Meet Téa | LIVMARLI® (maralixibat) oral solution and tablets | HCP

Téa struggled with persistent itching and disrupted sleep before LIVMARLI

Video of Téa

[00:00:00] [music]

Téa: My name is Téa. This is my dad, [00:00:30] Armin. This is my mom, Michelle. This is my grandpa, Peter. 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. And that's [00:01:00] my family.

[music]

Téa: 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.

[music]

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 like 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. So 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 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. 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." In reality, it was.

[music] [00:05:30]

Michelle: 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, but also very sad to know that it took [00:06:30] 8 years for her to sleep all night without sitting up and itching in the middle of the night. And 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 [00:07:00] not 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 Téa take LIVMARLI, I feel so happy helping her. She no 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.

Peter: Tell me when it's sauce time.

Téa: Okay.

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. 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. 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."

[laughter]

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.

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 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, and 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.

Speaker 5:

INDICATIONS

LIVMARLI (maralixibat) oral solution and tablets are indicated for the treatment of cholestatic pruritus in patients who are 3 months of age and older with Alagille syndrome.

LIVMARLI (maralixibat) oral [00:11:00] solution and tablets are indicated for the treatment of cholestatic pruritus in patients who are 12 months of age and older with progressive familial intrahepatic cholestasis (PFIC).

Limitations of Use: LIVMARLI is not recommended in a subgroup of PFIC type 2 patients with specific ABCB11 variants resulting in nonfunctional or complete absence of bile salt export pump (BSEP) protein.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

LIVMARLI (maralixibat) [00:11:30] oral solution and tablets are 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 (DILI). In the PFIC trial, treatment-emergent hepatic decompensation events and elevations of liver tests or worsening of liver tests occurred. Two patients experienced DILI [00:12:00] attributable to LIVMARLI. Two additional patients experienced DILI in the open-label extension portion of the trial. Of these 4 patients, 1 patient required liver transplant and another patient died.

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 [00:12:30] 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, or a hepatic decompensation event.

Gastrointestinal Adverse Reactions: Diarrhea and abdominal pain were reported as the most common adverse reactions. Monitor for dehydration and treat promptly. Consider reducing the dosage [00:13:00] or interrupting LIVMARLI treatment if a patient experiences persistent diarrhea or diarrhea with bloody stool, vomiting, dehydration requiring treatment, or fever.

Fat-Soluble Vitamin (FSV) Deficiency: Patients can have FSV deficiency (vitamins A, D, E, and K) at baseline, and LIVMARLI may adversely affect absorption of FSVs. Treatment-emergent bone fracture events have been observed more frequently with patients treated with LIVMARLI compared with patients treated with placebo. If bone fractures or bleeding occurs, [00:13:30] consider interrupting LIVMARLI and supplement with FSVs. LIVMARLI can be restarted once FSV deficiency is corrected and maintained at corrected levels.

Risk of Propylene Glycol Toxicity (Pediatric Patients Less Than 5 Years of Age): LIVMARLI oral solution contains propylene glycol. Total daily intake of propylene glycol should be considered for managing the risk of propylene glycol toxicity. Monitor patients for signs of propylene glycol toxicity. Discontinue LIVMARLI if toxicity is suspected. [00:14:00]

ADVERSE REACTIONS

Alagille syndrome: The most common adverse reactions are diarrhea, [00:16:30] abdominal pain, vomiting, FSV deficiency, liver test abnormalities, and bone fractures.

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

DRUG INTERACTIONS

Administer LIVMARLI at least 4 hours before or 4 hours after [00:14:30] administration of bile acid binding resins.

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

In patients with Alagille syndrome, LIVMARLI is taken once daily, 30 minutes before a meal in the morning. In patients with PFIC, LIVMARLI is taken [00:15:00] twice daily, 30 minutes before a meal. Refer to the dosing by weight guidelines and the full Prescribing Information for complete details on dosing for the oral solution and tablet formulations. The provided oral solution dosing dispenser must be used to accurately measure the dose. Any remaining LIVMARLI oral solution should be discarded 100 days after first opening the bottle. LIVMARLI tablets can be used in patients weighing ≥25 kg who can swallow tablets.

Please see full Prescribing Information [00:15:30] for LIVMARLI at LIVMARLIhcp.com.

[music] [00:16:00]

[00:16:01] [END OF AUDIO]

References:

1. Gonzales E, Hardikar W, Stormon M, et al. Efficacy and safety of maralixibat treatment in patients with Alagille syndrome and cholestatic pruritus (ICONIC): a randomised phase 2 study. Lancet. 2021;398(10311):1581-1592. doi:10.1016/S0140-6736(21)01256-3 2. LIVMARLI® (maralixibat). Prescribing Information. Mirum Pharmaceuticals, Inc. 3. Miethke AG, Moukarzel A, Porta G, et al. Maralixibat in progressive familial intrahepatic cholestasis (MARCH-PFIC): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Gastroenterol Hepatol. 2024;9(7):620-631. doi:10.1016/S2468-1253(24)00080-3

Indications

LIVMARLI (maralixibat) oral solution and tablets are indicated for the treatment of cholestatic pruritus in patients who are 3 months of age and older with Alagille syndrome.

LIVMARLI (maralixibat) oral solution and tablets are indicated for the treatment of cholestatic pruritus in patients who are 12 months of age and older with progressive familial intrahepatic cholestasis (PFIC).

