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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>Watch Téa Interview Dr Bass
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Watch Téa Interview Dr Bass

Explore the patient experience through the eyes of an 8-year-old. Téa, currently taking LIVMARLI, chats with her beloved physician, Dr Bass, about life with Alagille syndrome and shares how doctors can better help patients like her.

[00:00:00]

[music]

Téa: Hi.

Dr. Bass: Wow.

Téa: How are you?

Dr. Bass: Oh, this is so nice.

Téa: I just wanted you to come. I-

Dr. Bass: Oh, this is great.

Téa: -wanted to-

Dr. Bass: Can I sit here?

Téa: -surprise you. Yes.

Dr. Bass: Oh, great.

Téa: I wanted to surprise you.

Dr. Bass: You did. I'm so surprised. This is amazing. What are we going to be doing here? This is great.

Téa: [00:00:30] I'm going to be asking you some questions and I'm going to be interviewing you.

Dr. Bass: Oh, wow. Okay.

[music]

Téa: If you could only eat one food for the rest of your life, what would it be?

Dr. Bass: See, I have a hard time with this because there's foods that I love. I probably would say either pizza or french fries.

Téa: Okay. Good job. Who's your favorite superhero? [00:01:00] Batman.

Dr. Bass: [laughs] You knew this.

Téa: I knew.

Dr. Bass: You knew this, I like because he's smart and he has all these gadgets that he creates. It's really cool.

Téa: You're smart.

Dr. Bass: I like the fact that he could—that he didn't have a power, he had his mind.

Téa: Yes. What is Alagille syndrome in your own words?

Dr. Bass: Alagille syndrome is a condition that you're born with [00:01:30] and it impacts the ability of some parts of your organs to form. Alagille syndrome can cause you to have problems with your liver or with your heart or with your kidneys. One of the things with your liver that it can impact is that it can cause difficulty in getting something that your liver makes called bile. Bile is this yellowish liquid that dissolves fat. When you eat something that [00:02:00] has fat in it, bile is what's breaking it down and that bile backs up in the setting of Alagille syndrome and that's what causes some of the symptoms of Alagille syndrome.

Téa: How did you know that I had Alagille syndrome?

Dr. Bass: When you were a little baby you had jaundice, meaning you had elevated levels of bilirubin and your skin looked a little yellow and your eyes looked a little yellow. [00:02:30] There was a genetic test that was done that demonstrated that you had Alagille syndrome. Then we took care of you and tried to help you through that time and you've done really well since that time.

Téa: Why do kids like me with Alagille syndrome itch?

Dr. Bass: That's exactly it. There's not enough tubes for that bile to flow through and so it goes into other parts of the body. [00:03:00] You may not even notice that you're itching, but for some people it can feel like they're wiggly, it can feel like they are just scratching distractedly. They don't even know. (This is my example of doing this.) But it's the bile that's in their bloodstream that's causing that to happen because there's too much of it and the liver can't put it all down through the bile ducts or the tubes into the intestine.

Téa: What do you tell kids like [00:03:30] me and their families to look for with the itch?

Dr. Bass: There's a lot of different aspects and it's important that, as a doctor, I understand that itch can be impacting a lot of patients, because I see you—and I love seeing you when I see you—but I see you for 30 to 60 minutes a couple of times a year and so I get a really [00:04:00] small snapshot of your life. And if I'm not asking, "How are you sleeping? How's it going in school? Can you pay attention? Can you sit at the dinner table and eat? What happens when you're watching TV? Are you paying attention or are you potentially itching and distracted by it?"

It's important to me to make sure that I'm asking those questions that might be impacting you. [00:04:30] When you say what's important for you, it's to make sure that you communicate to me what's your life like, what is your daily life like, and how we can help understand that because that helps us understand how we can treat this.

Téa: Does your other patients feel like they're grasping their arm and go itch?

Dr. Bass: I don't know that I've heard that exact [00:05:00] description. The things that I look for is sometimes I come in the room and I'm talking to mom, but I'm looking at you. I want to see what you're doing when you're just sitting there, just like listening. So I see what the patients are doing and trying to make an observation of what's going on. It's one of the ways I can rate how kids are itching or if kids are scratching [00:05:30] and watching what they're doing as they're going.

Téa: Why did you decide that LIVMARLI was right for me?

