FDA Approves maralixibat as First and Only treatment of rare genetic liver disease
The U.S. Food and Drug Administration (FDA) has approved LIVMARLI™ (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older.
LIVMARLI, a minimally absorbed ileal bile acid transporter (IBAT) inhibitor, is the first and only FDA-approved medication in this rare liver disease which affects 2,000 to 2,500 children in the United States. LIVMARLI is now available for prescribing. In conjunction with the approval, Mirum received a rare pediatric disease priority review voucher.
"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, The Hospital for Sick Children (SickKids), Toronto, Ontario, Canada. "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."
ALGS is a rare genetic disorder caused by abnormalities in bile ducts that can lead to progressive liver disease. Malformed or reduced bile ducts cause cholestasis, the accumulation of bile acids in the liver, which leads to inflammation and liver injury, and prevents the liver from working properly. Cholestasis in ALGS is associated with pruritus which is among the most common indications for liver transplant in ALGS.
The approval of LIVMARLI is based on the pivotal ICONIC study as well as five years of data from supportive studies resulting in a robust body of evidence in 86 patients with ALGS. Data from ICONIC demonstrated statistically significant reductions in pruritus, one of the most common and arduous symptoms associated with the disease, which was maintained through four years.
"Today is a great day for the Alagille syndrome community with the approval of a much-needed new treatment option to address one of the most debilitating effects of this disease," said Chris Peetz, president and chief executive officer of Mirum. "We are grateful to the patients, families, and healthcare professionals who advanced the research and participated in the LIVMARLI clinical studies. Today is also a landmark day for Mirum as we take steps forward in developing potentially life-changing medicines for rare liver disease."
"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, Alagille Syndrome Alliance and an ALGS mom. "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."