Pfizer launches phase 3 gene therapy study in hemophilia B

Pfizer has begun early work on a phase 3 study of an investigational gene therapy to treat hemophilia B.

Pfizer, along with Spark Therapeutics, launched a phase 3 lead-in study at multiple centers to evaluate the efficacy and safety of current factor IX prophylaxis replacement therapy in the usual care setting. The efficacy data from the lead-in study will become the within-subject control group for patients who enroll in the next part of the phase 3 study, which will evaluate fidanacogene elaparvovec (formerly SPK-9001) for the treatment of hemophilia B.

Fidanacogene elaparvovec is a vector containing a bioengineered adeno-associated virus capsid and a high-activity human coagulation factor IX gene. In an ongoing phase 1/2 trial of fidanacogene elaparvovec, all 15 participants with hemophilia B were able to discontinue routine infusion of factor IX concentrates without serious adverse events, according to data released by Pfizer and Spark Therapeutics in May 2018.

mschneider@mdedge.com