Hemophilia and other blood disorders like sickle cell disease and beta thalassemia have drawn intense investment from genetic medicine developers. in December, the FDA approved two such treatments for sickle cell, clearing Vertex Pharmaceuticals’ gene editing drug Casgevy and Bluebird bio’s viral vector therapy Lyfgenia.
In hemophilia B — the more uncommon of the two types of the bleeding disorder — Hemgenix has been on the U.S. market since November 2022, where it has a list price of $3.5 million. Australia-based CSL, which licensed the treatment from gene therapy pioneer UniQure, hasn’t broken out sales revenue from Hemgenix in its most recent financial reports.
