BioMarin Pharmaceutical Inc.

Potential blockbuster drugs to watch in 2023

10 January 2023 | By Catherine Eckford (European Pharmaceutical Review)

Lecanemab for Alzheimer's and the first potential gene therapy primed for US launch for haemophilia A are two potential blockbuster Drugs to Watch™, says a 2023 report.

news

Europe approves first gene therapy for treatment of haemophilia A

30 August 2022 | By Caroline Peachey (European Pharmaceutical Review)

European approval of BioMarin’s ROCTAVIAN (valoctocogene roxaparvovec) one-time gene therapy represents a breakthrough in the treatment of adults with severe haemophilia A.

news

FDA approves Palynziq for PKU, a rare and serious genetic disease

28 May 2018 | By European Pharmaceutical Review

Novel enzyme therapy for adults with PKU who have uncontrolled blood phenylalanine concentrations with current treatment...

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Recommended

BioMarin Pharmaceutical Inc.

Potential blockbuster drugs to watch in 2023

Europe approves first gene therapy for treatment of haemophilia A

FDA approves Palynziq for PKU, a rare and serious genetic disease