BioMarin Pharmaceuticals

News

Priority review sets up Feb verdict on Sanofi, Sobi haemophi...

The FDA will carry out a speedy review of Sanofi and Sobi's once-weekly haemophilia A treatment efanesoctocog alfa (Bivv001), with a decision now

News

Roctavian okay sets up another gene therapy test for Europe

BioMarin Pharma has its landmark first approval for Roctavian – the first for a haemophilia gene therapy – and will now have to see if that can convert that into a viable business in Europe

Patients

Engaging change: Connecting patients and regulators can move...

Face-to-face discussion educates regulators on what matters most to patients, and guides future decision-making.

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