Company: BioMarin Pharmaceutical ($BMRN)
Based: Novato, CA
R&D budget: $147.3M (€109.3M)
Change from 2009: +30.1%
Net sales spent on R&D: 39%

Near the beginning of this year, BioMarin was touting the fact that it had launched one of the biggest enzyme replacement therapy trials ever, a Phase III for BMN-110 for a lysosomal storage disorder. That was one of six clinical trial programs the biotech has under way, with another preclinical drug being readied for its first human trial early next year.

The developer specializes in rare or carefully defined diseases, with clinical trials under way for phenylketonuria (or PKU, when an infant can't break down an amino acid called phenylalanine), Morquio Syndrome, Pompe disease, genetically defined cancers and more.

The biotech bought out an Irish biologics manufacturing facility from Pfizer earlier this year as it expanded its own facilities in Novato, CA. The developer hasn't been shy about using its check book to help feed the pipeline. In 2009/2010 BioMarin acquired a string of small developers that promised to add to its pipeline. The biotech agreed to pay up to $115 million to snare ZyStor Therapeutics and its preclinical candidate for Pompe disease. Lead Therapeutics cost $18 million while Huxley Pharmaceuticals agreed to a $15 million buyout.

Not all of its deals have worked in its favor, though. A $7.5 million pact to acquire a stake in the ill-fated La Jolla Pharmaceutical blew up after a study of its lupus drug failed.