JJ Bienaimé, BioMarin CEO (Via YouTube)

New da­ta show Bio­Mar­in's trou­bled he­mo­phil­ia gene ther­a­py con­tin­ues to fade, open­ing room for ri­vals

The ef­fects of Bio­Marin’s vaunt­ed gene ther­a­py for he­mo­phil­ia A are con­tin­u­ing to fade in the ear­li­est treat­ed pa­tients, re­new­ing ques­tions about the long-term prospects for a drug the com­pa­ny had billed as a one-time cure.

At the In­ter­na­tion­al So­ci­ety on Throm­bo­sis and Haemosta­sis meet­ing Wednes­day, Bio­Marin an­nounced that, af­ter five years, the first sev­en pa­tients treat­ed with the ther­a­py’s high dose ex­pressed a me­di­an of just 8.2% the amount of fac­tor VI­II that a healthy per­son would. That’s a small frac­tion of the 60.3% ex­pres­sion pa­tients saw one year af­ter re­ceiv­ing the ther­a­py, known as val­rox.

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