With new piv­otal da­ta, uniQure and CSL Behring dash to the FDA with a po­ten­tial block­buster gene ther­a­py for he­mo­phil­ia B

Af­ter a five-month hold put a damper on uniQure’s he­mo­phil­ia B gene ther­a­py pro­gram, things are fi­nal­ly look­ing up as a pos­i­tive piv­otal read­out puts the com­pa­ny and part­ner CSL Behring on track to file for ap­proval in the first half of next year — and an­a­lysts spy block­buster po­ten­tial.

The com­pa­nies’ po­ten­tial first gene ther­a­py for he­mo­phil­ia B, known as etranaco­gene deza­parvovec, met its pri­ma­ry non-in­fe­ri­or­i­ty end­point in an­nu­al­ized bleed­ing rate (ABR) af­ter 18 months com­pared to base­line Fac­tor IX pro­phy­lac­tic ther­a­py, uniQure an­nounced on Thurs­day. Fac­tor IX is a pro­tein that’s nat­u­ral­ly pro­duced in the body to help form blood clots and stop bleed­ing, and com­mon treat­ments to­day are de­signed to re­place the pro­tein to achieve ad­e­quate clot­ting.

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