BioMarin submits for US marketing approval in rare disease

3 July 2017

Rare disease drug developer BioMarin Pharmaceutical (Nasdaq: BMRN) has submitted an application in the USA for approval to market its pegvaliase, a candidate treatment for phenylketonuria (PKU), a genetic disorder.

Pegvaliase is designed to reduce blood phenylalanine (Phe) levels in adult patients with PKU who have uncontrolled levels on existing management.

"We believe that pegvaliase offers the promise of an important new treatment option for those adult patients with PKU unable to manage their condition with existing treatments,” said global R&D head Hank Fuchs.

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