Latest news

• EU nod for BioMarin’s second PKU therapy Palynziq

  Palyniq is significant, but gene therapy pipeline most anticipated. BioMarin has gained European Union approval for Palynziq (pegvaliase injection) for the rare metabolic condition phenylketonuria (PKU). ... The Palynziq approval was based on data from

• CHMP backs Lynparza in breast cancer, Dupixent in asthma

  Meanwhile, BioMarin got the nod for Palynziq (pegvaliase), a new enzyme replacement therapy for patients aged 16 and older with phenylketonuria, a rare but potentially serious inherited metabolic disease.

• BioMarin prepares for haemophilia A gene therapy data readout, filing

  The company expects to hit revenues of $2bn by 2020, helped on by another new product, phenylketonuria therapy Palynziq, which is also forecast to eventually hit peak annual sales of up

• BioMarin eyes 2019 FDA filing for haemophilia A gene therapy

  BioMarin already has seven drugs on the market, including recent launch Palynziq (pegvaliase) for phenylketonuria, and is steadily moving towards its objective of pushing sales above $2bn by 2020, recently predicting

• Daily Brief: GDPR and NHS Opt Out Day, BioMarin's new rare disease approval and more

  records. NEW RARE DISEASE APPROVAL. BioMarin sees blockbuster potential for newly-approved Palynziq. ... Palynziq is expected to be available in the US by the end of June.