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.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER