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BioMarin Receives FDA Fast Track Designation for PKU Investigational Gene Therapy americanpharmaceuticalreview
October 12, 2020
BioMarin Pharmaceutical announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to BMN 307, an investigational gene therapy for the treatment of individuals with PKU.
Censa Announces CNSA-001 Met Primary and Secondary Endpoints americanpharmaceuticalreview
December 27, 2019
Censa Pharmaceuticals has announced positive results from its Phase 2 trial for CNSA-001 (sepiapterin), a potential new medicine for the treatment of phenylketonuria (PKU).
BioMarin Submits CTA in UK for Investigational Gene Therapy for PKU americanpharmaceuticalreview
September 27, 2019
BioMarin Pharmaceutical has submitted a Clinical Trial Application (CTA) with the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. for BMN 307 ...
Sapropterin Dihydrochloride Tablets 100 mg drugs
June 17, 2019
Sapropterin Dihydrochloride Tablets 100 mg.
EU approval for Palynziq in adult phenylketonuria pharmatimes
May 09, 2019
The European Commission has approved BioMarin’s Palynziq (pegvaliase injection) for the treatment of phenylketonuria (PKU) in patients aged 16 years or older.
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.
FDA approves BioMarin’s enzyme substitution therapy to treat PKU pharmaceutical-technology
June 01, 2018
The US Food and Drug Administration (FDA) has approved pharmaceutical company Biomarin’s Palynziq to treat phenylketonuria (PKU).
FDA approves Palynziq for PKU, a rare and serious genetic disease europeanpharmaceuticalreview
May 29, 2018
Novel enzyme therapy for adults with PKU who have uncontrolled blood phenylalanine concentrations with current treatment…