Acknowledgment: This patient story is sponsored by Ipsen Biopharmaceuticals, Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted, and valuable information to patient communities. If you would like to learn more about a treatment option for PFIC, consider visiting this website for more information.

This is the unique experience of Victor and not representative of all people living with PFIC. The information provided is not intended to be a substitute for professional medical advice, diagnosis, or treatment. For questions about your health or the health of a loved one, please talk to your doctor.

Personally, Victor is a dad, husband, brother, son and friend to many, and professionally, he cares deeply about his community and helping others through his career in public service.

The road to today – 36-year-old Victor – has been shaped by many factors, including when Victor learned as a teenager that he was living with a progressive liver disease called progressive familial intrahepatic cholestasis (PFIC).

PFIC is one of several rare cholestatic liver diseases. In cholestatic liver disease, the flow of bile from the liver is slowed or blocked. Bile is a yellow fluid created in the liver to help break down food to release the nutrients inside. There are several types of PFIC, and symptoms can vary based on type. Signs and symptoms of PFIC include yellowing of the skin, poor weight gain and growth, gallstones, diarrhea and severe itching, known as cholestatic pruritus. PFIC is often diagnosed in infancy but can be diagnosed at any time.

Living with persistent itch

“It is hard to describe, but the itching I experienced was so intense. No matter how hard or often I scratched – I couldn’t find any relief despite having used hairbrushes and other miscellaneous items that made me tear my skin off,” shared Victor.

For Victor, severe itching is what triggered him to seek help and ultimately receive a PFIC diagnosis. When telling his pediatrician about the itching, the pediatrician also noticed his liver enzymes were outside of normal ranges and his liver function was abnormal. Victor was admitted to the hospital and following a liver biopsy and other testing, he was diagnosed with PFIC.

With a diagnosis, he became aware of how PFIC had impacted him and as a highschooler, he had yellowing in his eyes and skin, known as jaundice.

“As a teenager, the symptoms impacted me physically, socially, and academically and I isolated myself.”

While the complex nature of itching is not completely understood, one aspect that may cause itch is the buildup of bile acids in the liver and the bloodstream.

“Sometimes I would scratch hard enough to tear my skin, but it’s nothing compared to being kept up at night scratching something for hours without any relief. For 18 years following my diagnosis, I had to adjust, which became much more demanding as I grew up. I tried many things to find relief over the years, but nothing worked. I was restless.”

Treating the itch from PFIC

Three years ago, Victor’s hepatologist, a doctor who specializes in liver health, prescribed Bylvay® (odevixibat) for the treatment of itch. Bylvay is a prescription medicine that is approved to treat itch in people 3 months or older living with progressive familial intrahepatic cholestasis, known as PFIC. 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. Common side effects of Bylvay in patients with PFIC are diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency.

“I had tried many treatments, none of which were effective for me, so I was cautiously optimistic and ready to learn more.”

For Victor, finding a treatment that could help reduce the severe itching was important.

“With Bylvay, I experience less itching throughout the day and night. I feel I can focus more on other aspects of my life, like spending more time with my daughter. I have found that Bylvay is the right medicine for me.”

Although treatment options were not approved for PFIC when Victor was diagnosed, he is thankful for the progress being made for the PFIC community and that he has found a treatment that continues to be the right choice for him. Today, Victor finds himself able to dedicate more of his time on his career, fitness and family – especially his young daughter.

Read the Indication and Important Safety Information here. Access full Prescribing Information and learn more about Bylvay at Bylvay.com.

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

IMPORTANT SAFETY INFORMATION

• Speak with your healthcare provider if you experience abdominal pain, vomiting, diarrhea, hematoma, decreased weight, or dehydration as these have been reported with the use of Bylvay. Patients should contact their healthcare provider if they experience new onset or worsening of diarrhea
• Elevations in liver tests (for example, AST, ALT, TB) have been observed with use of Bylvay. The patient’s healthcare provider will obtain liver tests before starting Bylvay and periodically during treatment with Bylvay. Patients should report to their healthcare provider any symptoms of liver problems (for example, nausea, vomiting, skin or the whites of eyes turn yellow, dark or brown urine, pain on the right side of the abdomen, loss of appetite)
• Bylvay may impair absorption of fat-soluble vitamins (FSV), which include vitamins A, D, E and K (vitamin K is assessed by measuring INR). The patient’s healthcare provider will obtain serum levels of vitamins A, D, E, and INR (for vitamin K) at baseline and periodically during treatment to assess for worsening of 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. Take Bylvay in the morning with a meal
• 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 exposure registry that monitors pregnancy outcomes in women exposed to Bylvay during pregnancy. For more information, please call 1-855-252-4736

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.

Please click here for full Prescribing Information for Bylvay.

Bylvay is a registered trademark of Albireo Pharma, Inc., an Ipsen company.

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