BioMarin’s Hemophilia Gene Therapy Still Works After Four Years, But Effects Tail Off
FDA Approval Expected By Late 2020
Latest results show a show decline in effects, but still offers freedom from injections with almost no bleeds recorded in 13 patients.
You may also be interested in...
A PwC analysis finds biopharma aggregate deal value declined 87% during the first half compared to the second half of 2018; deal volume dropped 17%. The pandemic was a cause, but not the only factor.
Gene and cellular therapies that can replace decades of expensive therapy do not come cheap. Even the initial remedy of payment by installments could be irrelevant when not all patients respond in the same way.
Sanofi has unveiled encouraging Phase II data for fitusiran, a novel RNA-based hemophilia therapy which could eventually challenge Roche’s blockbuster Hemlibra.