Alagille Syndrome

Children with Alagille syndrome who received 48 weeks of Maralixibat had clinically meaningful reduction in pruritus and improvements in quality of life, according to a presentation at The Liver Meeting Digital Experience.

Mirum Pharmaceuticals completed a clinical pre-new drug application meeting with the FDA for maralixibat, the company’s therapeutic candidate for the treatment of Alagille syndrome-associated pruritus, according to a press release.

BOSTON — Maralixibat demonstrated durable control of serum bactericidal antibody, pruritus, and xanthoma in children with Alagille syndrome, according to data from the ICONIC study presented at The Liver Meeting 2019.