BioMarin Drug for Genetic Disorder Succeeds in late-stage study
Tuesday, March 22, 2016
BioMarin Pharmaceutical Inc said preliminary data showed its experimental drug met the main goal of reducing blood phenylalanine in patients with a genetic disorder, in a late-stage study.
The drug, pegvaliase, is being tested against a placebo in patients with phenylalanine hydroxylase (PAH) deficiency, a disorder diagnosed in about 50,000 patients in the developed world, the company said.
Source : reuters.com
