BioMarin Pharmaceutical Inc’s drug met a study’s main goal of reducing a toxic buildup of an amino acid in the blood of patients with a genetic disorder, but did not show enough benefit in related inattention and mood complications. BioMarin’s shares fell as much as 6 percent in early trading on Monday after the company released preliminary data from the late-stage study, which is testing the drug against a placebo in patients of phenylalanine hydroxylase (PAH) deficiency. The injectable drug, pegvaliase, reduced phenylalanine levels by about 62 percent compared with a placebo, but did not improve inattention or mood scores even though it showed a trend towards neurocognitive improvement.
Original post:
BioMarin drug fails to show inattention, mood benefit in study
