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NICE refuses to endorse Biomarin’s Batten disease drug pharmaceutical-technology
February 26, 2019
The UK National Institute for Health and Care Excellence (NICE) has published a draft guidance saying that Biomarin’s therapy for Batten disease, Brineura (cerliponase alfa), cannot be recommended for use in the National Health Service (NHS)....
Gene therapy firm BioMarin stages haemophilia musical pharmaphorum
November 27, 2018
BioMarin Pharmaceutical has put on a stage musical to raise awareness about haemophilia, and determine whether creative expression helps patients, as it develops an experimental cure for the condition.
BioMarin-sponsored hemophilia musical debuts with teen singers, writers and actors fiercepharma
November 15, 2018
It’s showtime for “Hemophilia the Musical,” a BioMarin-sponsored show created by teens.
US clears first enzyme therapy for PKU pharmatimes
July 11, 2018
Biomarin’s Palynziq has been approved as the first enzyme therapy to treat the rare brain-threatening genetic disease phenylketonuria (PKU) in adults in the US.
BioMarin scores long-sought FDA nod for Palynziq, but its $192K launch could be tough fiercepharma
May 28, 2018
More than two years after turning in disappointing results from a phase 3 trial of Palynziq to treat the rare inherited blood disorder phenylketonuria (PKU), BioMarin finally won FDA approval for the drug Thursday night.
BioMarin Announces First Patient Dosed in Valoctocogene Roxaparvovec Study americanpharmaceuticalreview
May 18, 2018
BioMarin Pharmaceutical has dosed the first patient in a Phase 1/2 study (BMN 270-203) evaluating its investigational gene therapy, valoctocogene roxaparvovec, in severe hemophilia A patients with pre-existing AAV5 antibodies.
FDA approves BioMarin’s manufacturing facility in Cork, Ireland europeanpharmaceuticalreview
June 19, 2017
The FDA has approved BioMarin Pharmaceutical’s bulk biologics manufacturing plant, located in Cork, Ireland for production of the formulated bulk substance.
BioMarin Announces Kuvan Patent Challenge Settlement americanpharmaceuticalreview
April 14, 2017
BioMarin Pharmaceutical Inc. announced it has entered into a settlement agreement with Par Pharmaceutical that resolves patent litigation in the United States (U.S.) related to BioMarin's Kuvan (sapropterin dihydrochloride) 100mg oral tablets and powder f

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