay logo outline.

Sleep parameters in ASSERT 14

Data shown here are secondary endpoints from ASSERT 1.4*

Decrease in disruptive sleep parameters at week 24 in patients taking Bylvay vs placebo (n=35, n=17)4†

  • 47% fewer days

    needing soothing
    from a caregiver

  • 43% fewer days

    needing help
    falling asleep

  • 35% fewer days

    needing to sleep
    with a caregiver

>1-point reduction in tiredness score from baseline at week 24 (-1.13 vs -0.53, Bylvay and placebo, respectively)4‡

Additional sleep parameters were number of awakenings, as well as days seeing blood due to scratching and days needing to take medication to induce sleep, which were not statistically significant.

*Limitation: Data are from a secondary endpoint that was not adjusted for multiplicity. Therefore, results should be interpreted with caution.

Reported data. Using the PRUCISION™ scale daily, caregivers answered yes or no to 7 questions about the effects that itch had on their child’s sleep and sleep-related parameters.4

† Mean improvements from baseline to weeks 21-24 were significant vs placebo in the percentages of days needing soothing from a caregiver (P<0.0001), needing help falling asleep (P=0.0016), needing to sleep with a caregiver (P=0.0017).

‡ Tiredness score measured on a 5-point scale from 0=Not tired at all; 1=A little tired; 2=Medium tired; 3=Very tired; 4=Very, very tired (P=0.0062).

sBA reductions with Bylvay over time1,2,5

Change in sBA: ASSERT 1 Through ASSERT 2 Week 24

Chart shows a reduction in sBA was shown as early as week 4 and was maintained through ASSERT 1 and ASSERT 2 week 24.
  • Reductions in sBA were seen as early as week 4 and sustained at week 241,2
  • Patients who received Bylvay from weeks 1 to 24 had further reductions in sBA by week 485

Secondary endpoint: Change in serum bile acid levels. ASSERT 2 is an ongoing open-label extension trial. Data shown are interim data.

Limitations:

The correlation between sBA and clinical outcomes is not known. sBA levels do not accurately predict bile acids in the liver.

The clinical trial was not designed to make conclusions regarding the clinical benefit of sBA reduction. It is unknown whether the sBA assay was qualified to assess sBA levels during the analysis.

Common adverse reactions (≥5%) reported in ASSERT 1 trial1

ASSERT 1: Clinical Adverse Reactions (≥5%)

Preferred Term Placebo
(N=17)
n (%) 		Bylvay
120 mcg/kg/day
(N=35)
n (%)
Diarrhea 1 (6) 10 (29)
Abdominal pain 1 (6) 5 (14)
Hematoma 0 3 (9)
Weight
decreased 		0 2 (6)
  • Most treatment-emergent adverse events were mild to moderate in severity4
  • All reports of diarrhea were grade 1 intensity (mild)4
  • Treatment-related diarrhea was reported in
  • 1 patient receiving Bylvay (duration: 4 days)4
  • 1 patient receiving placebo (duration: 24 days)4
  • In ASSERT 2, treatment-emergent adverse reactions were similar to those observed in ASSERT 1. The most common reason for Bylvay treatment interruption was gastrointestinal disorders, including diarrhea and abdominal pain1
See PFIC data

PFIC=progressive familial intrahepatic cholestasis; sBA=serum bile acid.

Young child with Alagille syndrome (ALGS) chasing after a volleyball on the beach.

References:
1. Bylvay Prescribing Information. Boston, MA: Albireo Pharma, Inc.; 2023. 2. Data on file A4250-012 Tables-Figures. 2022. Boston, MA: Albireo Pharma, Inc. 3. Data on file A4250-015 Pruritus Change. 2022. Boston, MA: Albireo Pharma, Inc. 4. Data on file A4250-012. November 10, 2022. Boston, MA: Albireo Pharma, Inc. 5. Data on file A4250-015 Table 14.2.2.1, sBA Change. Boston, MA: Albireo Pharma, Inc.

IMPORTANT SAFETY INFORMATION AND INDICATIONS

Warnings and Precautions:

Liver Test Abnormalities
Patients enrolled in PFIC and ALGS clinical trials had abnormal liver tests at baseline. In clinical trials, treatment-emergent elevations of liver tests or worsening of liver tests relative to baseline values were observed during the clinical trials.

