Hemophilia B competition builds with Pfizer gene therapy data

Phase III data from a second hemophilia B gene replacement therapy suggest competition may be on the way for the recently approved, high-priced Hemgenix, and provide additional insight into the level of protein activity needed to achieve clinical benefit.

Last week, Pfizer Inc. (NYSE:PFE) reported top-line data from fidanacogene elaparvovec, an in vivo gene replacement therapy in development for hemophilia B. Complications of cross-trial comparisons aside, the new data suggest comparable lowering of annualized bleeding rates, bleeding events that required treatment and annualized infusion rates, versus Hemgenix etranacogene dezaparvovec from CSL Ltd. (ASX:CSL) and uniQure N.V. (NASDAQ:QURE)...