BioMarin Has Mixed Longer Data on Hemophilia Gene Therapy

 BioMarin announced one-year follow-up data from a phase III study on its pipeline candidate, valoctocogene roxaparvovec in patients with hemophilia A.

With a mean follow-up of 71.6 weeks, data from the phase III study – GENEr8-1 – showed that treatment with a single dose (6e13 vg/kg) of the gene therapy candidate significantly reduced annualized bleeding rate (ABR) by 84% and the mean annualized factor VIII (a clotting protein) infusion rate in the rollover population by 99%. Meanwhile, valoctocogene roxaparvovec demonstrated superiority to current standard of care, FVIII prophylactic therapy. 

In a subset of patients who were dosed more than two years ago, the decline in Factor VIII expression was at a slower rate compared to that at the end of one year with continued hemostatic efficacy.

https://finance.yahoo.com/news/biomarin-bmrn-reveals-1-data-133701387.html