FDA, EMA ac­cept Pfiz­er's he­mo­phil­ia B gene ther­a­py, set­ting up show­down with CSL Behring

Pfiz­er said Tues­day that US and Eu­ro­pean reg­u­la­tors have ac­cept­ed its fil­ings seek­ing ap­proval for a gene ther­a­py that re­duces bleed rates in he­mo­phil­ia B pa­tients — and that may com­pete with CSL Behring’s Hem­genix.

The FDA gave fi­danaco­gene ela­parvovec a PDU­FA date in the sec­ond quar­ter of next year, ac­cord­ing to the drug­mak­er.

In De­cem­ber, Pfiz­er re­vealed Phase III da­ta that showed a 71% de­crease in the rate of bleed­ing in pa­tients who re­ceived the ther­a­py. The tri­al, which en­rolled 45 pa­tients, had an av­er­age an­nu­al­ized bleed rate of 1.3 bleeds over 12 months. It met sta­tis­ti­cal sig­nif­i­cance — bring­ing in a p-val­ue of p<0.0001.

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