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BioMarin's hemophilia gene therapy granted EMA accelerated assessment


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BioMarin's hemophilia gene therapy granted EMA accelerated assessment

BioMarin Pharmaceutical Inc.'s experimental hemophilia gene therapy has been granted accelerated assessment by the European Medicines Agency, ahead of the rare disease drug developer's planned application for approval.

During its Oct. 23 third-quarter earnings call, BioMarin executives said the therapy, called Valrox, will be submitted for European and U.S. approval in the fourth quarter.

If approved, Valrox could be launched in the second half of 2020, BioMarin said in its earnings release.

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San Rafael, Calif.-based BioMarin's shares were up about 4.3% to $72.00 in after-hours trading Oct. 23.

Valrox is intended to replace hemophilia A patients' missing factor VIII levels and thus reduce bleeding. According to a three-year study, patients treated with Valrox saw a 96% reduction in mean annualized bleed rate. Factor VIII levels in the study participants appeared to fall over three years, though analysts noted that the levels may have stabilized and reached a plateau at year three.

BioMarin said Valrox's effects should be sustained over eight or more years.

Currently, the standard of care for hemophilia A is factor VIII infusions, typically administered two to three times a week.

Accelerated assessment, granted by the EMA to medicines of major public health interest, speeds up the time it takes for the Committee for Medicinal Products for Human Use to review an application.