BioMarin

BioMarin Receives the US FDA’s Approval Allowing Maximum Dose of 60mg with Palynziq (pegvaliase-pqpz) for PKU

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Shots: The US FDA has approved the sBLA to increase the maximum allowable dose of 60mg with Palynziq for PKU. Previously, the maximum dose was 40mg The label expansion is based on OLE study out to 3yrs. demonstrating that 66% had a blood Phe level… Read More »BioMarin Receives the US FDA’s Approval Allowing Maximum Dose of 60mg with Palynziq (pegvaliase-pqpz) for PKU

BioMarin Reports NDA Submission to the US FDA for Vosoritide to Treat Children with Achondroplasia

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Shots: BioMarin has submitted an NDA to the US FDA for vosoritide to treat children with achondroplasia The company also reported that EMA has begun validation of MAA for the therapy on 13 Aug, 2020 Vosoritide is an investigational, once-daily injection analog of C-type Natriuretic… Read More »BioMarin Reports NDA Submission to the US FDA for Vosoritide to Treat Children with Achondroplasia

BioMarin Reports the Submission of MAA to EMA for Vosoritide to Treat Children with Achondroplasia

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Shots: The MAA is based on P-III study assessing the efficacy and safety of vosoritide, further supported by the long-term safety and efficacy from the ongoing P-II and P-III extension studies and extensive natural history data Following the completion of EMA’s validation, BioMarin expects the… Read More »BioMarin Reports the Submission of MAA to EMA for Vosoritide to Treat Children with Achondroplasia