BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc. specializes in creating and bringing to market treatments for individuals suffering from severe and often fatal rare disorders and other critical medical conditions. The company currently markets several key pharmaceutical products.

Among these are Vimizim, an enzyme replacement therapy targeting mucopolysaccharidosis (MPS) IV type A, a specific lysosomal storage disorder, and Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for individuals with MPS VI. Another notable offering is Kuvan, a unique synthetic oral version of 6R-BH4, prescribed for phenylketonuria (PKU), an inherited metabolic condition.

Further expanding its product portfolio, BioMarin provides Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme administered via subcutaneous injection to lower blood Phe levels.