In a clinical trial with PFIC patients, treatment-emergent elevations of liver tests or worsening of liver tests relative to baseline values were observed during the clinical trial. Most abnormalities included elevations in AST, ALT, or total and direct bilirubin. Treatment interruption days ranged from 3 days to 124 days; no PFIC patients permanently discontinued treatment due to liver test abnormalities.

In a clinical trial with ALGS patients, treatment-emergent elevations or worsening in liver tests relative to baseline values were observed during the trial. Most abnormalities included elevations in ALT or AST. One ALGS patient interrupted treatment for 40 days; no ALGS patients permanently discontinued treatment due to liver test abnormalities.

Obtain baseline liver tests and monitor during treatment. Dose reduction or treatment interruption may be required if abnormalities occur. For persistent or recurrent liver test abnormalities, consider treatment discontinuation.

Bylvay was not evaluated in PFIC or ALGS patients with cirrhosis. Closely monitor for liver test abnormalities; permanently discontinue Bylvay if a patient progresses to portal hypertension or experiences a hepatic decompensation event.

Diarrhea
In a PFIC clinical trial, diarrhea was reported in 2 (10%) placebo-treated patients, 9 (39%) Bylvay-treated 40 mcg/kg/day patients, and 4 (21%) Bylvay-treated 120 mcg/kg/day patients. Treatment interruption due to diarrhea occurred in 2 patients with 3 events during treatment with Bylvay 120 mcg/kg/day. Treatment interruption due to diarrhea ranged between 3 to 7 days. One patient treated with Bylvay 120 mcg/kg/day withdrew from the pivotal clinical trial due to persistent diarrhea.

In an ALGS clinical trial, diarrhea in ALGS patients was reported in 1 (6%) placebo-treated patient and in 10 (29%) Bylvay-treated patients. No patients interrupted or permanently discontinued Bylvay due to diarrhea.

If diarrhea occurs, monitor for dehydration and treat promptly. Interrupt Bylvay dosing if a patient experiences persistent diarrhea. Restart Bylvay at 40 mcg/kg/day when diarrhea resolves and increase the dose as tolerated if appropriate. If diarrhea persists and no alternate etiology is identified, stop Bylvay treatment.

Fat-Soluble Vitamin (FSV) Deficiency
Fat-soluble vitamins (FSV) include vitamin A, D, E, and K (measured using INR levels). PFIC patients can have FSV deficiency at baseline. Bylvay may affect absorption of fat-soluble vitamins. In a clinical trial, new onset or worsening of existing FSV deficiency was reported in 1 (5%) placebo-treated patient and 3 (16%) Bylvay-treated 120 mcg/kg/day patients; none of the Bylvay-treated 40 mcg/kg/day patients had new onset or worsening of existing FSV deficiency. In an ALGS clinical trial, new or worsening of existing FSV deficiency was reported in 3 (17.6%) placebo-treated patients and 3 (8.6%) Bylvay-treated patients.

Obtain serum FSV levels at baseline and monitor during treatment, along with any clinical manifestations. If FSV deficiency is diagnosed, supplement with FSV. Discontinue Bylvay if FSV deficiency persists or worsens despite adequate FSV supplementation.

Adverse Reactions
The most common adverse reactions for Bylvay in patients with PFIC are diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency.

The most common adverse reactions for Bylvay patients with ALGS are diarrhea, abdominal pain, hematoma, and decreased weight.

Drug Interactions
For patients taking bile acid binding resins, take Bylvay at least 4 hours before or 4 hours after taking a bile acid binding resin.

Use in Specific Populations
There are no human data on Bylvay use in pregnant persons 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. There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to Bylvay during pregnancy. For more information, please call 1-855-252-4736.

Indications and Usage

Bylvay is an ileal bile acid transporter (IBAT) inhibitor indicated for the treatment of cholestatic pruritus in:

  • Patients 12 months of age and older with Alagille syndrome (ALGS)
  • Patients 3 months of age and older with progressive familial intrahepatic cholestasis (PFIC)
  • Limitation of Use:
    Bylvay may not be effective 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

Please see full Prescribing Information.

