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BIOMARIN PHARMACEUTICAL INC logo

BMRN

NASDAQNOVATO, CA
BIOMARIN PHARMACEUTICAL INC

BioMarin develops and commercializes therapies for rare genetic diseases, with an approved product portfolio that includes BRINEURA, KUVAN, NAGLAZYME, PALYNZIQ, ROCTAVIAN, VIMIZIM, and VOXZOGO. The excerpt provided does not contain sufficient detail about specific therapeutic areas, modalities, or development-stage programs to characterize those aspects accurately.

$53.91-15.36%1Y
BMRN · daily close · illustrative · 0 catalysts marked
1Y high$64.081Y low$51.46range$12.62(25%)past catalysts

Pipeline11

Pegvaliase
Phase 3Phenylketonuria (PKU)

Pegvaliase-pqpz is a PEGylated phenylalanine ammonia lyase (PAL) enzyme that converts phenylalanine to ammonia and trans -cinnamic acid.

small-molecule
Valoctocogene roxaparvovec
Phase 3Hemophilia A
Phase 2Hemophilia A With Inhibitor
Phase 2Hemophilia A With Anti Factor VIII

Valoctocogene roxaparvovec-rvox is an adeno-associated virus serotype 5 (AAV5) based gene therapy vector, designed to introduce a functional copy of a transgene encoding the B-domain deleted SQ form of human coagulation factor VIII (hFVIII-SQ).

small-molecule
vosoritide
Phase 3Hypochondroplasia
Phase 2Achondroplasia

In patients with achondroplasia, endochondral bone growth is negatively regulated due to a gain of function mutation in fibroblast growth factor receptor 3 ( FGFR3 ).

small-molecule
BMN 333
Phase 2/3Achondroplasia
small-molecule
Vosoritide Injection [Voxzogo]
Phase 2/3Achondroplasia
small-molecule
Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Phase 2Achondroplasia
small-molecule
BMN 111
Phase 2Achondroplasia
small-molecule
BMN 307
Phase 2Phenylketonuria (PKU)
small-molecule
BMN 351
Phase 2Duchenne Muscular Dystrophy
small-molecule
Human Growth Hormone
Phase 2Idiopathic Short Stature
small-molecule
Vosoritide Injection
Phase 2Short Stature Homeobox- Containing Gene SHOX Deficiency
Phase 2Noonan Syndrome
Phase 2Turner Syndrome
Phase 2Idiopathic Short Stature
small-molecule

Catalyst Calendar

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