With ‘break­through’ sta­tus and a handy nick­name, Bio­Marin plots speedy PhI­II for gene ther­a­py

The FDA has hand­ed out its break­through ther­a­py des­ig­na­tion for Bio­Marin’s close­ly-watched gene ther­a­py drug BMN 270 for he­mo­phil­ia A.

In their Q3 call on Thurs­day CEO Jean-Jacques Bi­en­aimé out­lined the biotech’s Phase III game plan for this drug, which now has a nick­name — val­rox — as well as high ex­pec­ta­tions for a rel­a­tive­ly speedy trip down the aisle to reg­u­la­tors.

Bio­Marin $BM­RN is kick­ing off en­roll­ment for two piv­otal stud­ies with two dif­fer­ent dos­es. The en­roll­ment for the first study should be done by year end 2018, with piv­otal da­ta com­ing on that a year lat­er.

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