Mirum Pharmaceuticals announced the U.S. FDA has granted Breakthrough Therapy Designation for maralixibat for the treatment of pruritus associated with Alagille syndrome (ALGS) in patients 1 year of age and older. 

The Breakthrough Therapy Designation of maralixibat was granted based on evidence from the ICONIC Phase 2b clinical trial in children with ALGS. Results were recently presented at the International Liver Congress (EASL). In the trial, patients taking maralixibat had significant reductions in bile acids and pruritus compared to placebo. Maralixibat was generally well-tolerated throughout the studies. The most frequent adverse events were diarrhea, abdominal pain, and vomiting.

Breakthrough Therapy Designation is granted by the FDA to investigational medicines intended to treat a serious or life-threatening condition for which preliminary clinical evidence may demonstrate substantial improvement on at least one clinically significant endpoint over available therapy. Breakthrough Therapy Designation is intended to expedite development and review and conveys all of the fast track designation program features, including more intensive FDA interaction and guidance. 

About Alagille Syndrome 

ALGS is a rare genetic disorder in which bile ducts are abnormally narrow, malformed and reduced in number, which leads to bile accumulation in the liver and ultimately progressive liver disease. The estimated incidence of ALGS is one in every 30,000 to 50,000 births in the United States and Europe. In patients with ALGS, multiple organ systems may be affected by the mutation, including the liver, heart, kidneys and central nervous system. The accumulation of bile acids prevents the liver from working properly to eliminate waste from the bloodstream and leads to progressive liver disease that ultimately requires liver transplantation in 15% to 47% of patients. Signs and symptoms arising from liver damage in ALGS may include jaundice, pruritus, and xanthomas, which are disfiguring cholesterol deposits under the skin. The pruritus experienced by patients with ALGS is among the most severe in any chronic liver disease and is present in most affected children by the third year of life. 

About Maralixibat 

Maralixibat is a novel, minimally-absorbed, an orally administered investigational drug being evaluated in several rare cholestatic liver diseases for pediatric populations. Maralixibat inhibits the apical sodium-dependent bile acid transporter, which results in more bile acids being excreted in the feces, leading to lower levels of bile acids systemically, thereby potentially reducing bile acid-mediated liver damage and related effects and complications. 

Source: Mirum Pharmaceuticals

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