Dr. Bass: I was very ready to give you LIVMARLI as soon as I saw the FDA approval for it. I knew that you were doing okay. We had you on medicine that was keeping you okay, but I also knew that you weren't perfect. I knew that you would itch a lot. I knew that you would sometimes [00:06:00] have scratches on you when I would see you in the office. The medicines that we were using didn't seem to be enough to take away your itch and so my hope was that I could use LIVMARLI to improve that symptom for you.

Téa: How does LIVMARLI work?

Dr. Bass: What LIVMARLI does is it blocks the absorption of bile acids in the lower part of the intestines. When your liver—I almost like to think of this: Have you ever been to a water park?

Téa: Yes.

Dr. Bass: Okay. The way I like to think about this, the [00:06:30] bile is all going down this big water slide and what do you do at the end of a big water slide?

Téa: You go in the water.

Dr. Bass: You go in the water, and then what do you do right after that? You get up and you want to go right back up the water slide, right?

Téa: Yes.

Dr. Bass: That's really what's happening with all the bile. All the bile goes through and then it goes right back up to the liver, but if there's not enough tubes for that bile to flow, then that bile goes into the rest of the body. What LIVMARLI does is it blocks the ability of that bile [00:07:00] to get out of the water and go back up to the slide. It allows it to leave the body. That's how it works.

Téa: How do kids like me know if LIVMARLI is working?

Dr. Bass: For you, you should hope that you feel less itching. You should hope that you should sleep better. You should hope that you have less irritation and agitation. If sometimes [00:07:30] kids have some jaundice and hopefully that should go away too. For me, I can measure the bile in your blood and so one of the things that I've done as we've done some of the blood tests, and I know you don't love the blood test. As we've done that, we've measured and we've found that your bile acids, which were high, are much lower now on the LIVMARLI, so that improved things. Certainly, there are some side effects that can occur with LIVMARLI as we talked [00:08:00] about. When I talked about the blocking the absorption of the intestine in the bile well, and we get that bile out of your body.

How does stuff get out of your body? One of two different ways. You either go pee or you go poop and so it goes out through your poop. Sometimes people have to go to the bathroom and go poop more. They might have some diarrhea. They might, if they feel like they have to go poop, sometimes they [00:08:30] feel like that they have a little bit of pain and sometimes they can, sometimes even have a little bit of nausea and throw up but in reality, my experience has been that people have tolerated it really well so far.

[music]

Téa: When I went in, when I was younger, I used to be very scared. But since I got older, I was so happy that I got to see you [00:09:00] more and more often, and that's the memories I have with you.

Dr. Bass: Yes. I remember now actually it's really nice to see you grow up because at one point you were happy to see me and then I would examine you and you wouldn't like that I was doing anything. Now you actually are very informed about what's going on with your body and you're asking questions and you're taking [00:09:30] some ownership of that. That's really going to be useful for you, for your health going forward if that's a really good habit and thing to have for you.

Téa: That's amazing.

Dr. Bass: I always look forward to seeing you guys. As a doctor, I don't do this for thank-yous. I do it because I like doing it, but when I get thank-yous and appreciation from families, it's [00:10:00] really a wonderful thing and it keeps me going, and you and your family and your mom, your grandpa, you always show appreciation.

Téa: You have made me happy for the last—for my whole life. You always make me happy. You are just the best doctor I could ever have. I'm very, very glad you came here today.

Dr. Bass: I am so glad I came here. I'm so glad that [00:10:30] we could sit and talk like this. This has really been wonderful.

[music]

Dr. Bass: First off, what is your favorite sport or activity outside of school?

Téa: Gym.

Dr. Bass: Gym? What do you like to do in gym class?

Téa: I like to do cartwheels. I like to do the splits.

Dr. Bass: Okay.

Téa: I like to run, and I like [00:11:00] to do—I like to exercise.

Dr. Bass: Excellent. Now do you do cartwheels, or do you do roundoffs, because I learned that there was a difference?

Téa: Yes, roundoff.

Dr. Bass: Roundoffs? Excellent. When you had the itch, what did it feel like to you? How did you feel it inside?

Téa: It felt really bad. It felt like something was in my body, and when I was younger, I didn't know any of this stuff, but now since I'm getting older and I know what's in my body and LIVMARLI is [00:11:30] helping.

Dr. Bass: That's wonderful. Did you have certain times of day, like morning it was worse, right before you went to bed it was worse? What was—Did you have [crosstalk]

Téa: I think night was the worst.

