[00:00:00]
[music]
[Téa playing]
Téa: My name is Téa. This is my dad, [00:00:30] Armin. This is my mom, Michelle. This is my grandpa, Peter, and this is my dog, Lulu. I have a sister named Isla. She lives in Arizona, a long way from me. She's a very good sister, and I love her to the moon and back. This is Peppermint, and this is Kiwi. That's [00:01:00] my family.
I was born with Alagille syndrome, and it hurts, and it's painful, and your body tells you to itch, and I'm trying to stop.
Michelle: When I first found out that Téa had Alagille syndrome, [00:01:30] my heart broke, and I was lost. I felt like everything that I've always wanted, not that it was taken away from me, but that joy of a newborn and having a baby in your arms. It was so hard. It was hard on us. It was hard on our marriage.
Armin: I think I was in denial, and I still might be. My first instinct was, [00:02:00] why? We didn't know what to do. We were just lost. The girl that you always want, seeing her going through something that no kid should go through, so tiny, so small, was hard.
Michelle: When we heard about her condition, they were saying, [00:02:30] there can definitely be lots of itching. She was 3 months old when she was diagnosed. She couldn't tell me that she was itching, so I really had to look for the signs. There was lots of itching and wiggling around. The physicians described it to me as having internal itching. It's almost like having poison ivy inside your body. For her, it was a lot of reaching for the ears, lots of scabs, lots of bleeding. Same with the head. She would take both of her hands and [00:03:00] just scratch.
Armin: She couldn't sleep. She would scratch all night. You, as a parent, don't know what to do, how to help them. We would get up and just scratch her until she goes to sleep.
Peter: My daughter, she go to work. She bring the baby by us. We have a crib for her, and we put them in a bed. Somehow, she laying down and her hands go all the way around in the [00:03:30] body. More time go in the legs, back, all over.
Téa: At nighttime, the itching was the worst on my ears, my legs, and my toes. That was the worst part of the night. In the morning, I would wake up and I would see that there's blood all over my sheets. I said to myself, "Why am I the only one who has this?" [00:04:00] At school, it was hard. During PE class, I think that was the worst time of the day that I itched. Some kids at school were not being very nice and I did not like it. I would say to the kids who were not being nice to me, I would say, "You should understand that I have Alagille syndrome and now I'm itching and I can't help it." [00:04:30] I don't know what to do.
Michelle: I categorized her itch as mild. I think because I just made it so—it was just so normal, it was just a part of our life at that point, that I regret it, because it wasn't normal. It was affecting her daily life. There was an opportunity for us to get into a trial with LIVMARLI. [00:05:00]. Téa wasn't able to access the trial because I categorized her as mild. Hearing other people's stories on their itching, I was like, "Okay, well, she's not doing that. It's not that bad," or, "If it's cool in the home, she's not itching that bad." I always found something to be like, "Okay, it's not that bad," but in reality, it was. [00:05:30]
[music]
We first learned about LIVMARLI through the clinical trials. We knew that there was something in the works for itching, which we were really excited about. Téa started LIVMARLI in February of 2022 [00:06:00] when she was 8 years old. After about 14 days on LIVMARLI, we noticed a drastic change in her itching. Her first night, we knew that LIVMARLI was working for her. In the middle of the night, I didn't hear her itching. She was snoring. It was so funny to me. Also, very sad to know that it took [00:06:30] eight years for her to sleep all night without sitting up and itching in the middle of the night. In the morning, she ran into my room, and she's like, "Mama, I slept so good."
Armin: That night was definitely one of the best nights of our life. Just hearing her snore and sleep and not [00:07:00] itch.
Michelle: When she gets home from school, she's not running to take her socks off and itch. She is itching less and sleeping through the night, which is amazing for her. She doesn't have the blood on the sheets like we did before. She's rested. She's happy.
Peter: Now that Téa takes LIVMARLI, I feel so happy helping her. She's not scratching. She sleeps good. [00:07:30] She's full of energy.
Téa: LIVMARLI helps me sleep better because I itch less. I'm not scratching and it makes me feel better. I'm excited that I can do more things.
Armin: Tell me when it's sauce time.
