FDA, Gene Therapy

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.

Jean-Jacques Bienaimé

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.

“Coupled with the EU prime designation granted earlier this year by the EMA, (the BTD) demonstrates a strong support of Global Health Authority for our product and its expedited development,” said the CEO.

Much of the gene therapy work in this field has focused on hemophilia B, but BioMarin isn’t alone. Pfizer signed up for a partnership with Sangamo earlier this year, though they are well behind BioMarin in the hunt.

Their key data so far has been good to great.

Excluding one of the patients in the high dose group, leaving 6 for analysis, the mean annualized bleeding rate was reduced by 97% — from 16.3 to 0.5. “The median ABR for those same patients was reduced from 16.5 to zero. The mean annualized Factor VIII infusions were reduced by 94% from 136.7 to 8.5. The median annualized Factor VIII infusions were reduced from 138.5 to zero.” And the lower, mid-range dose also demonstrated an excellent score on bleeding and infusion rates, with a drop to zero.


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