Shots:

• The approval was based on the P-IIb (ICONIC) OLE study incl. 6yr. data of Livmarli vs PBO in patients with ALGS
• The (ICONIC) study showed a reduction in pruritus with a mean difference of -1.4 points, reductions in serum bile acids which were durably maintained over multiple yrs. of treatment, reductions in xanthoma severity, cholesterol & improvements in transplant-free survival & growth
• The product is expected to be available in Germany in early 2023 & is being studied in late-stage clinical studies in other rare cholestatic liver diseases & also received BTD for ALGS & PFIC type 2; ODD for ALGS, PFIC & biliary atresia. The therapy is currently under regulatory review in China, Taiwan, South Korea, and Great Britain

Ref: Businesswire | Image: Mirum