Ticker: BMRN
Company summary: BioMarin focuses on developing treatments for rare diseases. It recently launched Palynziq, which treats adult phenylketonuria, a rare inherited disorder in which the body can't process an amino acid called phenylalanine. Its pipeline also includes vosoritide (treats achondroplasia, a type of short-limbed dwarfism), val-rox (hemophilia A gene therapy), and BMN250 (treats Sanfilippo Syndrome B).
Market cap: $17.6 billion