BioMarin Pharmaceutical has commenced a Phase III study of pegylated recombinant phenylalanine ammonia lyase (PEG-PAL) for the treatment of phenylketonuria (PKU), a genetic disorder.
Around 90 treatment-naïve adult PKU subjects will self-administer PEG-PAL as part of the open-label, multi-centre study, which is designed to assess the drug’s safety and tolerability during induction, titration and maintenance dosing.
The study’s secondary objective is to assess blood Phe levels.
BioMarin chief medical officer Dr Hank Fuchs said the previous Phase II trial demonstrated a dramatic reduction in blood Phe levels due to PEG-PAL, and a similar outcome is expected of the Phase III programme.
"Adult patients with PKU and patients on the severe end of the disease spectrum still represent a very high unmet medical need," Fuchs said.
"With PEG-PAL, it may be possible to provide a treatment benefit to this population."
On completion of study, the subjects are expected to be enrolled in a second Phase III study, along with 60 subjects currently being treated with PEG-PAL in the Phase II programme.
Designed to determine the efficacy and safety of PEG-PAL in self-administered by PKU adults, the primary objective of this second Phase III double-blind, placebo-controlled study will be to evaluate blood Phe levels.
The secondary objective will be to evaluate changes in neuropsychiatric assessments as measured by the Inattentive portion of the Attention Deficit and Hyperactivity Disorder Rating Scale and the Profile of Mood States.