Investigational Drug Could Be First-Ever Approved Treatment for Alagille Syndrome скачать в хорошем качестве

Investigational Drug Could Be First-Ever Approved Treatment for Alagille Syndrome

Chris Peetz, President and CEO of Mirum Pharmaceuticals, discusses the investigational drug, maralixibat that is being developed to treat alagille syndrome. Alagille syndrome is an inherited disorder that can affect the liver, heart, skeleton, eyes and kidneys. As Mr. Peetz explains, liver damage caused by cholestasis is a major feature of the disease. Bile ducts may be narrow, malformed or fewer in number. As a result, bile builds up in the liver and causes scarring. Signs and symptoms may include jaundice, poor weight gain and growth, and severe pruritus that generally presents in infancy or early childhood. Symptoms range from mild to severe, sometimes requiring transplantation. Currently, there is no approved treatment for Alagille syndrome. 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. Mirum has filed a rolling submission to the FDA for maralixibat as a treatment of alagille syndrome and the FDA has granted them Breakthrough Therapy designation for maralixibat as a treatment of Alagille syndrome as well. The submission was supported by positive results of the ICONIC Phase 2b clinical trial, in which patients taking maralixibat had significant reductions in bile acids and pruritus compared to placebo. Mirum also launched an extended access program for maralixibat available to Alagille syndrome patients. To learn more about Alagille syndrome and other rare metabolic diseases, visit checkrare.com/diseases/metabolic-disorders/