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Tyler, a person with Alagille syndrome

REAL PEOPLE TAKING LIVMARLI

Hear from real patients and their families as they share their unique experiences—life before diagnosis, challenges before starting LIVMARLI, and the difference LIVMARLI is making in their lives.*

*Not all patients taking LIVMARLI will have the same experiences.

Videos>Meet Téa
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Meet Téa

Téa struggled with persistent itching and disrupted sleep until she started LIVMARLI.

[00:00:00]

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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 is a very good sister and I love her to the moon and back. This is Peppermint and this is Kiwi, and 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.

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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.

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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, it 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 and 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 and 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 goes to work. She brings the baby by us. We have a crib for her, and we put them in a bed, the same house she laying down and her hands go all around in the [00:03:30] body, more time go in her 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, and I think because I just made it so—it was 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 [00:05:00] LIVMARLI. 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]

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Michelle: We first learned about LIVMARLI through the clinical trials, so 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, and she was snoring, and it was so funny to me, but also very sad to know that it took 8 years [00:06:30] 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 itch. [00:07:00]

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, Téa is taking LIVMARLI, I feel so happy. It’s helping her. She’s not scratching. She sleeps good. [00:07:30] She's full energy.

Téa: LIVMARLI helps me sleep better because I itch less. I'm not scratching and it makes me feel better, and I'm excited that I can do more things.

Armin: Tell me when it's sauce time.

Téa: Okay.

Michelle: When Téa was prescribed LIVMARLI, I received a call from Mirum Access Plus, and it was just a rundown of the [00:08:00] medication, potential side effects, how delivery was going to work, if I had any questions or concerns, who to call, and 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. We went through, it was approved through insurance, and 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, and 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 like, we don't want to talk about it. Let's just keep going. I definitely feel like the Alagille Syndrome Alliance is such a great—there's so much amazing information. Roberta is very communicative on the Alliance [00:09:30] and it's just a great resource, and it's amazing to see how many kids there really are and you're not the only one that's feeling this way.

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Armin: Advice that I would give other dads going through this, would be to 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 light at the end of the tunnel. He might not know everything, but that light just seems a little bit brighter.

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Narrator:

INDICATION

LIVMARLI is indicated for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 3 months of age and older. [00:11:00]

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Liver Test Abnormalities: Patients enrolled in clinical trials had abnormal liver tests at baseline. In the main clinical trial, treatment-emergent elevations or worsening of liver tests (ALT, AST or T/DB) relative to baseline were observed. Obtain baseline liver tests and monitor during treatment. Dose reduction or treatment interruption may be considered if abnormalities occur in the absence of other causes. [00:11:30] For persistent or recurrent liver test abnormalities, consider treatment discontinuation. Discontinue permanently if a patient progresses to portal hypertension or experiences a hepatic decompensation event.

GI Adverse Reactions: Diarrhea, abdominal pain and vomiting were reported as the most common adverse reactions. If diarrhea, abdominal pain and/or vomiting occur and no other etiologies are found, consider reducing the dose or interrupting LIVMARLI. [00:12:00] For diarrhea or vomiting, monitor for dehydration and treat promptly. Consider interrupting LIVMARLI dosing if a patient experiences persistent diarrhea or has diarrhea with accompanying signs and symptoms such as bloody stool, vomiting, dehydration requiring treatment, or fever. Restart LIVMARLI at 190 mcg/kg/day when diarrhea, abdominal pain or vomiting resolve, and increase the dose as tolerated. If they recur upon re-challenge, consider stopping [00:12:30] LIVMARLI treatment.

Fat-Soluble Vitamin Deficiency: ALGS patients can have fat-soluble vitamin (FSV) deficiency (vitamins A, D, E, and K) at baseline, and LIVMARLI may affect absorption of FSV. In the main clinical trial, treatment emergent FSV deficiency was reported in 3 (10%) patients during 48 weeks of treatment. Obtain baseline serum levels and monitor during treatment, along with any clinical manifestations. Supplement if [00:13:00] deficiency is observed. Consider discontinuing LIVMARLI if FSV deficiency persists or worsens despite adequate FSV supplementation.

