Valoctocogene Roxaparvovec Phase III trials will begin before the end of 2017.
In the wake of successful Phase I and Phase II clinical trials, the FDA has granted Breakthrough Therapy Designation status to BioMarin’s investigational gene therapy Valoctocogene Roxaparvovec, formerly BMN 270.
Noting the impact of the designation, Hank Fuchs, President, Worldwide Research and Development at BioMarin commented, "The news of the FDA granting Breakthrough Therapy Designation coupled with EU PRIME designation granted in early 2017 by EMA, demonstrates the strong support of global health authorities for valoctocogene roxaparvovec and its expedited development and registration pathway. There is a tremendous need to achieve normal steady state Factor VIII levels to eliminate spontaneous bleeding, to avoid the complications of suboptimally corrected bleeding disorder, to improve quality of life and enable patients to live to their fullest potential."
Breakthrough Designation is granted when clinical trials have proven successful; drug development is thus expedited to provide a therapeutic answer to a serious condition.
According to BioMarin, the IND application included 52-week data for two doses of the therapy, at the 6e13 vg/kg dose; protocol for the second Phase III study using the 4e13 vg/kg dose was also given to the FDA. BioMarin plans to begin global Phase III enrollment before the year ends.