Michelle: When Téa was prescribed LIVMARLI, I received a call from Mirum Access Plus. It was just like a rundown of [00:08:00] the medication, potential side effects, how delivery was going to work, if I had any questions or concerns, who to call. Quite frankly, it was probably the easiest start of a new medication I think I've ever been through. We received our first dose of LIVMARLI 2 weeks after I spoke to Mirum Access Plus. It was approved through insurance. We received front door service of our prescription. [00:08:30]
Our doctor did mention that there were some side effects with LIVMARLI. Téa did experience some cramping, stomach cramping, but those have now subsided. It's better to talk about it than to hide from it. It's good for her, it's good for us, and it's good for our family. [00:09:00] We were never ashamed. But it was almost like you're almost in denial. There's more people out there like us who are just like, "We don't want to talk about it, let's just keep going."
[Téa playing]
[laughter]
Peter: Come on.
Michelle: I definitely feel like the Alagille Syndrome Alliance is such a great—there's so much amazing information. Roberta is very communicative on the [00:09:30] Alliance, and it's just a great resource. It's amazing to see how many kids there really are, and you're not the only one that's feeling this way.
[Téa playing]
Armin: Advice that I would give other dads going through this would be, "Be strong, do a lot of research, be there for your kids, don't look at your kids [00:10:00] any other way, as they're not normal; they're the same as every other kid. Spend as much time you can with them."
Michelle: If I can give other caregivers advice, it would be, "Join the Alagille Alliance. It really makes a huge difference. Build that relationship with your physician. They're all different. Find one that suits you and your family because it really made [00:10:30] a huge difference for us. There's a light at the end of the tunnel. He might not know everything, but that light just seems a little bit brighter."
INDICATION
LIVMARLI (maralixibat) oral solution and tablets are a prescription medicine used to treat cholestatic pruritus (itch) in patients who are 3 months of age and older [00:11:00] with Alagille syndrome.
It is not known if LIVMARLI is safe and effective in children with Alagille syndrome who are under 3 months of age. It is not known if LIVMARLI is safe and effective in adults who are 65 years of age and older.
IMPORTANT SAFETY INFORMATION
What are the possible side effects of LIVMARLI (maralixibat) oral solution and tablets?
LIVMARLI can cause serious side effects, including:
- Liver injury. Changes in certain liver tests are common in patients but may worsen during treatment with LIVMARLI. [00:11:30] These changes may be a sign of liver injury and can be serious. Your health care provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your health care provider right away if you get any signs or symptoms of liver problems, including:
- nausea or vomiting
- your skin or the white part of your eye turns yellow
- dark or brown urine
- pain on the right side of your stomach (abdomen)
- fullness, bloating, or fluid in your stomach area (ascites)
- loss of appetite
- bleeding or bruising more [00:12:00] easily than normal, including vomiting blood
- Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain during treatment. Diarrhea can also cause the loss of too much body fluid (severe dehydration). Your health care provider may advise you to monitor for new or worsening stomach problems, including stomach pain, diarrhea, blood in your stool, or vomiting. Tell your health care provider right away if you have any new or worsening signs or [00:12:30] symptoms of stomach and intestinal problems, including:
- diarrhea
- more frequent bowel movements than usual
- stools that are black, tarry, or sticky, or have blood or mucous
- severe stomach-area pain or tenderness
- vomiting
- urinating less often than usual
- dizziness
- headache
- A condition called Fat-Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat. FSV deficiency is common [00:13:00] in patients with Alagille syndrome but may worsen during treatment. Your health care provider should do blood tests before starting and during treatment, and may monitor for bone fractures and bleeding, which are common side effects.
Tell your health care provider [00:13:30] about all medicines that you take, as LIVMARLI may interact with other medicines. If you take a medicine that lowers cholesterol by binding bile acids, such as cholestyramine, colesevelam, or colestipol, take LIVMARLI at least 4 hours before or 4 hours after you take that medicine.
Your health care provider may change your dose, or temporarily or permanently stop treatment if you have certain side effects.
LIVMARLI is available in oral solution and tablet formulations. LIVMARLI is taken by mouth, 1 time each day, 30 minutes before a meal in the morning. For the oral solution, be sure to use the provided oral dosing dispenser to accurately measure the dose of medicine.
These are not all of the possible side effects of LIVMARLI. For more information, ask your health care provider or pharmacist. Call your doctor for medical advice about side effects. You may report side effects [00:14:00] to the FDA at 1-800-FDA-1088.
Please see full Prescribing Information, including Patient Information and Instructions for Use, for LIVMARLI at LIVMARLI.com.
[00:14:24] [END OF AUDIO]
