Pfizer Reports the US FDA Acceptance of BLA for Fidanacogene Elaparvovec to Treat Hemophilia B
Shots:
- The submission was based on the P-III study (BENEGENE-2) evaluating fidanacogene elaparvovec in 55 patients aged 18–65yrs. The trial met its 1EPs of non-inferiority & superiority in ABR of total bleeds post-fidanacogene elaparvovec infusion over a prophylaxis regimen with FIX
- Superiority with a mean ABR for all bleeds of 1.3 for 12mos. from 12-15wks. vs 4.43 during the lead-in pre-treatment period of 6mos., 71% reduction in ABR after a single dose. In the 2EPs, 78% reduction in ABR & 92% in annualized infusion rate; 27% mean FIX activity @15mos. by one-stage SynthASil assay & 25% @24mos., mean steady-state FIX: C was higher over the pre-specified threshold
- The therapy was well-tolerated with a safety profile consistent with P-I/II results. The MAA for fidanacogene elaparvovec is under EMA’s review
Ref: Pfizer | Image: Pfizer
Related News:- Pfizer Reports P-III Study (BENEGENE-2) Results of Fidanacogene Elaparvovec for Hemophilia B
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