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.
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Pfizer’s Marstacimab Wins In Phase III, But Caution On Market Position, Clotting Remain
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.
BridgeBio’s Achondroplasia Data Portend Competition With BioMarin’s Voxzogo
Already planning to position infigratinib as a safer, more convenient oral therapy for dwarfism compared with the BioMarin injectable, BridgeBio now has Phase II efficacy data showing a higher growth rate.
Sanofi’s Altuviiio Set To Reenergize Factor VIII Space
The once-weekly Factor VIII replacement therapy for hemophilia A, newly approved by the US FDA, appears well positioned to compete with Roche’s blockbuster Hemlibra.