Pfizer scores win in hemophilia B gene therapy study

Pfizer's hemophilia B gene therapy that's designed for the patient to produce factor IX, a blood-clotting protein, was effective and safe in a phase 3 study, the drugmaker said Dec. 29. 

The experimental therapy, fidanacogene elaparvovec, reduced the annualized bleeding rate of total bleeds by 71 percent. The trial compared the one-time gene therapy treatment to the current treatment for the rare blood disorder, which is a prophylaxis regimen. 

Pfizer said it will share the data with regulators in 2023. 

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