Mirum Snags First Approval for LIVMARLI Drug for Treating Rare, Genetic Liver Disorder
LIVMARLI is now FDA approved.
Mirum Pharmaceuticals has received approval from the U.S. Food and Drug Administration (FDA) to start selling its oral medication LIVMARLI to treat cholestatic pruritus in patients diagnosed with Alagille syndrome (ALGS).
LIVMARLI (maralixibat) is indicated for ages one year and older. It is an orally administered, once-daily ileal bile acid transporter (IBAT) inhibitor. It will now be accessible to patients with a prescription through Mirum Access Plus, the company's patient support services program and single-source specialty pharmacy. The MAP program works with healthcare providers and families to provide access support, insurance coverage, and financial support options for the drug.
The Significance of LIVMARLI's Approval
ALGS is a rare genetic disease caused by bile duct abnormalities, leading to progressive liver disease. The shrunken or malformed bile ducts cause cholestasis, which eventually develops into inflammation and liver injury. It affects 2,000 to 2,5000 children in the U.S. alone. The estimated incidence of ALGS is one in every 30,000 people.
"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," commented Binita M. Kamath, MBBChir, pediatric pathologist at The Hospital for Sick Kids (SickKids) in Toronto, 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," she continued.
The FDA's green light comes with a rare pediatric priority review voucher. The regulator's decision is based on positive results from Mirum's Phase IIB ICONIC study and five years' worth of data from supportive research that produced a body of evidence in 86 patients with ALGS. The ICONIC trial showed statistically significant reductions in pruritus, which was maintained through four years. Pruritus is one of the most common and arduous symptoms associated with ALGS.
"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. 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," said Chris Peetz, president and chief executive officer of Mirum.
Mirum Pharmaceuticals announced in March that the FDA accepted its New Drug Application (NDA) for maralixibat for filing and priority review.
LIVMARLI is also currently being assessed in late-stage clinical studies for its ability to treat other rare cholestatic liver diseases, including progressive familial intrahepatic cholestasis and biliary atresia, both of which have received Breakthrough Therapy and Orphan Drug designations.