Swissmedic authorizes CSL's Hemgenix for hemophilia B

Medicines regulator Swissmedic has authorized Hemgenix (etranacogene dezaparvovec), from Australian biotech CSL Limited (ASX: CCSL), the first and currently only gene therapy for the treatment of male adults aged >18 years with severe or moderately severe hemophilia B (congenital Factor IX deficiency).

Hemgenix, licensed from Dutch gene therapy company uniQure (Nasdaq: QURE), is a one-time, single-dose treatment option that allows people living with hemophilia B to produce their own factor IX, which can lower the risk of bleeding. When first approved in the USA, Hemgenix was dubbed the most expensive medicine in the world, with a list price of around $3.5 million per treatment.

Leerink Partner analysts have previously suggested peak (2030E) non risk-adjusted global sales of $2.4 billion for Hemgenix, of which uniQure is eligible for royalties of percentages in the mid-teens to low-20s.