Browsing Tag
BioMarin Pharmaceutical Inc.
4 posts
BioMarin’s BMN 401 setback raises regulatory questions after ENPP1 deficiency trial meets only one endpoint
BioMarin moved the biomarker, not the rickets score. BMN 401 now tests how much regulators will accept in ultra-rare disease trials.
BioMarin Pharmaceutical (Nasdaq: BMRN) secures FDA approval to expand PALYNZIQ use in adolescents with phenylketonuria
Find out how BioMarin Pharmaceutical Inc.’s FDA approval for adolescent PALYNZIQ use could reshape PKU treatment and long-term disease control.
FDA grants priority review for BioMarin’s PALYNZIQ to treat adolescents with phenylketonuria (PKU)
Find out how BioMarin’s PALYNZIQ priority review could transform PKU treatment for adolescents and reshape the company’s rare-disease strategy.
BioMarin shares rise as new VOXZOGO and PALYNZIQ data reinforce market potential
Discover how BioMarin’s latest clinical data on VOXZOGO and PALYNZIQ at ACMG 2025 offers promising insights into rare disease treatments and what it means for the market.