Bylvay was approved for marketing by the US Food and Drug Administration (FDA) in July 2021, becoming the first targeted drug for the treatment of progressive familial intrahepatic cholestasis (PFIC).

1、 Drug name

1. Common name: Odevixibat

2. Product Name: BYLVAY

2、 Indications

Used to treat itching symptoms in patients with progressive familial intrahepatic cholestasis (PFIC) aged 3 months and above. If there is a mutation in the ABCB11 gene that leads to complete loss or non function of bile salt efflux pump protein (BSEP-3) in PFIC2 patients, it may be ineffective.

3、 Specifications and characteristics

1. Capsules: 400mcg

2. Main ingredients: Ovexibat sesquihydrate, excipients include hydroxypropyl methylcellulose, microcrystalline cellulose, etc.

4、 Usage and dosage

1. Recommended dosage: 40mcg/kg/day, taken with meals in the morning. If itching does not improve after 3 months, it can be adjusted in increments of 40mcg/kg to a maximum of 120mcg/kg/day (total daily dose not exceeding 6mg).

2. Example of segmented dosage for body weight: Patients weighing 7.5-12.4kg should receive 400mcg per day, while patients weighing 35.5-45.4kg should receive 1600mcg per day.

5、 Dose adjustment

1. When liver enzyme abnormalities (ALT/AST elevation, bilirubin elevation) occur, medication should be interrupted. After recovery, it can be restarted from 40mcg/kg and gradually increased.

2. Continuous diarrhea requires medication to be suspended, and restarted at the lowest dose after symptoms have improved.

3. Fat soluble vitamin deficiency needs to be supplemented, and if it continues to worsen, the medication should be discontinued.

6、 Medication precautions

1. Medication time: It must be taken with meals in the morning and cannot be taken on an empty stomach.

2. Omission handling: Not clearly stated, it is recommended to take the next normal dose.

3. Vomiting treatment: If vomiting occurs after taking medication, it is not recommended to take it again. Wait until the next normal administration.

4. Other:

(1) Capsules should not be chewed or crushed, and those who have difficulty swallowing can open them and mix them with soft food;

(2) Resins that bind with bile acids (such as colexamide) should be taken every 4 hours.

7、 Medication for special populations

1. Pregnant women: Animal experiments have shown that it may cause fetal heart malformations, and human data is insufficient, so the risks need to be balanced.

2. Breastfeeding period: Low drug absorption rate and low risk of breastfeeding, but it is necessary to monitor the levels of fat soluble vitamins in infants.

3. Children: Patients aged 3 months to 17 years old are safe and effective, with insufficient data for patients under 3 months old.

4. Liver injury: The safety of patients with cirrhosis or portal hypertension has not been established and requires close monitoring.

8、 Adverse reactions

1. Common (>2%): Abnormal liver enzymes, diarrhea, abdominal pain, vomiting, and deficiency of fat soluble vitamins.

2. Serious risks: hepatotoxicity (requiring regular monitoring of liver function), dehydration (requiring fluid replacement in case of diarrhea).

9、 Contraindications

There are no clear contraindications.

10、 Drug interactions

1. Bile acid binding resin may reduce efficacy and should be taken every 4 hours.

2. The combination with itraconazole (P-gp inhibitor) may increase the exposure of ovixibat, but its clinical significance is not significant.

11、 Storage method

Store at room temperature of 20 ° C-25 ° C, and allow short-term exposure to an environment of 15 ° C-30 ° C.

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