Pfizer gene therapy reduced hemophilia bleeds, but questions remain

Pfizer said Wednesday that its gene therapy for hemophilia A successfully reduced patients’ bleeds for at least 15 months in a large Phase 3 trial.

The data may be enough to muster approval, but they are unlikely to quell doubts about whether the treatment can provide a cure — as had once been hoped — or a meaningful option for most patients in the U.S. and Europe with the rare blood disorder.

“There’s a primary endpoint, and then there’s what will predict meaningful commercial success,” said Lindsey George, head of in vivo gene therapy at Children’s Hospital of Philadelphia.

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