The U.S. Food and Drug Administration (FDA) has approved maralixibat (Livmarli) for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older.
Alagille syndrome is an inherited disorder that can affect the liver, heart, skeleton, eyes, and kidneys. Liver damage caused by cholestasis is a major feature of the disease. Symptoms range from mild to severe, sometimes requiring transplantation.
Maralixibat inhibits the apical sodium dependent bile acid transporter (ASBT), resulting in more bile acids being excreted in the feces, leading to lower levels of bile acids systemically, thus reducing bile acid mediated liver damage and related effects and complications.
In a news release, Binita M. Kamath, MBBChir, from the Hospital for Sick Children (SickKids) in Toronto, Canada said,
“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,” adding, “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.”
In the clip below, Chris Peetz, president and chief executive officer of Mirum discussed the drug and its eminent approval in an exclusive interview with CheckRare earlier this year.
Mirum Pharmaceuticals, the developers of the drug, also received a rare pediatric disease priority review voucher. These vouchers are given to drugs approved for a pediatric indication. The vouchers can be used for future new drug applications with the FDA and reduce the time for a application approval from the standard 10 months to six months. More commonly, the vouchers are sold by smaller companies such as Mirum Pharmaceutical to larger companies wanting a competitive edge. Earlier this year, Rhythm Pharmaceuticalssold their Priority Review Voucher to Alexion for $100 million.