WASHINGTON (dpa-AFX) - BioMarin Pharmaceutical (BMRN) announced new data
supporting the long-term safety and efficacy of ROCTAVIAN. The company said the
data from the longest Hemophilia Gene Therapy Study showed that durable and
sustained bleed control and factor VIII expression maintained four years
Post-ROCTAVIAN Infusion.

Hank Fuchs, president of Worldwide Research and Development at BioMarin, said:
'Importantly, these Phase 3 data also indicate a plateauing of factor VIII
levels after year three with the majority of patients remaining off prophylaxis,
which shows ROCTAVIAN can offer long-term bleed protection for adults with
severe hemophilia A and may provide relief from the burden of chronic infusions
and injections.'

ROCTAVIAN is an adeno-associated virus vector-based gene therapy used for the
treatment of adults with severe hemophilia A who do not have antibodies to
adeno-associated virus serotype 5, which is determined by a blood test. The
European Commission granted conditional marketing authorization to ROCTAVIAN on
August 24, 2022. The FDA approved ROCTAVIAN on June 29, 2023.

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