BioMarin Announces First Patient Dosed in Valoctocogene Roxaparvovec Study

BioMarin Pharmaceutical has dosed the first patient in a Phase 1/2 study (BMN 270-203) evaluating its investigational gene therapy, valoctocogene roxaparvovec, in severe hemophilia A patients with pre-existing AAV5 antibodies.

"Administration of valoctocogene roxaparvovec to this first patient seropositive for the AAV5 capsid is an important next step in our plan to expand the number and types of severe hemophilia A patients who may benefit from gene therapy and have antibodies to the vector," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "The goal with this study is to determine if patients that already have antibodies to AAV5 can be effectively treated with valoctocogene roxaparvovec. Our objective is to develop a therapy with the potential to eliminate the need for chronic treatment in severe hemophilia A across all patient sub-groups."

The study is an open-label, single-arm, titer-escalation trial evaluating the safety and efficacy of valoctocogene roxaparvovec in AAV5+ hemophilia A patients. Patients with pre-existing AAV5 antibodies will be sequentially enrolled into two titer cohorts that will encompass the range of observed AAV5 antibody titer levels generally observed in the hemophilia population and be treated with the 6e13 vg/kg dose. The primary endpoint will evaluate safety of valoctocogene roxaparvovec in this population. Secondary endpoints include assessment of FVIII activity level, frequency of required FVIII replacement therapy, and the number of bleeding episodes requiring treatment after therapy.

BioMarin is also evaluating the 6e13 vg/kg dose in GENEr8-1 and a second dose of 4e13 vg/kg in GENEr8-2, both global Phase 3 studies in patients without pre-existing AAV5 antibodies.

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