EMA’s CHMP backs two orphan drugs, rebuffs FDA-approved NPC therapy

At its July meeting, EMA’s CHMP recommended eight new medicines, including orphan therapies from GSK/Alfasigma and Minoryx/Neuraxpharm, but rebuffed three new medicines, including Zevra’s FDA-approved drug Zevra for Niemann-Pick disease type C.

Among the positive opinions was one for Lynavoy linerixibat, an oral IBAT inhibitor from GSK plc (LSE:GSK; NYSE:GSK), to treat cholestatic pruritus in adults with primary biliary cholangitis, a rare autoimmune disease. The recommendation was based on the therapy meeting its primary and secondary endpoints in the Phase III GLISTEN trial...