FAQs for Alagille syndrome | Bylvay® (odevixibat) - Patient site

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This site is intended for US residents only

Frequently asked questions about Bylvay and Alagille syndrome (ALGS)

What is Alagille syndrome?

Alagille syndrome (ALGS) is a rare genetic disease that causes damage to the liver and can affect the heart, eyes, skeleton, kidneys, face, and nervous system. The way Alagille syndrome affects the liver can cause itch (cholestatic pruritus), which can be severe in some patients.

What is cholestatic pruritus?

Doctors may describe the itch from Alagille syndrome using this term. Cholestatic (pronounced koh-luh-stat-ik) means the flow of bile from the liver is slowed or blocked. Pruritus (pronounced proo-rahy-tuhs) is a medical term for itching.

What is Bylvay?

Bylvay is a medicine to treat cholestatic pruritus (itch) in patients with Alagille syndrome as young as 12 months of age and in patients with progressive familial intrahepatic cholestasis (PFIC) as young as 3 months of age.

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

How is Bylvay thought to work?

Bylvay is a type of medication called an ileal bile acid transporter (IBAT) inhibitor. While the exact way Bylvay reduces itch isn’t fully understood, IBAT inhibition may reduce bile acid buildup as measured by bile acid in the blood. The buildup of bile acids in the liver and bloodstream may be the cause of itch (cholestatic pruritus) in Alagille syndrome.

What resources can help me access/afford Bylvay?

IPSEN CARES patient support program can help you get access to your Bylvay^® (odevixibat) prescription with the information and support you need.

To learn more about IPSEN CARES:

Monday-Friday, 8:00 AM – 8:00 PM ET

support@ipsencares.com

Uses

IMPORTANT SAFETY INFORMATION

BYLVAY is indicated for the treatment of itching 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 might not work for some PFIC type 2 patients who have specific ABCB11 gene changes. Talk with your healthcare provider if you have questions.

You should not use BYLVAY if you have had or have an advanced stage of liver disease. Talk with your healthcare provider if you have any liver conditions.
Abnormal liver tests have been observed with use of BYLVAY. Liver tests should be obtained before starting and periodically during therapy to monitor for liver injury. Immediately report to your healthcare provider any signs or symptoms of liver injury, such as nausea, vomiting, skin or the whites of eyes turn yellow, dark or brown urine, pain on the right side of the abdomen, or loss of appetite.
Notify your healthcare provider if you experience new onset or worsening of diarrhea.
BYLVAY may decrease the absorption of certain vitamins, which include vitamins A, D, E, and K (FSV or fat-soluble vitamins). Your blood levels of these vitamins should be measured before starting and periodically during treatment to assess for FSV deficiency. You may bleed more easily or may bleed longer. Call your healthcare provider if you have any signs or symptoms of bleeding.
The most common side effects of BYLVAY treatment in patients with ALGS are diarrhea, abdominal pain, abnormal bleeding, such as bruising, and decreased weight.
The most common side effects of BYLVAY in patients with PFIC are diarrhea, liver test abnormalities, vomiting, abdominal pain, and FSV deficiency.
Do not swallow the 200 mcg or 600 mcg capsule(s) containing Oral Pellets whole. These are intended to be opened and the contents mixed into soft food.
For patients taking bile acid binding resins, take BYLVAY at least 4 hours before or 4 hours after taking a bile acid binding resin.
There is a pregnancy safety study that collects outcome data in women taking BYLVAY during pregnancy. Pregnant women exposed to BYLVAY should report if they have used BYLVAY during pregnancy by calling 1-855-463-5127.

Uses

BYLVAY is indicated for the treatment of itching 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 might not work for some PFIC type 2 patients who have specific ABCB11 gene changes. Talk with your healthcare provider if you have questions.

You are encouraged to report side effects to FDA at 1-800-FDA-1088 or at www.fda.gov/medwatch. You may also report side effects to Ipsen Biopharmaceuticals at 1-855-463-5127.

Please see full Prescribing Information for BYLVAY.