In the past, there have not been any available treatments to tackle cholestatic pruritus, or a severe and unrelenting itch, for patients with liver diseases such as Alagille syndrome. However, as of September 29, this has now changed! In a news release, biopharmaceutical company Mirum Pharmaceuticals, Inc. (“Mirum”) shared that its product LIVMARLI (maralixibat), which treats pruritus, has now been FDA-approved for use in patients with Alagille syndrome over 1 year old.
Alagille Syndrome
NOTCH2 or JAG1 gene mutations cause Alagille syndrome, a rare genetic disorder which affects multiple organs throughout the body. For example, patients with Alagille syndrome may see liver, kidney, eye, or even heart inclusion. However, liver disease is one of the major facets of Alagille syndrome, alongside reduced bile ducts and heart abnormalities. Because of the lower amount of bile ducts, bile accumulates in the liver (cholestasis), causing health issues, impaired organ function, and extreme itching. This itching can interrupt sleep, cause skin scarring or scratching, and is an indicator for a future liver transplantation need. In fact, an estimated 60- 75% of patients require a liver transplant before adulthood. Other symptoms include:
- Failure to thrive
- Jaundice (yellowing of the skin and eyes)
- Poor weight gain
- Pale, loose stools and dark urine
- Heart murmurs and other congenital heart defects
- Xanthomas under the skin
- Spinal growth changes
- Enlarged spleen
- Distinctive facial features (deeply-set and widely spaced eyes, broad forehead, pointed chin)
Learn more about Alagille syndrome.
LIVMARLI
Now, patients suffering from the chronic itch associated with Alagille syndrome have a new option: LIVMARLI. This ileal bile acid transporter inhibitor is administered orally. Currently, LIVMARLI is the first and only FDA-approved medicine within this sphere. It has the potential to improve quality of life and reduce itching, for up to 2,500 pediatric patients with Alagille syndrome within the U.S. The approval hinged on data from the ICONIC clinical trial, as well as 5 years of supportive data. Altogether, these highlighted the drug’s benefits.
Beyond Alagille syndrome, Mirum is also exploring LIVMARLI as a potential treatment option for patients with biliary atresia and progressive familial intrahepatic cholestasis (PFIC).
Patients may be prescribed LIVMARLI through Mirum Access Plus. To learn more, head to the LIVMARLI website.
LIVMARLI Safety Information
In clinical trials, LIVMARLI was shown to be relatively safe and well-tolerated (hence its approval)! However, some adverse reactions may occur. If you are taking LIVMARLI, please speak with your doctor if you experience the following:
- Diarrhea
- Nausea and vomiting
- Stomach pain
- Appetite loss
- Dark or brown urine
- Jaundice
- Abdominal pain on the right side
- Gastrointestinal bleeding
- Fat-soluble vitamin deficiency
- Bone fracturing
