BioMarin’s $1m Gene Therapy Roctavian Hits Payer Problems In Germany
Doubts About Blockbuster Prospects Linger
BioMarin has yet to treat a patient with Roctavian, the first ever hemophilia A gene therapy, eight months after its first approval in Europe, as pricing delays cloud its commercial prospects.
You may also be interested in...
BioMarin, Sarepta and bluebird bio are all launching new gene therapies in the US. The drug makers updated investors on the commercial execution during second quarter financial earnings calls.
BioMarin has finally brought its hemophilia A gene therapy Roctavian market and is determined prove naysayers wrong, but must work with its limitations and a confusing US label.
The drug showed efficacy in hemophilia A and B with no sign of clots, an issue that historically has dogged anticoagulation agents, but it remains unclear whether that advantage will hold.