Jean-Jacques Bienaimé, BioMarin chairman and CEO

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

Bio­Marin has post­ed some top-line re­sults for their 4-year fol­lowup on the most ad­vanced gene ther­a­py for he­mo­phil­ia A — ex­tend­ing its streak on keep­ing a hand­ful of pa­tients free of bleeds and off Fac­tor VI­II ther­a­py, but like­ly stir­ring fresh wor­ries over a con­tin­ued drop in Fac­tor VI­II lev­els.

We just don’t know how big a drop.

We’ll see more da­ta when the re­sults are pre­sent­ed at the World Fed­er­a­tion of He­mo­phil­ia in a cou­ple of weeks. But in a state­ment out Sun­day night, Bio­Marin $BM­RN re­port­ed that none of the pa­tients re­quired Fac­tor VI­II treat­ment, adding:

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