Pfizer's Hemophilia Therapy Study Meets Key Targets

By Dean Seal

Pfizer Inc. said a Phase 3 study of its investigational gene therapy for the treatment of the blood disorder hemophilia B in adult men met its primary endpoint in reducing bleeding.

The company said Thursday that the study demonstrated non-inferiority and superiority in the annualized bleeding rate of total bleeds after an infusion of fidanacogene elaparvovec, the gene therapy candidate, when compared with a prophylaxis regimen administered as part of usual care.

Key secondary endpoints showed a 78% reduction in annualized bleeding rate and a 92% reduction in annualized infusion rate, Pfizer said.

Fidanacogene elaparvovec was also generally well tolerated and had a safety profile consistent with prior results. A total of 14 serious adverse events were reported in seven, or 16%, of the patients, two of which were assessed as being related to treatment. No deaths were reported.

Pfizer said it will discuss the data with regulatory authorities early next year.

Write to Dean Seal at [e-mail]

(END) Dow Jones Newswires

December 29, 2022 07:40 ET (12:40 GMT)

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