BioMarin advances drugs for amino acid disease PKU, dwarfism

BioMarin's Jean-Jacques Bienaime.

BioMarin Pharmaceutical Inc. will take a mid-stage drug for the treatment of a rare phenylketonuria into Phase III testing, the company said Wednesday. Novato-based BioMarin (NASDAQ: BMRN) said preliminary results from its four Phase II trials of pegylated recombinant phenylalanine ammonia lyase, or PEG-PAL, showed it was well tolerated and could prove efficacious. The Phase III program, generally the final stage of clinical trials before seeking Food and Drug Administration approval for a drug, will begin in second-quarter 2013.