ADVERSE REACTIONS

The most common adverse reactions (≥5%) are diarrhea, abdominal pain, vomiting, fat-soluble vitamin deficiency, liver test abnormalities, gastrointestinal bleeding and bone fractures.

DRUG INTERACTIONS

Administer bile acid binding resins at least 4 hours [00:13:30] before or 4 hours after administration of LIVMARLI.
A decrease in the absorption of OATP2B1 substrates (eg, statins) due to OATP2B1 inhibition by LIVMARLI in the GI tract cannot be ruled out. Consider monitoring the drug effects of OATP2B1 substrates as needed.

DOSING INFORMATION

LIVMARLI should be taken 30 minutes before a meal in the morning. The provided oral dosing dispenser must be used to accurately measure the dose. Any [00:14:00] remaining LIVMARLI should be discarded 100 days after first opening the bottle.

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[00:14:25] [END OF AUDIO]

Root Out
Excess Bile

Learn how LIVMARLI—the first FDA-approved treatment for cholestatic pruritus in Alagille syndrome—battles bile acid buildup.1

See How LIVMARLI Works

Encourage patients to download the Itch✓ app to help them track symptom patterns over time and generate customized reports to share at appointments.

Check Out the Itch✓ App

Mirum Access Plus assists both you and your patients at every turn, helping you navigate the payer approval process—and beyond—with ease.

Learn More About
Mirum Access Plus
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Important Safety Information

Contraindications

LIVMARLI is contraindicated in patients with prior or active hepatic decompensation events (eg, variceal hemorrhage, ascites, or hepatic encephalopathy).

Warnings and Precautions

Hepatotoxicity: LIVMARLI treatment is associated with a potential for drug-induced liver injury. In the Alagille syndrome trial, treatment-emergent elevations of liver tests or worsening of liver tests occurred.

Obtain baseline liver tests and monitor during treatment. Liver-related adverse reactions and physical signs of hepatic decompensation should also be monitored. Dose reduction or treatment interruption may be considered if abnormalities occur in the absence of other causes. For persistent or recurrent liver test abnormalities, consider treatment discontinuation. Permanently discontinue LIVMARLI if a patient experiences the following: persistent or recurrent liver test abnormalities, clinical hepatitis upon rechallenge, or a hepatic decompensation event.

GI Adverse Reactions: Diarrhea and abdominal pain were reported as the most common adverse reactions. Monitor for dehydration and treat promptly. Consider reducing the dosage or interrupting dosing if a patient experiences persistent diarrhea or has diarrhea with bloody stool, vomiting, dehydration requiring treatment, or fever.

Fat-Soluble Vitamin Deficiency: Patients can have fat-soluble vitamin (FSV) deficiency (vitamins A, D, E, and K) at baseline, and LIVMARLI may adversely affect absorption of FSV. If bone fractures or bleeding occur, consider interrupting LIVMARLI and supplement with FSVs. LIVMARLI can be restarted once FSV deficiency is corrected and maintained at corrected levels.

Adverse Reactions

The most common adverse reactions are diarrhea, abdominal pain, vomiting, FSV deficiency, liver test abnormalities, and bone fractures.

Drug Interactions

Administer bile acid binding resins at least 4 hours before or 4 hours after administration of LIVMARLI. A decrease in the absorption of OATP2B1 substrates (eg, statins) due to OATP2B1 inhibition by LIVMARLI in the GI tract cannot be ruled out. Consider monitoring the drug effects of OATP2B1 substrates as needed.

Dosing Information

LIVMARLI should be taken 30 minutes before a meal. The provided oral dosing dispenser must be used to accurately measure the dose. Any remaining LIVMARLI should be discarded 100 days after first opening the bottle.