FDA rejects hemophilia gene therapy

The U.S. Food and Drug Administration issued a Complete Response Letter to the BioMarin Pharmaceutical’s Biologics License Application for valoctocogene roxaparvovec gene therapy for severe hemophilia A, according to a press release.

Possibly approval is likely delayed till late in 2022.

“We remain committed to the hemophilia community and to leading the way to the first ever gene therapy in hemophilia A,” said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin in the press release.  “We are surprised and disappointed that the FDA introduced new expectations for the first time in the Complete Response Letter.  We are confident in valoctocogene roxaparvovec gene therapy and its potential to redefine the treatment paradigm for people with hemophilia A.”

Read the full press release here.