“He Doesn’t Let It Stop Him”: Tanner’s Journey with Alagille Syndrome

To the casual observer, Tanner seems like most other 10-year-old kids. He loves spending time with his family, playing the trombone, and reading. He’s competitive, funny, and full of energy. If you met him today, you’d probably never guess the challenges he’s faced.

Tanner’s early life

When Tanner was six weeks old, his parents, Kristen and Jay, noticed that he became jaundiced (his whole body turned yellow). After testing, doctors at their local children’s hospital ordered a liver biopsy. Kristen recalls this time, saying, “Sending our seven-pound baby off for surgery was scary. And not knowing what was going on was the hardest part. We spent a month just waiting for an answer.”

When Kristen received the call that genetic tests confirmed a diagnosis of Alagille syndrome (ALGS), it was an overwhelming moment, setting them on the journey of being parents to an “Alagille warrior.” ALGS is a rare genetic condition that affects the liver and other organs, including the heart, eyes, and skeleton. ALGS makes it so that the body has difficulty absorbing certain fat-soluble vitamins, which are crucial to growing. For Tanner, this meant he spent years with a feeding tube to help him get the nutrients he needed to grow.

Intense itching

One of Tanner’s most challenging symptoms of ALGS has been pruritus, or constant itching. In the early years, Kristen describes the itching as, “intense and debilitating.” She recalls, “We’d read bedtime stories and then gently scratch his back until he fell asleep. Sometimes, it took an hour. Sometimes, it was longer. That was our normal for years.”

Jay recalls how the itching would leave Tanner with bloody ears and scars on his arms and face, and impact their whole family. “At home and at school, he’d just scratch. Our two older sons worried about him, and eventually came to understand when Tanner was having a bad day and just needed to be left alone. It was hard for them to see their little brother go through that.”

Treatment goals: controlling the itch

When Tanner was seven, his parents and doctor made the decision for him to join a clinical trial for a medication called Bylvay® (odevixbat). Bylvay is a prescription medicine for the treatment of cholestatic pruritus in patients 12 months of age and older with ALGS.

For Tanner, Bylvay has provided relief from his constant itching. Kristen describes the change she noticed in Tanner, saying, “All of a sudden, he wasn’t scratching anymore. It was like meeting a new version of our son.” Please see below for important safety information regarding Bylvay for ALGS.

Hope for future Alagille warriors

Today, at ten years old, Tanner’s world looks different than in the early days. He started playing the trombone, which he calls his favorite part of the school day, and leaned into the things he loves most: music, animals, and family time. “He’s the one who gets us all to play games together,” Kristen says. “He’s always rallying us.”

Jay adds, “He’s just a happy kid now. He takes his medication in the morning, feeds the cats, eats breakfast, and goes on with his day. It’s a part of his routine that makes a big difference.”

Reflecting back, Kristen is thankful for the progress being made. “When Tanner was first diagnosed, there wasn’t an approved treatment option, and we felt so alone. Today, we’re hopeful for the future for all families on the ALGS journey.” Kristen and Jay hope that sharing their family’s story helps others to feel less alone.

As for Tanner, he’s just enjoying each day as it comes. In the future, he has big goals of becoming a set designer for plays.

“When I look at what he’s overcome at just 10 years old, it blows me away,” Kristen says. “He doesn’t let Alagille syndrome stop him. And I hope he never does.”

Read the indication and important safety information below. Learn more about a treatment option for Alagille syndrome at Bylvay.com.

WHAT IS BYLVAY?
BYLVAY is a prescription medicine used for the treatment of itching in patients 12 months of age and older with Alagille syndrome (ALGS).

IMPORTANT SAFETY INFORMATION

You should not use BYLVAY if you have or have had 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 FSV deficiency. You may bleed more easily or may bleed longer. Call your healthcare provider for 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.
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.

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, Inc. at 1-855-463-5127.

Please see full Prescribing Information.

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