Pfizer's Hemophilia Therapy Study Meets Key Targets
December 29 2022 - 7:55AM
Dow Jones News
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 dean.seal@wsj.com
(END) Dow Jones Newswires
December 29, 2022 07:40 ET (12:40 GMT)
Copyright (c) 2022 Dow Jones & Company, Inc.
Pfizer (NYSE:PFE)
Historical Stock Chart
From Mar 2024 to Apr 2024
Pfizer (NYSE:PFE)
Historical Stock Chart
From Apr 2023 to Apr 2024
