BioMarin’s ROCTAVIAN Shows Sustained Efficacy, Safety Over 5 Years for Severe Hemophilia A

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the most undervalued US stocks according to analysts. On June 24, BioMarin Pharmaceutical Inc. presented new 5-year data from its Phase 3 GENEr8-1 trial at the 33rd Congress of the International Society on Thrombosis and Haemostasis/ISTH in Washington, D.C., which took place from June 21 to 25.

The data reinforced the long-term efficacy and safety of ROCTAVIAN (valoctocogene roxaparvovec-rvox) as a gene therapy for severe hemophilia A. The GENEr8-1 trial is the longest and largest hemophilia gene therapy study to date, and demonstrated sustained Factor VIII (FVIII) expression and durable bleed control 5 years after a single treatment with ROCTAVIAN.

BioMarin's ROCTAVIAN Shows Sustained Efficacy, Safety Over 5 Years for Severe Hemophilia A

A pharmaceutical plant manufacturing a proprietary synthetic oral form of a C-type natriuretic peptide.

FVIII activity remained consistent with previously reported results. Importantly, no new safety signals were observed over the 5-year study period. Across all 134 participants who received ROCTAVIAN in the study, there were no cases of FVIII inhibitors or thromboembolic events, and no treatment-related malignancies were observed.

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is a biotechnology company that develops and commercializes therapies for life-threatening rare diseases and medical conditions internationally.

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Disclosure: None. This article is originally published at Insider Monkey.