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BioMarin's Long-term Clinical Data On Severe Hemophilia A Show Continued, Clinical Benefit

BioMarin Pharmaceutical Inc.'s (BMRN) long-term clinical data from the ongoing phase 1/2 study of valoctocogene roxaparvovec in adults with Severe Hemophilia A demonstrated continued, durable clinical benefit.

BioMarin announced the new data from its open-label Phase 1/2 study during an oral presentation at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress.

Five-year and four-year post-treatment follow-up of the 6e13 vg/kg and 4e13 vg/kg cohorts, respectively, showed a sustained treatment benefit of valoctocogene roxaparvovec. All participants in both cohorts remain off prophylactic Factor VIII treatment.

The company noted that phase 1/2 and subset of pivotal GENEr8-1 studies demonstrated consistent bleed control following treatment with valoctocogene roxaparvovec.

Overall, the safety profile of valoctocogene roxaparvovec in the Phase 1/2 study remains consistent with previously reported data with no delayed-onset treatment related adverse events. All participants continue to remain off corticosteroids since the first year. No participants developed inhibitors to Factor VIII, and no participants withdrew from the study. No participants have developed thrombotic events, the company said.

The most common adverse events associated with valoctocogene roxaparvovec occurred early after a single infusion and included short-lived infusion-associated reactions and transient, asymptomatic, and mild to moderate rise in the levels of certain proteins and enzymes measured in liver function tests with no long-lasting clinical sequelae.

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