Mirum Pharmaceuticals has launched a novel drug Maralixibat, for the treatment of pruritus associated with Alagille syndrome (ALGS) in patients one year of age and older.
ALGS is a rare genetic disorder within which digestive juice ducts are abnormally slim, unshapely and reduced in a range that ends up in digestive juice within the liver and ultimately progressive disease.
The multiple organ systems are also littered with the mutation, together with the heart, liver, kidneys and central nervous system, in patients with ALGS.
Maralixibat was granted breakthrough therapy designation based on evidence from the ICONIC Phase 2b clinical trial in children with ALGS.
Maralixibat is an ileal bile acid transporter inhibitor in clinical development for pediatric cholestasis, Alagille syndrome, and progressive familial intrahepatic cholestasis.
The breakthrough designation is granted by the FDA to investigational medicines supposed to treat a serious or life-threatening condition for which preliminary clinical evidence could demonstrate substantial improvement on at least one clinically significant endpoint over available therapy.
The breakthrough therapy designation is intended to expedite development and review and conveys all the fast track designation program options, including a lot of intensive federal agency interaction and guidance.