Limitations of Use: LIVMARLI is not recommended in a subgroup of PFIC type 2 patients with specific ABCB11 variants resulting in nonfunctional or complete absence of bile salt export pump (BSEP) protein.

Important Safety Information

CONTRAINDICATIONS

LIVMARLI (maralixibat) oral solution and tablets are 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 (DILI). In the PFIC trial, treatment-emergent hepatic decompensation events and elevations of liver tests or worsening of liver tests occurred. Two patients experienced DILI attributable to LIVMARLI. Two additional patients experienced DILI in the open-label extension portion of the trial. Of these 4 patients, 1 patient required liver transplant and another patient died.

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, or a hepatic decompensation event.

Gastrointestinal 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 LIVMARLI treatment if a patient experiences persistent diarrhea or diarrhea with bloody stool, vomiting, dehydration requiring treatment, or fever.

Fat-Soluble Vitamin (FSV) Deficiency: Patients can have FSV deficiency (vitamins A, D, E, and K) at baseline, and LIVMARLI may adversely affect absorption of FSVs. Treatment-emergent bone fracture events have been observed more frequently with patients treated with LIVMARLI compared with patients treated with placebo. If bone fractures or bleeding occurs, consider interrupting LIVMARLI and supplement with FSVs. LIVMARLI can be restarted once FSV deficiency is corrected and maintained at corrected levels.

Risk of Propylene Glycol Toxicity (Pediatric Patients Less Than 5 Years of Age): LIVMARLI oral solution contains propylene glycol. Total daily intake of propylene glycol should be considered for managing the risk of propylene glycol toxicity. Monitor patients for signs of propylene glycol toxicity. Discontinue LIVMARLI if toxicity is suspected.

ADVERSE REACTIONS

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

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

DRUG INTERACTIONS

Administer LIVMARLI at least 4 hours before or 4 hours after administration of bile acid binding resins.

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

In patients with Alagille syndrome, LIVMARLI is taken once daily, 30 minutes before a meal in the morning. In patients with PFIC, LIVMARLI is taken twice daily, 30 minutes before a meal. Refer to the dosing by weight guidelines in the full Prescribing Information for complete details on dosing for the oral solution and tablet formulations. The provided oral solution dosing dispenser must be used to accurately measure the dose. Any remaining LIVMARLI oral solution should be discarded 100 days after first opening the bottle. LIVMARLI tablets can be used in patients weighing ≥25 kg who can swallow tablets.

Please see full Prescribing Information for LIVMARLI.

Indications

LIVMARLI (maralixibat) oral solution and tablets are indicated for the treatment of cholestatic pruritus in patients who are 3 months of age and older with Alagille syndrome.

LIVMARLI (maralixibat) oral solution and tablets are indicated for the treatment of cholestatic pruritus in patients who are 12 months of age and older with progressive familial intrahepatic cholestasis (PFIC).

Limitations of Use: LIVMARLI is not recommended in a subgroup of PFIC type 2 patients with specific ABCB11 variants resulting in nonfunctional or complete absence of bile salt export pump (BSEP) protein.

Important Safety Information

CONTRAINDICATIONS

LIVMARLI (maralixibat) oral solution and tablets are 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 (DILI). In the PFIC trial, treatment-emergent hepatic decompensation events and elevations of liver tests or worsening of liver tests occurred. Two patients experienced DILI attributable to LIVMARLI. Two additional patients experienced DILI in the open-label extension portion of the trial. Of these 4 patients, 1 patient required liver transplant and another patient died.

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, or a hepatic decompensation event.

Gastrointestinal 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 LIVMARLI treatment if a patient experiences persistent diarrhea or diarrhea with bloody stool, vomiting, dehydration requiring treatment, or fever.

Fat-Soluble Vitamin (FSV) Deficiency: Patients can have FSV deficiency (vitamins A, D, E, and K) at baseline, and LIVMARLI may adversely affect absorption of FSVs. Treatment-emergent bone fracture events have been observed more frequently with patients treated with LIVMARLI compared with patients treated with placebo. If bone fractures or bleeding occurs, consider interrupting LIVMARLI and supplement with FSVs. LIVMARLI can be restarted once FSV deficiency is corrected and maintained at corrected levels.

Risk of Propylene Glycol Toxicity (Pediatric Patients Less Than 5 Years of Age): LIVMARLI oral solution contains propylene glycol. Total daily intake of propylene glycol should be considered for managing the risk of propylene glycol toxicity. Monitor patients for signs of propylene glycol toxicity. Discontinue LIVMARLI if toxicity is suspected.

ADVERSE REACTIONS

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

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

DRUG INTERACTIONS

Administer LIVMARLI at least 4 hours before or 4 hours after administration of bile acid binding resins.

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

In patients with Alagille syndrome, LIVMARLI is taken once daily, 30 minutes before a meal in the morning. In patients with PFIC, LIVMARLI is taken twice daily, 30 minutes before a meal. Refer to the dosing by weight guidelines in the full Prescribing Information for complete details on dosing for the oral solution and tablet formulations. The provided oral solution dosing dispenser must be used to accurately measure the dose. Any remaining LIVMARLI oral solution should be discarded 100 days after first opening the bottle. LIVMARLI tablets can be used in patients weighing ≥25 kg who can swallow tablets.

Please see full Prescribing Information for LIVMARLI.