Officials with the FDA have approved maralixibat (Livmarli, Mirum Pharmaceuticals) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older. According to a press release from Mirum, maralixibat is the first and only FDA-approved medication to treat ALGS, a rare liver disease that affects 2000 to 2500 children in the United States. Maralixibat is a minimally absorbed ileal bile acid transporter inhibitor.
“Children with Alagille syndrome suffer from cholestatic pruritus, which is serious, unremitting, and debilitating. Their sleep is disrupted, and they endure bleeding and scarring of the skin due to unrelenting scratching,” said Binita M. Kamath, MBBChir, pediatric hepatologist at The Hospital for Sick Children in Toronto, Ontario, Canada, in a press release. “There have been no approved treatments to date for cholestatic pruritus in Alagille syndrome, and many children ultimately require major surgical interventions such as liver transplantation for refractory pruritus. The approval of Livmarli signifies a meaningful shift in the treatment paradigm for Alagille syndrome and provides hope for the many families who have lived with persistent itch for far too long.”
The current approval is based on the results of the ICONIC study, as well as 5 years of data from supportive studies. A total of 86 patients with ALGS were included in the analysis. According to the investigators, the data from ICONIC demonstrated a statistically significant reduction in pruritus maintained through 4 years.
“We have had the pleasure of being part of and closely following the clinical progress of Livmarli in many ways. Since the first study’s initiation more than a decade ago, we have dreamed of today, seeing Livmarli receive FDA approval, marking an incredibly meaningful milestone for the ALGS community,” said Roberta Smith, president of the Alagille Syndrome Alliance and an ALGS mom, in the release. “Until now, patients have had limited-to-no treatment options to address the severe and unrelenting itch that significantly impacts both patients and their families. Additionally, because pruritus associated with ALGS greatly impacts caregivers, having a strong support program like Mirum Access Plus to reduce the strain on families is so important. The ALGS community has been waiting for a long time for a treatment and we’re so pleased that Livmarli is now available in the United States.”
ALGS is a rare genetic disorder that is caused by abnormalities in bile ducts that can lead to progressive liver disease. These malformed bile ducts cause cholestasis—the accumulation of bile acids in the liver—which can lead to inflammation and liver injury. Cholestasis in patients with ALGS can result in pruritus, which is among the most common indications for liver transplant for these patients.
U.S. FDA approves Livmarli (maralixibat) as the first and only approved medication for the treatment of cholestatic pruritus in patients with alagille syndrome one year of age and older [news release]. BusinessWire; September 29, 2021. Accessed September 30, 2021. https://www.businesswire.com/news/home/20210929005810/en