Dr. Bass: Nighttime was worse? Okay. This is actually a really good question, I've never asked you this before. How would you describe Alagille syndrome to your friends?

Téa: I would describe that I have Alagille syndrome [00:12:00] and sometimes they would ask me, "What is that?" "It's something that makes you itch a lot and you can't stop, and even though you're trying to stop, it's not working."

Dr. Bass: Okay.

Téa: Now that I'm taking LIVMARLI, it's helping, and you guys don't see me itching more. You just see me itching less.

Dr. Bass: That's good. That's a good description. I like the description. What advice [00:12:30] would you give other kids with Alagille syndrome?

Téa: I would say to ask your doctor if you can have LIVMARLI like me.

Dr. Bass: Okay. That's great. One of the other things that I was curious about is would you tell other kids to say, "Don't be afraid to talk about the itch"?

Téa: Do not be afraid.

Dr. Bass: Yes.

Téa: You tell anybody.

Dr. Bass: Right, because people don't know they should tell people. We want other kids to know that they can talk about what they're feeling.

Téa: Yes. [00:13:00] Anybody you know, you immediately tell. It's better than just keeping it away.

Dr. Bass: Excellent. Well, those are all the questions I have for you.

Téa: Well, I have a special guest coming named Mom.

Dr. Bass: Really?

Téa: Yes.

Dr. Bass: Oh, wow. I'd love to see her.

[music]

Téa: Yes, she's coming.

Téa's Mother: Hi.

Dr. Bass: Hi.

Téa's Mother: Hi, Dr. Bass. It's so good to see you, Dr. Bass.

Dr. Bass: Yes.

Téa's Mother: I definitely wanted to just thank you—[tears up] [00:13:30]

Téa: For everything?

Téa's Mother: [laughs] Sorry.

Dr. Bass: It's okay.

[silence]

Téa's Mother: Not only are you her doctor, but you're just such an amazing person. You saved her, you saved me. I would hope everybody has relationships with their doctors as we do with you. It was hard at first. We couldn't find a doctor that was just [00:14:00] a right fit. You're our third doctor that we saw and I would never change you. You speak to me like a human. Sometimes the scientific words get out of hand and you never do that to me. You talk to me, you draw me diagrams, and we appreciate that.

You hold such a special place in all of our family's hearts and everything that you have done for us. Nothing in the world can ever [00:14:30] repay you what you have done for Téa mentally, physically, and for all of us. Téa loves coming to see you. A lot of kids are scared of their doctor, and they don't really talk to them, but no, I've seen you dance in the room with her and you make her laugh, and your jokes are funny.

[laughter]

She talks about you all the time. She loves to come see you and that's [00:15:00] huge. It's so heartfelt. When she tells everybody and she's like, "I'm going to go see Dr. Bass, he's my favorite." It means a lot as a parent for her to be, not just you, the whole team. She's so excited to see your team and your nurses and she knows that there's a special place for her there. The fact that she's excited to come there is all I can ever ask for, and I can't thank you enough.

Dr. Bass: [00:15:30] For me this is why I do what I do. It's to see her grow up. It's to have the ability to see her really sick as a baby and to get her better and to see her like this today is the biggest reason that I do what I do to have a relationship with you guys. It's not just you, it's the entire family. It's all Team Téa and [00:16:00] has been just amazing to work with you. Then the advocacy to be like, we know there's something better out there for her. We know we can do this. We know we are watching for this and being able to try and find the next thing to help her get better, which is how we got to this today with this drug, is that we're working together to do it.

Téa's Mother: I agree.

Dr. Bass: For her, and of course the [00:16:30] most important thing is her.

Téa's Mother: Always. [laughs]

Téa: Would you like some tea?

Dr. Bass: I would love some tea.

Téa: Here is your tea, here is your coffee and—

Téa's Mother: [laughter] Can we get a cheers?

Téa: Yes. Let's do it. [crosstalk] Cheers.

[music]

Speaker 3:

INDICATION

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

IMPORTANT SAFETY INFORMATION [00:17:00]

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 [00:17:30] other causes. 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. [00:18:00] 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 [00:18:30] stopping 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 [00:19:00] if 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 [00:19:30] 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 in the morning. The provided oral dosing dispenser must be used to accurately measure the dose. [00:20:00] Any remaining LIVMARLI should be discarded 100 days after first opening the bottle.

[00:20:26] [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.