IMPORTANT SAFETY INFORMATION AND INDICATIONS

Warnings and Precautions:

Liver Test Abnormalities
Patients enrolled in PFIC and ALGS clinical trials had abnormal liver tests at baseline. In clinical trials, treatment-emergent elevations of liver tests or worsening of liver tests relative to baseline values were observed during the clinical trials.

In a clinical trial with PFIC patients, treatment-emergent elevations of liver tests or worsening of liver tests relative to baseline values were observed during the clinical trial. Most abnormalities included elevations in AST, ALT, or total and direct bilirubin. Treatment interruption days ranged from 3 days to 124 days; no PFIC patients permanently discontinued treatment due to liver test abnormalities.

In a clinical trial with ALGS patients, treatment-emergent elevations or worsening in liver tests relative to baseline values were observed during the trial. Most abnormalities included elevations in ALT or AST. One ALGS patient interrupted treatment for 40 days; no ALGS patients permanently discontinued treatment due to liver test abnormalities.

Obtain baseline liver tests and monitor during treatment. Dose reduction or treatment interruption may be required if abnormalities occur. For persistent or recurrent liver test abnormalities, consider treatment discontinuation.

Bylvay was not evaluated in PFIC or ALGS patients with cirrhosis. Closely monitor for liver test abnormalities; permanently discontinue Bylvay if a patient progresses to portal hypertension or experiences a hepatic decompensation event.

Diarrhea
In a PFIC clinical trial, diarrhea was reported in 2 (10%) placebo-treated patients, 9 (39%) Bylvay-treated 40 mcg/kg/day patients, and 4 (21%) Bylvay-treated 120 mcg/kg/day patients. Treatment interruption due to diarrhea occurred in 2 patients with 3 events during treatment with Bylvay 120 mcg/kg/day. Treatment interruption due to diarrhea ranged between 3 to 7 days. One patient treated with Bylvay 120 mcg/kg/day withdrew from the pivotal clinical trial due to persistent diarrhea.

In an ALGS clinical trial, diarrhea in ALGS patients was reported in 1 (6%) placebo-treated patient and in 10 (29%) Bylvay-treated patients. No patients interrupted or permanently discontinued Bylvay due to diarrhea.

If diarrhea occurs, monitor for dehydration and treat promptly. Interrupt Bylvay dosing if a patient experiences persistent diarrhea. Restart Bylvay at 40 mcg/kg/day when diarrhea resolves and increase the dose as tolerated if appropriate. If diarrhea persists and no alternate etiology is identified, stop Bylvay treatment.

Fat-Soluble Vitamin (FSV) Deficiency
Fat-soluble vitamins (FSV) include vitamin A, D, E, and K (measured using INR levels). PFIC patients can have FSV deficiency at baseline. Bylvay may affect absorption of fat-soluble vitamins. In a clinical trial, new onset or worsening of existing FSV deficiency was reported in 1 (5%) placebo-treated patient and 3 (16%) Bylvay-treated 120 mcg/kg/day patients; none of the Bylvay-treated 40 mcg/kg/day patients had new onset or worsening of existing FSV deficiency. In an ALGS clinical trial, new or worsening of existing FSV deficiency was reported in 3 (17.6%) placebo-treated patients and 3 (8.6%) Bylvay-treated patients.

Obtain serum FSV levels at baseline and monitor during treatment, along with any clinical manifestations. If FSV deficiency is diagnosed, supplement with FSV. Discontinue Bylvay if FSV deficiency persists or worsens despite adequate FSV supplementation.

Adverse Reactions
The most common adverse reactions for Bylvay in patients with PFIC are diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency.

The most common adverse reactions for Bylvay patients with ALGS are diarrhea, abdominal pain, hematoma, and decreased weight.

Drug Interactions
For patients taking bile acid binding resins, take Bylvay at least 4 hours before or 4 hours after taking a bile acid binding resin.

Use in Specific Populations
There are no human data on Bylvay use in pregnant persons 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. There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to Bylvay during pregnancy. For more information, please call 1-855-252-4736.

Indications and Usage

Bylvay is an ileal bile acid transporter (IBAT) inhibitor indicated for the treatment of cholestatic pruritus in:

  • Patients 12 months of age and older with Alagille syndrome (ALGS)
  • Patients 3 months of age and older with progressive familial intrahepatic cholestasis (PFIC)
  • Limitation of Use:
    Bylvay may not be effective 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

Please see full Prescribing Information.